[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myelodysplastic-syndromes\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myelodysplastic-syndromes":29},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,150,0,25,[9,53,84,109,121,145,169,190,218,398,427,452,495,520,544,564,590,609,641,669,703,738,755,774,801],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":41,"lastUpdatePostDateStruct":42,"startDateStruct":45,"completionDateStruct":47,"leadSponsor":49,"locationsCount":52},"100054185","phase-1-pacritinib-a-kinase-inhibitor-of-csf1r-irak1-jak2-and-flt3-in-adults-and-pediatric-participants-12-years-of-age-or-older-with-myelodysplastic-syndromes-or-myelodysplasticmyeloproliferative-neoplasms-100054185",false,"NCT06303193","Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","Phase I\u002FII Trial of Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","* INCLUSION CRITERIA:\n* Participants must have histologically or cytologically confirmed MDS or MDS\u002FMPN, including therapy-related MDS or MDS\u002FMPN, and MDS or MDS\u002FMPN with germline predisposition, by the Department of Laboratory Medicine Hematology Laboratory, CC or by the Laboratory of Pathology, NCI as defined according to the 2016 WHO criteria, 2022 WHO criteria, or 2022 International Consensus Classification\n* Age 12-17 years for phase I and age \\>= 18 years for phase II\n* Participants \\>= 18 years of age with HR-MDS must have resistance to hypomethylating agents as defined as failure to show improvement after at least 4 cycles of treatment (primary resistance) or relapse in participants with initial response to long-term treatment (secondary resistance) OR have intolerance to hypomethylating agents OR have a contraindication to hypomethylating agents\n* Participants \\>= 18 years of age with LR-MDS must be refractory to or ineligible to receive standard of care therapies, i.e. erythropoietin-stimulating agents, lenalidomide, luspatercept, and present with one of the following characteristics:\n\n  * Severe neutropenia defined by absolute neutrophils count \\\u003C=0.5(SqrRoot) 10\\^9\u002FL without the use of granulocyte colony-stimulating factors\n  * Symptomatic anemia defined by hemoglobin 16-week average \\\u003C10 g\u002FdL and symptoms that may include fatigue, weakness, reduced exercise tolerance, dyspnea on exertion, palpitations, (orthostatic) hypotension, near syncope and restless legs\n  * Thrombocytopenia defined as platelets \\\u003C20(SqrRoot) 10\\^9\u002FL or platelets \\\u003C50(SqrRoot) 10\\^9\u002FL and a history of clinically relevant non-major or major bleeding according to the ISTH classification\n* Participants 12-17 years of age with MDS must be relapsed\u002Frefractory OR ineligible to receive immunosuppressive therapy and hematopoietic stem cell transplantation\n\n  --Ineligibility to receive hematopoietic stem cell transplantation will include participants who are not anticipated to be candidates to receive transplantation within the next 3 months due to medical comorbidities, lack of appropriate donor, or logistical barriers to transplant\n* Participants with MDS\u002FMPN must be relapsed\u002Frefractory (failed a minimum of 1 standard of care therapy) OR ineligible to receive standard of care OR without known life-prolonging therapy options OR have a diagnosis for which no known standard of care exists\n* Participants 12-17 years of age must weigh \\>= 35 kg\n* If any of the prior therapies noted below were given to the participant, they must have been completed within the following timeframes:\n\n  * 7 days from last dose of short-acting myeloid growth factors (i.e., filgrastim) and \\>= 14 days for long-acting (i.e., pegfilgrastim)\n  * 14 days from last dose of short-acting thrombopoietic growth factors (i.e.,eltrombopag) and \\>= 28 days for long-acting (i.e., romiplostim)\n  * 14 days or 5 pharmacokinetic half-lives from biological therapy agent\n  * 21 days from myelosuppressive chemotherapy\n  * 28 days from last dose of immunosuppressive therapy (e.g., ATG, cyclosporine, steroids greater than physiologic replacement)\n  * 28 days from last dose of lenalidomide\n  * 28 days from last dose of venetoclax\n  * 28 days from any other investigational agent\n  * 42 days from last dose of erythropoiesis stimulating agents\n  * 56 days from last dose of luspatercept\n  * 100 days from stem cell transplant with no evidence of active graft vs. host disease in participants who relapsed following transplant\n  * 150 days from total body irradiation\n* Performance status:\n\n  * For participants \\>= 16 years of age, ECOG performance status \\\u003C 2 (Karnofsky \\>= 60%)\n  * For participants \\\u003C 16 years of age, Lansky \\>= 60%\n* Participants must have adequate organ function as defined below:\n\n  * Total bilirubin: \\\u003C= 1.5 X institutional upper limit of normal OR \\\u003C= 3 x institutional upper limit of normal in participants with Gilbert s syndrome\n  * AST(SGOT)\u002FALT(SGPT): \\\u003C= 2.5 X institutional upper limit of normal\n  * Creatinine clearance: \\>= 50 mL\u002Fmin\u002F1.73 m\\^2 for participants with creatinine levels above institutional normal\n  * PT and PTT: \\\u003C= 1.5 X institutional upper limit of normal, except in the setting of PTT elevation due to lupus anticoagulant, in which case these participants would be exempt from meeting this inclusion criterion\n* Individuals of child-bearing potential (IOCBP) and individuals able to father a child with a partner able to become pregnant must agree to use one (1) highly effective form of contraception (e.g., intrauterine device \\[IUD\\], surgical) or two (2) effective forms of contraception (e.g., barrier method) while on study drug and for 30 days after the last dose of study drug\n* Nursing participants must discontinue breastfeeding and\u002For not begin breastfeeding until 2 weeks after the last dose of study drug\n* Ability of participant or parent\u002Fguardian (for participants 12-17 only) to understand and the willingness to sign a written informed consent document\n\nEXCLUSION CRITERIA:\n\n* Participants with platelet transfusion-refractory thrombocytopenia, with inability to keep platelet threshold above 10 K\u002FmcL with transfusions\n* Participants with evidence of ongoing hemorrhage, active signs\u002Fsymptoms of bleeding, or history of severe (grade \\>= 3) unprovoked bleeding complications in the one year prior to enrollment, or any unprovoked grade 2 bleeding complications in the 3 months prior\n\nto enrollment\n\n* Use of anti-platelet or anticoagulant medication other than low-dose aspirin (100 mg daily or less) in the 14 days prior to enrollment, or any ongoing requirement for these medications\n* Participants who are unwilling to accept blood transfusions\n* Participants with ANC \\\u003C 500 cells\u002FmcL AND hospitalization for a fungal infection in the 12 months prior to enrollment\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib\n* Concomitant administration with sensitive substrates\u002Fnarrow therapeutic index drugs of CYP3A4, CYP1A2, P-gp BCRP, and OCT1 should be avoided. Concurrent use of strong inhibitors and inducers of CYP3A4 are not allowed. Prior use is allowed as long as medication is stopped two weeks prior to study drug initiation. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment\u002Finformed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.\n* Concomitant administration of medications with significant potential to cause QTc prolongation.\n* Participants with the following cardiac conditions at screening:\n\n  * Symptomatic congestive heart failure\n  * Unstable angina pectoris\n  * Uncontrolled cardiac dysrhythmias\n  * QTc(F) prolongation \\>450 ms, or other factors that increase the risk for QT prolongation (i.e., heart failure, or a history of long QT interval syndrome)\n* Grade \\>= 3 cardiac complication in the 6 months prior to enrollment\n* Left ventricular ejection fraction \\\u003C= 50% by transthoracic echocardiogram (TTE) at screening\n* Participants with any active, uncontrolled viral, bacterial, or fungal infection, including active HIV-1, Hepatitis B (HBV) and\u002For Hepatitis C (HCV) infection (positive HBV or HCV viral load in the setting of positive HBV core antibody or surface antibody or HCV antibody); history of HIV, HBV, or HCV is allowed if there is no uncontrolled viral infection\n* Pregnancy\n* Presence of another known cause of cytopenia or dysplastic marrow that is untreated and may limit interpretation of results\n* Uncontrolled intercurrent illness or any significant disease, evaluated by history, physical exam and chemistries or social situations that may limit interpretation of results, limit compliance with study requirements, or that could increase risk to the participant","ALL","12 Years","120 Years",{"count":21,"type":22},160,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","Background:\n\nMyelodysplastic syndrome (MDS) and myelodysplastic\u002Fmyeloproliferative neoplasm (MDS\u002FMPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS\u002FMPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed.\n\nObjective:\n\nTo test a study drug (pacritinib) in adults and children with MDS or MDS\u002FMPN.\n\nEligibility:\n\nChildren (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS\u002FMPN.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone.\n\nPacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants.\n\nParticipants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years.\n\nBone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.",[29],"Myelodysplastic Syndromes",[31,32,33,34,35,36,37,38,39],"Myelodysplastic Syndrome","bone marrow disorder","Myelodysplasia","myeloproliferative disorders","pediatric bone marrow failure","5q deletion","multi-lineage dysplasia","Clonal Hematopoiesis","Aplastic Anemia","RECRUITING","2026-07-10",{"date":43,"type":44},"2026-07-13","ACTUAL",{"date":46,"type":22},"2026-07-16",{"date":48,"type":22},"2035-01-01",{"name":50,"class":51},"National Cancer Institute (NCI)","NIH",1,{"id":54,"slug":55,"hasResults":12,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":4,"eligibilityCriteria":59,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":61,"targetDuration":4,"studyType":23,"phases":63,"briefSummary":64,"conditions":65,"keywords":68,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":41,"lastUpdatePostDateStruct":75,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":83},"100054019","phase-1-a-study-of-bgb-11417-in-participants-with-myeloid-malignancies-100054019","NCT04771130","A Study of BGB-11417 in Participants With Myeloid Malignancies","A Phase 1b\u002F2, Open-Label, Dose Finding, and Expansion Study of the Bcl-2 Inhibitor BGB-11417 in Patients With Myeloid Malignancies","Key Inclusion Criteria:\n\n1. Confirmed diagnosis of one of the following by 2016 World Health Organization criteria:\n\n   * AML, nonacute promyelocytic leukemia\n   * MDS\n   * MDS\u002FMPN\n2. Eastern Cooperative Oncology Group performance status of 0 to 2.\n3. Adequate organ function defined as:\n\n   * Creatinine clearance ≥ 50 milliliters\u002Fminute (mL\u002Fmin) (or between 30 and 49 mL\u002Fmin in unfit AML cohort)\n   * Adequate liver function\n4. Life expectancy of \\> 12 weeks.\n5. Ability to comply with the requirements of the study.\n\nKey Exclusion Criteria:\n\n1. A diagnosis of acute promyelocytic leukemia.\n2. History of prior malignancy, with the exception of either a history of MDS or MDS\u002FMPN that has transformed to AML, or other prior malignancy that was treated with a full curative intent and no evidence of recurrence within the past 2 years (eg, localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast, or localized Gleason score ≤ 6 prostate cancer)\n3. Antecedent MPN including myelofibrosis, essential thrombocytosis, polycythemia vera, or chronic myelogenous leukemia with or without BCR-ABL1 translocation and AML with BCR-ABL1 translocation.\n4. Prior therapy with a B-cell lymphoma-2 inhibitor\n5. Known central nervous system involvement by leukemia.\n\nNote: Other protocol defined Inclusion\u002FExclusion criteria may apply.","18 Years",{"count":62,"type":22},260,[25,26],"The study will determine the safety, tolerability, recommended Phase 2 dose (RP2D) and preliminary efficacy of BGB-11417 as monotherapy and in combination with azacitidine in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)or MDS\u002Fmyeloproliferative neoplasm (MPN) .",[66,29,67],"Acute Myeloid Leukemia","Myelodysplastic\u002FMyeloproliferative Neoplasm",[69,70,71,72,73,74],"BGB-11417","Azacitidine","Posaconazole","AML","MDS","MDS\u002FMPN",{"date":43,"type":44},{"date":77,"type":44},"2021-05-24",{"date":79,"type":22},"2028-02-08",{"name":81,"class":82},"BeOne Medicines","INDUSTRY",46,{"id":85,"slug":86,"hasResults":12,"nctId":87,"briefTitle":88,"officialTitle":88,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":90,"enrollmentInfo":91,"targetDuration":4,"studyType":23,"phases":93,"briefSummary":94,"conditions":95,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":4},"100054233","phase-2-dexamethasone-intravenous-injection-of-human-immunoglobulin-and-increased-infusion-of-mononuclear-cells-to-reduce-donor-specific-antibodies-in-haploid-hematopoietic-stem-cell-transplantation-a-prospective-multicenter-study-100054233","NCT07698080","Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploid Hematopoietic Stem Cell Transplantation: a Prospective, Multicenter Study","Inclusion Criteria:\n\n1. Diagnosis of benign or malignant hematological diseases (including leukemia, lymphoma, thalassemia, aplastic anemia, myelodysplastic syndromes, etc.) confirmed by NCCN guidelines, and determined by the investigator to require allogeneic hematopoietic stem cell transplantation.\n2. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 3.\n3. Donor-specific antibody (DSA) mean fluorescence intensity (MFI) \\> 500.\n4. Age between 18 and 65 years (age limits are also captured separately in the eligibility module).\n5. Body weight between 40 kg and 100 kg.\n6. No other suitable donor available (i.e., no DSA-negative related donor or unrelated donor).\n\nExclusion Criteria:\n\n1. Patients unsuitable for transplantation or without willingness to undergo transplantation, or diagnosed with non-hematological diseases.\n2. Estimated life expectancy \\\u003C 1 month.\n3. Known allergy to any drug or intervention used in the study regimen.\n4. Pregnancy, lactation, active severe infection, or severe major organ dysfunction.\n5. Severe psychiatric or neurological disorders that may affect the ability to provide informed consent and\u002For to report adverse events or comply with observation.\n6. Refusal or inability to sign the informed consent form.","65 Years",{"count":92,"type":22},60,[26],"This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower harmful antibodies called donor-specific antibodies (DSA) in patients who need a stem cell transplant from a half-matched (haploidentical) family donor.\n\nIn these transplants, DSA are antibodies made by the patient's own body that attack the donor's stem cells. If DSA levels are high, the transplant is more likely to fail - the donor cells may not \"take\" (engraft). Currently, there is no single, simple, and reliable way to reduce DSA, and many existing methods have drawbacks.\n\nBased on our earlier experience in 11 patients, this three-part approach seemed to work well. All patients successfully engrafted, and DSA levels dropped quickly. Now we want to confirm these results in a larger, prospective, multicenter study.\n\nWe plan to enroll 60 patients aged 18-65 with blood cancers or other blood disorders who need a haploidentical transplant, have DSA levels above 500 MFI (a measure of antibody strength), and have no other suitable donor available. Participants will receive:\n\n* Dexamethasone (25 mg\u002Fm²) for 4 days before transplant,\n* IVIG (1 g\u002Fkg) one day before transplant,\n* Extra mononuclear cells on transplant day - the extra amount depends on how high their DSA level is (low, medium, or high).\n\nThe main goal is to see how many patients have primary graft failure (when the donor cells never engraft). We will also measure how long it takes for blood counts to recover, rates of graft-versus-host disease, survival, and side effects. All participants will be followed for 1 year.\n\nThis study will help us find out whether this combination is a safe, simple, and effective way to improve transplant success for patients with DSA who have no other donor options.",[96,97,98,39,29],"Leukemia","Lymphoma","Thalassemia","NOT_YET_RECRUITING","2026-07-07",{"date":43,"type":44},{"date":103,"type":22},"2026-07-03",{"date":105,"type":22},"2028-07-03",{"name":107,"class":108},"Hematology department of the 920th hospital","OTHER",{"id":110,"slug":4,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":111,"targetDuration":4,"studyType":23,"phases":112,"briefSummary":27,"conditions":113,"keywords":114,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":118,"completionDateStruct":119,"leadSponsor":120,"locationsCount":52},"100539388",{"count":21,"type":22},[25,26],[29],[31,32,33,34,35,36,37,38,39],"2026-07-01",{"date":117,"type":44},"2026-07-02",{"date":100,"type":22},{"date":48,"type":22},{"name":50,"class":51},{"id":122,"slug":123,"hasResults":12,"nctId":124,"briefTitle":125,"officialTitle":125,"acronym":4,"eligibilityCriteria":126,"healthyVolunteers":127,"sex":17,"minAge":60,"maxAge":19,"enrollmentInfo":128,"targetDuration":4,"studyType":130,"phases":4,"briefSummary":131,"conditions":132,"keywords":133,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":139,"startDateStruct":140,"completionDateStruct":142,"leadSponsor":144,"locationsCount":52},"100484446","collection-of-blood-bone-marrow-skin-saliva-and-stool-samples-from-healthy-volunteers-used-for-comparative-analysis-of-myeloid-malignancies-100484446","NCT05588154","Collection of Blood, Bone Marrow, Skin, Saliva, and Stool Samples From Healthy Volunteers Used for Comparative Analysis of Myeloid Malignancies","* INCLUSION CRITERIA:\n* Age \\>= 18 years old\n* Healthy volunteers; the following confirmed by the Principal Investigator or designees based on recent (within 3 months before study intervention(s))\n* medical history\n* physical exam\n* complete blood count (CBC) within the normal reference range per the reporting clinical laboratory, established published literature and reports, or as deemed acceptable by the medical team based on the age and condition of the volunteer consistent with established clinical standards.\n* The ability of the participant to understand and the willingness to sign a written consent document.\n\nEXCLUSION CRITERIA:\n\n\\- Active illnesses, immunodeficiency, history of opportunistic infection, autoimmune disease, history of or active malignancy, prior organ, bone marrow, or peripheral blood stem cell transplant or antibiotic treatment within 3 months before study intervention(s).\n\nNote: participants with non-melanoma skin cancer or carcinoma in situ of the cervix or breast are eligible.\n\n* Current immunosuppressive medication.\n* Any one of the following symptoms as declared by the participant at least one day per week within 3 months before study intervention(s) (Rome IV criteria \\[36\\])\n* Diarrhea characterized as frequent (\\>2) loose stools\n* Constipation defined as \\\u003C 3 spontaneous bowel movements per week\n* Bloating and\u002For distention\n* Abdominal pain.\n* Participants with a history of the human immunodeficiency virus (HIV), hepatitis C (HCV), or hepatitis B (HBV) as confirmed by a seropositive blood test.\n* Pregnancy confirmed with beta-Human Chorionic Gonadotropin (Beta-HCG) serum or urine pregnancy test performed in women of childbearing potential at screening.\n* Breastfeeding participants.",true,{"count":129,"type":22},1000,"OBSERVATIONAL","Background:\n\nMyelodysplastic syndromes (MDS) are disorders of blood stem cells that can develop into blood cancers. Treatment options are limited. To find better treatments, researchers need to better understand how MDS develops. To do that, they must be able to compare biospecimens from people with the disease to those of healthy people.\n\nObjective:\n\nThis study will create a database of biospecimens collected from healthy volunteers.\n\nEligibility:\n\nHealthy people aged 18 and older.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood and urine tests.\n\nUp to 5 types of samples will be collected on 1 or more days within 1 month of screening:\n\nBlood: Blood will be drawn by inserting a needle into a vein.\n\nSaliva: Participants will scrape the insides of their cheeks with a brush.\n\nStool: Participants will be given a container to collect stool at home. They will use a prepaid envelope to mail in the sample.\n\nBone marrow: A sample of the soft tissue inside the bones will be drawn out. The area to be biopsied, usually the lower back, will be numbed. A needle will be inserted through a small cut to remove the sample. Participants' pain will be monitored; additional numbing medicine may be used.\n\nSkin: A piece of skin about 1\u002F6 of an inch across will be cut away. Stitches may be used to close the wound. Participants will return to the clinic to have the stitches removed.\n\nParticipants do not have to provide all of the samples listed. They will give each sample only once.",[29],[134,66,135,136,137,138],"Hematopoietic Stem Cell Malignancies","DNA methyltransferase inhibitors","Treatment-Related Complications","Cytotoxic Therapy","Natural History",{"date":117,"type":44},{"date":141,"type":44},"2023-01-11",{"date":143,"type":22},"2052-12-01",{"name":50,"class":51},{"id":146,"slug":147,"hasResults":12,"nctId":148,"briefTitle":149,"officialTitle":150,"acronym":4,"eligibilityCriteria":151,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":152,"enrollmentInfo":153,"targetDuration":4,"studyType":23,"phases":155,"briefSummary":156,"conditions":157,"keywords":4,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":160,"lastUpdatePostDateStruct":161,"startDateStruct":162,"completionDateStruct":164,"leadSponsor":166,"locationsCount":168},"100347357","phase-1-a-phase-1-study-of-orca-q-in-recipients-undergoing-allogeneic-transplantation-for-hematologic-malignancies-100347357","NCT03802695","A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies","A Phase 1 Dose Escalation and Expansion Study of Orca-Q, an Engineered Donor Graft Derived From Mobilized Peripheral Blood, in Recipients Undergoing Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies","Key Inclusion Criteria:\n\n1. Age at the time of enrollment:\n\n   1. For MAC with fully matched donor (Arm A with 8\u002F8 donor and Arm C) and NMA\u002FRIC: Age ≥ 12 and ≤ 78 years\n   2. For MAC with mismatched donors (Arm A with 7\u002F8 donor and Arm B): Age ≥ 12 and ≤ 65 years\n2. Diagnosed acute myeloid, lymphoblastic or mixed phenotype leukemia, or high or very high risk myelodysplastic syndrome (MDS) either in complete remission (CR) or with ≤ 10 percent of blast cells in bone marrow (BM)\n3. Indicated for allogeneic hematopoietic stem cell transplant (alloHCT)\n4. Matched to a 8\u002F8 or 7\u002F8 related or unrelated donor, or to a related haploidentical donor\n5. Estimated glomerular filtration rate (eGFR) \\> 50 mL\u002Fminute (MAC with tacrolimus) or \\> 30 mL\u002Fminute (NMA\u002FRIC or MAC without tacrolimus)\n6. Cardiac parameters: Cardiac ejection fraction ≥ 45 percent (MAC) or ≥ 40 percent (NMA\u002FRIC)\n7. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50 percent for MAC or ≥ 40 percent for NMA\u002FRIC\n8. Liver function: Total bilirubin \\\u003C 1.5 times upper limit of normal (ULN) (MAC) or \\\u003C 3 times ULN (NMA\u002FRIC); alanine transaminase (ALT)\u002Faspartate transaminase (AST) \\\u003C 3 times ULN (MAC) or \\\u003C 5 times ULN (NMA\u002FRIC)\n9. Participants enrolling on NMA\u002FRIC-alloHCT arms must be deemed unfit for a myeloablative alloHCT per assessment of the principal investigator (PI)\n\nKey Exclusion Criteria:\n\n1. Prior alloHCT\n2. Currently receiving corticosteroids or other immunosuppressive therapy except for approved disease-specific therapy for the patient's underlying hematologic malignancy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg\u002Fday are allowed\n3. Planned donor lymphocyte infusion (DLI)\n4. Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy) or alemtuzumab\n5. Positive anti-donor HLA antibodies against a mismatched allele in the selected donor\n6. Low performance score: For MAC: Karnofsky Performance Score (KPS) \\\u003C 70 percent, For NMA\u002FRIC: \\\u003C60 percent\n7. High HCT-specific Comorbidity Index (HCT-CI): For MAC \\> 4, For NMA\u002FRIC \\>6\n8. Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment\n9. Seropositive for human immunodeficiency virus (HIV)-1 or -2, human T-lymphotropic virus (HTLV)-1 or -2 or Hepatitis B surface antigen (HbsAg) or anti-Hepatitis C virus (HCV) antibody (Ab)\n10. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment\n11. Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected. Patients with concurrent indolent hematologic malignancies that do not require active treatment and are under active surveillance only (such as CLL, low-grade lymphomas, smoldering MM, MZL) may be included with the approval of Medical Monitor\n12. History of idiopathic or secondary myelofibrosis\n13. Women who are pregnant or breastfeeding","78 Years",{"count":154,"type":22},300,[25],"This study will evaluate the safety, tolerability, and efficacy of engineered donor grafts (\"OrcaGraft\"\u002F\"Orca-Q\") in participants undergoing allogeneic hematopoietic cell transplant (alloHCT) transplantation for hematologic malignancies.",[66,29,158,159],"Mixed Phenotype Acute Leukemia","Acute Lymphoblastic Leukemia","2026-06-29",{"date":115,"type":44},{"date":163,"type":44},"2019-04-08",{"date":165,"type":22},"2027-12",{"name":167,"class":82},"Orca Biosystems, Inc.",12,{"id":170,"slug":171,"hasResults":12,"nctId":172,"briefTitle":173,"officialTitle":174,"acronym":4,"eligibilityCriteria":175,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":90,"enrollmentInfo":176,"targetDuration":4,"studyType":23,"phases":178,"briefSummary":179,"conditions":180,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":181,"lastUpdatePostDateStruct":182,"startDateStruct":184,"completionDateStruct":186,"leadSponsor":188,"locationsCount":52},"100644986","phase-2-age-stratified-conditioning-regimen-efficacy-for-mds-haplo-hsct-100644986","NCT07677306","Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT","Prospective Cohort Study of Age-Stratified Busulfan-Based Conditioning Regimens in Adult Patients With Myelodysplastic Syndrome Receiving Haploidentical Hematopoietic Stem Cell Transplantation","Inclusion Criteria:\n\n* Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation\n\nExclusion Criteria:\n\n* Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure",{"count":177,"type":22},120,[26],"This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu\u002FFlu\u002FCy\u002FATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu\u002FCy+ATG conditioning regimen (for patients aged \\\u003C55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical transplantation.",[29],"2026-06-24",{"date":183,"type":44},"2026-06-30",{"date":185,"type":22},"2026-07-15",{"date":187,"type":22},"2028-06-30",{"name":189,"class":108},"Peking University People's Hospital",{"id":191,"slug":192,"hasResults":12,"nctId":193,"briefTitle":194,"officialTitle":195,"acronym":196,"eligibilityCriteria":197,"healthyVolunteers":127,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":198,"targetDuration":4,"studyType":23,"phases":199,"briefSummary":201,"conditions":202,"keywords":208,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":181,"lastUpdatePostDateStruct":210,"startDateStruct":212,"completionDateStruct":214,"leadSponsor":216,"locationsCount":52},"100429254","biocollection-in-myelodysplastic-syndrome-p-mds-100429254","NCT04869683","Biocollection in MyeloDysplastic Syndrome (P-MDS)","Biocollection in Patients With Myelodysplastic Syndrome (P-MDS)","P-MDS","Inclusion Criteria:\n\n* Major\n* patient with or suspected of myelodysplastic syndrome (WHO definition) at diagnosis and\u002For during follow-up, which is managed at the level of the Cancer-Hematology Institute of the Brest CHRU\n* Presence of biological material collected within the CRB\n* Patient's consent obtained\n\nExclusion Criteria :\n\n* Minor and pregnant woman\n* Lack of biological material collected within the CRB\n* Refusal to participate: lack of consent - Unable to consent\n* Patient under judicial protection: guardianship, curatorship ...",{"count":5,"type":22},[200],"NA","Myelodysplastic syndromes (MDS) are chronic myeloid hemopathies characterized by ineffective hematopoiesis (with peripheral cytopenias) and which contrast with a marrow of normal richness. MDS is considered one of the four most common blood diseases. The incidence is estimated at 4,059 cases \u002F year in 2012 with an average age of 78 years in men and 81 years in women (INCA report, Cancers in France in 2015). The incidence increases with lengthening of the lifespan. The main risk of MDS is transformation to acute leukemia in 30 to 40% of cases. Treatment options depend on clinical, hematologic and chromosomal abnormalities. The prognosis is considered to be at low or high risk of developing acute leukemia. This distinction will therefore have an impact on the therapeutic solution (s). MDS exhibit clinical, morphological and genetic heterogeneity. It is therefore necessary to form subgroups of patients to better understand the physiopathogenesis of this pathology. The constitution of a biocollection will make it possible to search for clinical and biological prognostic markers in order to identify patients progressing to acute myeloid leukemia.",[29,203,204,205,206,207],"Myelodysplastic Anemia","Myelodysplastic Syndrome With Isolated Del(5Q)","Myelodysplastic Syndrome With Ring Sideroblasts","Acute Myeloid Leukemia With Multilineage Dysplasia","Chromosome Abnormality",[73,36,209,72],"ring sideroblasts",{"date":211,"type":44},"2026-06-25",{"date":213,"type":44},"2022-10-19",{"date":215,"type":22},"2032-10-19",{"name":217,"class":108},"University Hospital, Brest",{"id":219,"slug":220,"hasResults":12,"nctId":221,"briefTitle":222,"officialTitle":223,"acronym":4,"eligibilityCriteria":224,"healthyVolunteers":12,"sex":17,"minAge":90,"maxAge":4,"enrollmentInfo":225,"targetDuration":4,"studyType":130,"phases":4,"briefSummary":227,"conditions":228,"keywords":235,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":181,"lastUpdatePostDateStruct":390,"startDateStruct":391,"completionDateStruct":393,"leadSponsor":395,"locationsCount":397},"100143410","registry-of-older-patients-with-cancer-100143410","NCT01137825","Registry of Older Patients With Cancer","Carolina Senior: UNC Registry for Older Cancer Patients","DISEASE CHARACTERISTICS:\n\n* Patients must have an appointment at the North Carolina Cancer Hospital and agree to complete the Geriatric Assessment NOTE: Not all patients included in the registry will ultimately be diagnosed with cancer. Patients who complete a GA and are not diagnosed with cancer will remain in the database, but will be categorized into a separate group and will not have their medical records accessed.\n\nPATIENT CHARACTERISTICS:\n\n* Able to read and speak English\n\nPRIOR CONCURRENT THERAPY:\n\n* Not specified",{"count":226,"type":22},3000,"RATIONALE: Gathering information about older patients with cancer may help the study of cancer in the future.\n\nPURPOSE: This research study is gathering information from older patients with cancer into a registry.",[229,230,96,97,231,232,29,233,234],"Chronic Myeloproliferative Disorders","Cognitive\u002FFunctional Effects","Lymphoproliferative Disorder","Multiple Myeloma and Plasma Cell Neoplasm","Myelodysplastic\u002FMyeloproliferative Neoplasms","Unspecified Adult Solid Tumor, Protocol Specific",[236,237,238,239,240,241,242,243,244,245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,276,277,278,279,280,281,282,283,284,285,286,287,288,289,290,291,292,293,294,295,296,297,298,299,300,301,302,303,304,305,306,307,308,309,310,311,312,313,314,315,316,317,318,319,320,321,322,323,324,325,326,327,328,329,330,331,332,333,334,335,336,337,338,339,340,341,342,343,344,345,346,347,348,349,350,351,352,353,354,355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389],"cognitive\u002Ffunctional effects","unspecified adult solid tumor, protocol specific","accelerated phase chronic myelogenous leukemia","acute undifferentiated leukemia","adult acute lymphoblastic leukemia in remission","adult acute myeloid leukemia in remission","adult acute myeloid leukemia with 11q23 (MLL) abnormalities","adult acute myeloid leukemia with inv(16)(p13;q22)","adult acute myeloid leukemia with t(15;17)(q22;q12)","adult acute myeloid leukemia with t(16;16)(p13;q22)","adult acute myeloid leukemia with t(8;21)(q22;q22)","atypical chronic myeloid leukemia, BCR-ABL negative","blastic phase chronic myelogenous leukemia","chronic myelomonocytic leukemia","chronic phase chronic myelogenous leukemia","mast cell leukemia","meningeal chronic myelogenous leukemia","progressive hairy cell leukemia, initial treatment","prolymphocytic leukemia","recurrent adult acute lymphoblastic leukemia","recurrent adult acute myeloid leukemia","recurrent adult T-cell leukemia\u002Flymphoma","refractory chronic lymphocytic leukemia","refractory hairy cell leukemia","relapsing chronic myelogenous leukemia","secondary acute myeloid leukemia","stage 0 chronic lymphocytic leukemia","stage I adult T-cell leukemia\u002Flymphoma","stage I chronic lymphocytic leukemia","stage II adult T-cell leukemia\u002Flymphoma","stage II chronic lymphocytic leukemia","stage III adult T-cell leukemia\u002Flymphoma","stage III chronic lymphocytic leukemia","stage IV adult T-cell leukemia\u002Flymphoma","stage IV chronic lymphocytic leukemia","T-cell large granular lymphocyte leukemia","untreated adult acute lymphoblastic leukemia","untreated adult acute myeloid leukemia","untreated hairy cell leukemia","recurrent adult Hodgkin lymphoma","stage I adult Hodgkin lymphoma","stage II adult Hodgkin lymphoma","stage III adult Hodgkin lymphoma","stage IV adult Hodgkin lymphoma","anaplastic large cell lymphoma","angioimmunoblastic T-cell lymphoma","cutaneous B-cell non-Hodgkin lymphoma","recurrent cutaneous T-cell non-Hodgkin lymphoma","stage I cutaneous T-cell non-Hodgkin lymphoma","stage II cutaneous T-cell non-Hodgkin lymphoma","stage III cutaneous T-cell non-Hodgkin lymphoma","stage IV cutaneous T-cell non-Hodgkin lymphoma","recurrent mycosis fungoides\u002FSezary syndrome","stage I mycosis fungoides\u002FSezary syndrome","stage II mycosis fungoides\u002FSezary syndrome","stage III mycosis fungoides\u002FSezary syndrome","stage IV mycosis fungoides\u002FSezary syndrome","adult grade III lymphomatoid granulomatosis","adult nasal type extranodal NK\u002FT-cell lymphoma","Waldenstrom macroglobulinemia","extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue","nodal marginal zone B-cell lymphoma","splenic marginal zone lymphoma","contiguous stage II adult Burkitt lymphoma","contiguous stage II adult diffuse large cell lymphoma","contiguous stage II adult diffuse mixed cell lymphoma","contiguous stage II adult diffuse small cleaved cell lymphoma","contiguous stage II adult immunoblastic large cell lymphoma","contiguous stage II adult lymphoblastic lymphoma","contiguous stage II grade 1 follicular lymphoma","contiguous stage II grade 2 follicular lymphoma","contiguous stage II grade 3 follicular lymphoma","contiguous stage II mantle cell lymphoma","contiguous stage II marginal zone lymphoma","contiguous stage II small lymphocytic lymphoma","stage I adult Burkitt lymphoma","stage I adult diffuse large cell lymphoma","stage I adult diffuse mixed cell lymphoma","stage I adult diffuse small cleaved cell lymphoma","stage I adult immunoblastic large cell lymphoma","stage I adult lymphoblastic lymphoma","stage I grade 1 follicular lymphoma","stage I grade 2 follicular lymphoma","stage I grade 3 follicular lymphoma","stage I mantle cell lymphoma","stage I marginal zone lymphoma","stage I small lymphocytic lymphoma","noncontiguous stage II adult Burkitt lymphoma","noncontiguous stage II adult diffuse large cell lymphoma","noncontiguous stage II adult diffuse mixed cell lymphoma","noncontiguous stage II adult diffuse small cleaved cell lymphoma","noncontiguous stage II adult immunoblastic large cell lymphoma","noncontiguous stage II adult lymphoblastic lymphoma","noncontiguous stage II grade 1 follicular lymphoma","noncontiguous stage II grade 2 follicular lymphoma","noncontiguous stage II grade 3 follicular lymphoma","noncontiguous stage II mantle cell lymphoma","noncontiguous stage II marginal zone lymphoma","noncontiguous stage II small lymphocytic lymphoma","stage III adult Burkitt lymphoma","stage III adult diffuse large cell lymphoma","stage III adult diffuse mixed cell lymphoma","stage III adult diffuse small cleaved cell lymphoma","stage III adult immunoblastic large cell lymphoma","stage III adult lymphoblastic lymphoma","stage III grade 1 follicular lymphoma","stage III grade 2 follicular lymphoma","stage III grade 3 follicular lymphoma","stage III mantle cell lymphoma","stage III marginal zone lymphoma","stage III small lymphocytic lymphoma","stage IV adult Burkitt lymphoma","stage IV adult diffuse large cell lymphoma","stage IV adult diffuse mixed cell lymphoma","stage IV adult diffuse small cleaved cell lymphoma","stage IV adult immunoblastic large cell lymphoma","stage IV adult lymphoblastic lymphoma","stage IV grade 1 follicular lymphoma","stage IV grade 2 follicular lymphoma","stage IV grade 3 follicular lymphoma","stage IV mantle cell lymphoma","stage IV marginal zone lymphoma","stage IV small lymphocytic lymphoma","recurrent adult Burkitt lymphoma","recurrent adult diffuse large cell lymphoma","recurrent adult diffuse mixed cell lymphoma","recurrent adult diffuse small cleaved cell lymphoma","recurrent adult grade III lymphomatoid granulomatosis","recurrent adult immunoblastic large cell lymphoma","recurrent adult lymphoblastic lymphoma","recurrent grade 1 follicular lymphoma","recurrent grade 2 follicular lymphoma","recurrent grade 3 follicular lymphoma","recurrent mantle cell lymphoma","recurrent marginal zone lymphoma","recurrent small lymphocytic lymphoma","intraocular lymphoma","post-transplant lymphoproliferative disorder","chronic eosinophilic leukemia","chronic neutrophilic leukemia","primary myelofibrosis","essential thrombocythemia","polycythemia vera","extramedullary plasmacytoma","isolated plasmacytoma of bone","stage I multiple myeloma","stage II multiple myeloma","stage III multiple myeloma","primary systemic amyloidosis","refractory multiple myeloma","de novo myelodysplastic syndromes","previously treated myelodysplastic syndromes","secondary myelodysplastic syndromes","myelodysplastic\u002Fmyeloproliferative neoplasm, unclassifiable",{"date":160,"type":44},{"date":392,"type":4},"2009-09",{"date":394,"type":22},"2030-12",{"name":396,"class":108},"UNC Lineberger Comprehensive Cancer Center",7,{"id":399,"slug":400,"hasResults":12,"nctId":401,"briefTitle":402,"officialTitle":403,"acronym":404,"eligibilityCriteria":405,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":90,"enrollmentInfo":406,"targetDuration":4,"studyType":23,"phases":408,"briefSummary":409,"conditions":410,"keywords":414,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":418,"lastUpdatePostDateStruct":419,"startDateStruct":421,"completionDateStruct":423,"leadSponsor":425,"locationsCount":52},"100577747","phase-2-intensity-modulated-total-marrow-irradiation-in-fully-human-leukocyte-antigen-hla-matched-and-partially-hla-mismatched-allogeneic-transplantation-patients-with-high-risk-acute-myeloid-leukemia-aml-chronic-myeloid-leukemia-cml-and-myelodysplastic-syndrome-mds-100577747","NCT06802315","Intensity Modulated Total Marrow Irradiation in Fully Human Leukocyte Antigen (HLA)-Matched and Partially-HLA Mismatched Allogeneic Transplantation Patients With High-Risk Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), and Myelodysplastic Syndrome (MDS)","A Phase II Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Myeloablative Fludarabine\u002FBusulfan and Post-Transplant Cyclophosphamide (PTCY) for Fully Human Leukocyte Antigen (HLA)-Matched and Partially-HLA Mismatched Allogeneic Transplantation Patients With High-Risk AML, CML, and MDS","BMT-13","Inclusion Criteria:\n\n* 1\\. Age 18-65 years.\n* 2\\. Patients with CML, AML, or MDS who meet one of the following criteria: 2a. Relapsed or refractory AML (including AML in CR2) 2b. Poor-risk AML in first remission, with remission defined as \\\u003C5% bone marrow blasts morphologically:\n* AML arising from MDS, a myeloproliferative disorder, or secondary AML\n* Poor risk molecular features according to Leukemia Net including ASXL1, BCOR, EZH2, RUNX1, SF3B1, SRSF2, STAG2, U2AF1, and\u002For ZRSR2\n* Poor-risk cytogenetics: Monosomal karyotype, complex karyotype (\\> 3 abnormalities), inv (3), t(3;3), t(6;9), MLL rearrangement with the exception of t(9;11), or abnormalities of chromosome 5 or 7. 2c. Primary refractory disease 2d. MDS with at least one of the following poor-risk features:\n* Poor-risk cytogenetics including 3q abnormalities, 7\u002F7q minus or complex cytogenetics (\\>3 abnormalities).\n* Current or previous INT-2 or high IPSS score.\n* Treatment-related MDS.\n* MDS diagnosed before the age of 21 years.\n* Progression on or lack of response to standard DNA-methyltransferase inhibitor therapy.\n* Life-threatening cytopenias, including those requiring regular PRBC or platelet transfusions. 2e. CML with a history of accelerated or blast phase.\n\nExclusion Criteria:\n\n* 1\\. Presence of significant co-morbidity as shown by:\n* 1a. Left ventricular ejection fraction \\\u003C 50%\n* 2b. Creatinine clearance \\\u003C30ml\u002Fmin.\n* 3c. Bilirubin \\> 2.0 mg\u002FdL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST \\> 5 x ULN.\n* 4d. FEV1 and FVC \\\u003C 50% of predicted or DLCO \\\u003C50% of predicted once corrected for anemia.\n* 5e. Karnofsky score \\\u003C70\n* 6f. Active viral hepatitis or HIV infection.\n* 7g. Cirrhosis.\n* 2\\. Pregnancy or breast feeding\n* 3\\. Patients unable to sign informed consent.\n* 4\\. Patients previously received radiation to \\>20% of bone marrow-containing areas.",{"count":407,"type":22},38,[26],"The study is a Phase II clinical trial. Patients will receive intensity-modulated total marrow irradiation (TMI) at a dose of 9 Gray (Gy) with standard myeloablative fludarabine intravenous (IV) and targeted busulfan (FluBu4) conditioning prior to allogeneic hematopoietic stem cell transplant (HSCT). Graft-versus-host disease (GVHD) prophylaxis will include Cyclophosphamide on Day +3 and +4, tacrolimus, and mycophenolate mofetil.",[411,412,413,29],"Acute Myeloid Leukemia, Relapsed, Adult","Acute Myeloid Leukemia Refractory","Chronic Myeloid Leukemia - Accelerated Phase",[415,416,417],"Total Marrow Irradiation","Stem Cell Transplant","Allogenic Transplant","2026-06-23",{"date":420,"type":44},"2026-06-26",{"date":422,"type":44},"2025-02-04",{"date":424,"type":22},"2032-03",{"name":426,"class":108},"University of Illinois at Chicago",{"id":428,"slug":429,"hasResults":12,"nctId":430,"briefTitle":431,"officialTitle":431,"acronym":432,"eligibilityCriteria":433,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":434,"targetDuration":4,"studyType":23,"phases":436,"briefSummary":437,"conditions":438,"keywords":441,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":444,"lastUpdatePostDateStruct":445,"startDateStruct":446,"completionDateStruct":448,"leadSponsor":450,"locationsCount":52},"100636995","phase-2-a-pragmatic-clinical-trial-to-prevent-relapse-for-myeloid-malignancies-with-measurable-disease-prior-to-allogeneic-transplant-100636995","NCT07572929","A Pragmatic Clinical Trial to Prevent Relapse for Myeloid Malignancies With Measurable Disease Prior to Allogeneic Transplant","PREeMPT","Inclusion Criteria:\n\n* Age ≥18 years at time of transplant.\n* Must have a diagnosis of a myeloid malignancy, myelodysplastic syndrome, myeloproliferative neoplasm (including primary or secondary myelofibrosis), or acute myeloid leukemia or biphenotypic acute leukemia.\n* Pre-transplant disease assessment must include at least one:\n* Immunophenotypic assessment by flow for aberrant blasts\n* NGS to identify persistence of malignant clones using a standard clinically available assay\n* PCR based measurement of prior identified mutations (if applicable)\n* Ability to understand a written informed consent document, and the willingness to sign it.\n\nExclusion Criteria:\n\nNone",{"count":435,"type":22},450,[26],"This is a prospective, open-label investigation to determine whether measurable residual disease (MRD) guided assignment of maintenance therapy is effective in patients with myeloid malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). Pre-HCT, patients will undergo usual disease assessments which should include immunophenotypic and\u002For molecular testing. Based on the results of these tests, patients may or may not be recommended to receive maintenance therapy post-transplant, depending on the presence or absence of a residual malignant clone.",[439,29,440,66],"Myeloid Disorders","Myeloproliferative Neoplasm",[442,443],"Allogeneic hematopoietic cell transplantation","Measurable residual disease","2026-06-19",{"date":418,"type":44},{"date":447,"type":22},"2026-08-01",{"date":449,"type":22},"2034-02-01",{"name":451,"class":108},"Medical College of Wisconsin",{"id":453,"slug":454,"hasResults":12,"nctId":455,"briefTitle":456,"officialTitle":457,"acronym":4,"eligibilityCriteria":458,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":459,"targetDuration":4,"studyType":23,"phases":461,"briefSummary":462,"conditions":463,"keywords":465,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":487,"lastUpdatePostDateStruct":488,"startDateStruct":489,"completionDateStruct":491,"leadSponsor":493,"locationsCount":494},"100609581","phase-2-trial-of-orca-t-following-reduced-intensity-or-nonmyeloablative-conditioning-in-patients-with-acute-myeloid-leukemia-or-myelodysplastic-syndrome-100609581","NCT07216443","Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome","A Phase 2 Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome","Inclusion Criteria:\n\n1. Age ≥18 years at the time of enrollment\n2. Diagnosed with 1 of the following diseases:\n\n   1. Acute myeloid, or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi), with or without the presence of known minimal residual disease.\n   2. Myelodysplastic syndrome that is indicated for alloHCT per the 2017 International Expert Panel recommendations and\u002For therapy-related\u002Fsecondary MDS as defined by the World Health Organization (WHO) classification of myeloid malignancies, with ≤10% blast burden in the bone marrow.\n3. Planned to undergo 1 of the following preparative regimens as per Investigator discretion:\n\n   1. RIC cohort: Planned RIC-alloHCT including RIC regimen with TBI\u002Fthiotepa\u002Ffludarabine\n   2. NMA cohort: Planned NMA-alloHCT including NMA regimen with fludarabine\u002Fcyclophosphamide\u002FTBI\n4. Identified related or unrelated donor who is an 8\u002F8 match for HLA-A, -B, -C, and -DRB1\n5. Estimated glomerular filtration rate ≥30 mL\u002Fminute\n6. Cardiac ejection fraction at rest ≥40% or shortening fraction of ≥22% by echocardiogram or radionuclide scan (MUGA)\n7. Diffusing capacity of the lung for carbon monoxide (adjusted for hemoglobin) ≥40%\n8. Negative serum or urine β-HCG test in persons of childbearing potential\n9. Alanine transaminase (ALT)\u002Faspartate transaminase (AST) \\\u003C5 times the upper limit of normal (ULN)\n10. Total bilirubin \\\u003C3 × ULN\n11. Deemed ineligible for a fully myeloablative alloHCT per assessment of the principal investigator\n\nExclusion Criteria:\n\n1. Prior alloHCT\n2. Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg\u002Fday are allowed.\n3. Planned donor lymphocyte infusion (DLI)\n4. Planned pharmaceutical in vivo or ex vivo T-cell depletion\n5. Recipient-positive antidonor HLA antibodies against a mismatched allele in the selected donor\n6. Karnofsky performance score \\\u003C60%\n7. For RIC cohort only: HCT-Specific Comorbidity Index (HCT-CI) ≥6\n8. Uncontrolled bacterial, viral, or fungal infection (currently taking antimicrobial therapy and with progression or no clinical improvement) at the time of enrollment\n9. Seropositive for HIV-1 or -2, HTLV-1 or -2, hepatitis B surface antigen, or HCV antibody unless previously treated with curative therapy and are HCV NAT negative\n10. Known allergy or hypersensitivity to or intolerance of tacrolimus\n11. Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal, or Streptomyces avidinii proteins\n12. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment\n13. Concurrent malignancy within 1 year except nonmelanoma skin cancer that has been curatively resected\n14. Psychosocial circumstances that preclude the participant being able to go through transplantation or participate responsibly in follow-up care\n15. Persons who are pregnant or breastfeeding\n16. Person of childbearing potential (POCBP) or men who have sexual contact with POCBP who are unwilling to use effective forms of birth control or abstinence for 1 year after transplantation.\n17. Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's or medical monitor's judgment, precludes the recipient's safe participation in and completion of the trial or which could affect compliance with the protocol or interpretation of results",{"count":460,"type":22},80,[26],"This study will evaluate the safety, tolerability, and efficacy of Orca-T in participants undergoing reduced intensity or non-myeloablative allogeneic hematopoietic cell transplantation (alloHCT) for hematologic malignancies. Orca-T is an allogeneic stem cell and T-cell immunotherapy biologic manufactured for each patient (transplant recipient) from the mobilized peripheral blood of a specific, unique donor. It is composed of purified hematopoietic stem and progenitor cells (HSPCs), purified regulatory T cells (Tregs), and conventional T cells (Tcons).",[464,29,158],"Leukemia, Myeloid, Acute",[464,29,158,466,467,468,469,470,471,472,473,474,475,476,477,478,479,480,481,482,96,483,484,485,486],"Therapy-Related Myelodysplastic Syndrome","Hematopoietic Stem Cell Transplantation","Humans","Graft vs Host Disease","SERENE-T","ORCA-T","Disease","Pathologic Processes","Neoplasms by Histologic Type","Neoplasms","Hematologic Diseases","Bone Marrow Diseases","Precancerous Conditions","Neoplasms by Site","Disease Attributes","Immunoproliferative Disorders","Immune System Diseases","Preleukemia","Hematologic Neoplasms","Syndrome","Acute Disease","2026-06-18",{"date":418,"type":44},{"date":490,"type":44},"2025-12-09",{"date":492,"type":22},"2028-12",{"name":167,"class":82},5,{"id":496,"slug":497,"hasResults":12,"nctId":498,"briefTitle":499,"officialTitle":500,"acronym":4,"eligibilityCriteria":501,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":502,"enrollmentInfo":503,"targetDuration":4,"studyType":23,"phases":505,"briefSummary":506,"conditions":507,"keywords":509,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":487,"lastUpdatePostDateStruct":513,"startDateStruct":514,"completionDateStruct":516,"leadSponsor":518,"locationsCount":52},"100558474","phase-1-trial-for-patients-w-advanced-hematologic-malignancies-undergoing-allogeneic-hct-100558474","NCT06551584","Trial for Patients w\u002F Advanced Hematologic Malignancies Undergoing Allogeneic HCT","Trial for Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation From an HLA-Mismatched Donor (7\u002F8) With Orca-T","Inclusion Criteria:\n\n1. Eligible diseases:\n\n   * Acute myeloid, lymphoid or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi) as defined in Section 6.1.3; with or without the presence of known minimal residual disease, or\n   * Myelodysplasic syndrome (MDS) myelodysplastic syndromes eligible for alloHSCT and\u002For treatment-related MDS \\\u003C10% blasts\n2. Age ≥ 18 and ≤ 70 years at the time of enrollment.\n3. Eligible for myeloablative alloHCT including one of two the myeloablative conditioning regimens (fractionated total body irradiation plus cyclophosphamide or busulfan, fludarabine, and thiotepa)\n4. Has a related or unrelated donor available who is 7\u002F8 match (single allele mismatched) at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods.\n5. Estimated glomerular filtration rate (eGFR) ≥ 50 mL\u002Fminute or creatinine \\\u003C 2 mg\u002FdL.\n6. Cardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA).\n7. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50%.\n8. Total bilirubin \\\u003C 2 times upper limit of normal (ULN) (patients with Gilbert's syndrome may be included once hemolysis has been excluded).\n9. Ability to understand and the willingness to provide written informed consent.\n10. Negative serum or urine beta-HCG test in females of childbearing potential (FCBP) within 3 weeks of enrollment.\n\n    A female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).\n11. Able to give informed consent. Legal authorized representative (LAR) is permitted if subject is cognitively able to provide verbal assent.\n12. Karnofsky Performance Score ≥70%\n\nExclusion Criteria:\n\n1. Prior allogeneic HCT.\n2. Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg\u002Fday are allowed.\n3. Planned donor lymphocyte infusion (DLI).\n4. Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy), peri-transplant anti-thymocyte globulin (ATG), or alemtuzumab. For patients that have previously been exposed to a T cell-depleting agent, a 5 half-life washout of the agent must occur prior to planned Day 0 (day of infusion of Orca-T HSPC and Tregs ).\n5. Recipient positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:\n\n   1. Positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing), or\n   2. Presence of anti-donor HLA antibody to any of the following HLA loci: HLA-A, -B, -C, -DRB1, -DQB1, -DQA1, -DPB1, or -DPA1, with mean fluorescence intensity (MFI) \\>1000 by solid phase immunoassay.\n6. Uncontrolled bacterial, viral, or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment including known, active tuberculosis infection.\n7. Seropositive for HIV-1 or -2, HTLV-1 or -2, Hepatitis B sAg, and\u002For Hepatitis C antibody.\n\n   \\*History of hepatitis B or hepatitis C is permitted if viral load is undetectable per quantitative PCR and\u002For NAT. In this case, monitoring for hepatitis B or hepatitis C by PCR at 3, 6, and 12 months is recommended.\n8. Known allergy or hypersensitivity to, or intolerance of, any investigational agent or ingredient therein, or planned GVHD prophylactic medications.\n9. Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal or Streptomyces avidinii proteins.\n10. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment.\n11. Concurrent malignancy diagnosed within 12 months of enrollment, except non-melanoma skin cancers that have been curatively resected.\n12. Females of childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use effective forms of birth control or abstinence for one year after transplantation.\n\n    (FCBP definition: A female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).\n13. History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment. History of stroke or pulmonary embolism within 6 months of enrollment.\n14. Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the recipient's safe participation in and completion of the study, or which could affect compliance with the protocol or interpretation of results.","70 Years",{"count":504,"type":22},24,[25],"The study goal is to characterize the safety of the combination of Orca-T with dual agent GVHD prophylaxis.",[66,508,158,29],"Acute Lymphoid Leukemia",[510,511,512],"Orca-T","Hematopoietic Cell Transplantation","Advanced Hematologic Malignancies",{"date":418,"type":44},{"date":515,"type":44},"2025-12-01",{"date":517,"type":22},"2026-12",{"name":519,"class":108},"Stanford University",{"id":521,"slug":522,"hasResults":12,"nctId":523,"briefTitle":524,"officialTitle":524,"acronym":4,"eligibilityCriteria":525,"healthyVolunteers":127,"sex":17,"minAge":526,"maxAge":19,"enrollmentInfo":527,"targetDuration":4,"studyType":130,"phases":4,"briefSummary":529,"conditions":530,"keywords":531,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":487,"lastUpdatePostDateStruct":537,"startDateStruct":539,"completionDateStruct":541,"leadSponsor":543,"locationsCount":52},"100466209","comprehensive-molecular-and-clinical-evaluation-of-pediatric-and-adult-mds-100466209","NCT05350748","Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS","* INCLUSION CRITERIA - MDS Participants\n* Either sex, any age.\n* Histologically or cytologically suspected or confirmed myelodysplastic syndromes (MDS), myelodysplastic syndromes\u002Fmyeloproliferative neoplasms (MDS\u002FMPN), MDS\u002Fmyeloproliferative neoplasm with ringed sideroblasts and thrombocytosis (MDS\u002FMPN-RS-T), myelodysplastic syndromes\u002Fmyeloproliferative neoplasms unclassified (MDS\u002FMPN-U), chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), secondary acute myeloid leukemia (sAML) with antecedent MDS or MDS\u002FMPN, or participants who have precursor conditions that are associated with a risk of progression to MDS, including but not limited to clonal hematopoiesis of indeterminate potential (CHIP) and clonal cytopenia of unknown significance (CCUS).\n* Participants may have had any amount of prior therapy and may be receiving MDS-directed therapy at time of enrollment.\n* Participants must have an identified primary oncologist, hematologist or generalist outside of NIH who agrees to manage participant care and any diagnostic findings provided by this study.\n\nINCLUSION CRITERIA - Marrow Control Donor Participants\n\n* Either sex, and must be eligible for marrow donation per NIH Clinical Center requirements.\n* No history of hematological malignancies as listed as inclusion in 'Inclusion Criteria - MDS Participants' or current autoimmune disease.\n* Must be scheduled for bone marrow harvest for clinical application (e.g., marrow donation); or, if being evaluated for malignancy, have a clinical bone marrow aspirate scheduled (e.g., to rule out bone marrow involvement).\n\nINCLUSION CRITERIA - All Participants\n\n* Stated willingness to comply with all study procedures and availability for the duration of the study.\n* Ability of participant or parent\u002Fguardian to understand and the willingness to sign a written consent document.\n\nEXCLUSION CRITERIA - All Participants\n\n-Uncontrolled intercurrent illness, psychiatric illness, or other that would limit compliance with study requirements, or at the investigator s discretion.","1 Day",{"count":528,"type":22},1100,"Background:\n\nMyelodysplastic syndromes (MDS) occur when the cells that make blood cells are abnormal. There are limited treatment options for MDS. Researchers want to learn more through this natural history study so they can develop better treatments.\n\nObjective:\n\nTo study the natural course of MDS and MDS\u002Fmyeloproliferative neoplasms (MPN) and collect biological samples that can help researchers understand the disease.\n\nEligibility:\n\nPeople with suspected or confirmed MDS or MDS\u002FMPN. Healthy donors are also needed. They can be people who are scheduled to donate bone marrow at NIH for a relative, or they may be providing bone marrow in another study.\n\nDesign:\n\nParticipants will be screened with a medical history.\n\nParticipants will have a physical exam. They will give blood and urine samples. They will discuss their symptoms, medications, and ability to perform their normal activities. They will complete surveys about how they are feeling.\n\nParticipants will have a bone marrow biopsy. A needle will be inserted through a small cut. Bone marrow will be removed. A small piece of bone may be removed.\n\nParticipants may have an optional skin biopsy.\n\nParticipants may give optional saliva and stool samples. They may collect these samples at home and mail them to NIH.\n\nParticipants may undergo optional apheresis. One or two needles or intravenous (IV) lines will be placed in their arm, neck, or groin veins. Blood will be removed. A machine will separate out the white cells. The rest of the blood will be returned to the participant.\n\nParticipants will be contacted for follow-up once a year for up to 20 years.\n\nHealthy donors will have marrow collected for this study during their scheduled procedure with no follow-up.",[29],[532,533,534,535,536,66,138],"Heterogenous Stem Cell Disorders","Gene Mutations","Dysplasia","cytopenias","Malignancies",{"date":538,"type":44},"2026-06-22",{"date":540,"type":44},"2022-08-18",{"date":542,"type":22},"2042-05-01",{"name":50,"class":51},{"id":545,"slug":546,"hasResults":12,"nctId":547,"briefTitle":548,"officialTitle":549,"acronym":4,"eligibilityCriteria":550,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":551,"targetDuration":4,"studyType":23,"phases":553,"briefSummary":554,"conditions":555,"keywords":4,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":487,"lastUpdatePostDateStruct":557,"startDateStruct":558,"completionDateStruct":560,"leadSponsor":561,"locationsCount":563},"100407137","phase-1-study-to-evaluate-the-safety-and-tolerability-of-ep0042-100407137","NCT04581512","Study to Evaluate the Safety and Tolerability of EP0042","A Modular, Multipart, Multi-arm, Open-label, Phase I\u002FIIa Study to Evaluate the Safety and Tolerability of EP0042 Alone and in Combination With Anti-cancer Treatments in Patients With Advanced Malignancies","General\n\n1. Male or female patients aged ≥ 18 years of age, at the time of informed consent, with histological or cytological confirmation of AML\n2. Ability to understand and provide written informed consent before any study-specific procedures, sampling, or analyses, including access to archival tumor tissue\n3. Ability to swallow and retain oral medication\n4. Sufficient life expectancy to allow the patient to complete at least 1 cycle (28 days) of the treatment period.\n5. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2 at screening\n6. In the opinion of the investigator, all other relevant medical conditions must be well-managed and stable for at least 28 days prior to first administration of study drug\n7. Patients with pathologically confirmed\u002Fdocumented AML or MDS, as defined by the 2022 European LeukaemiaNet (ELN) recommendations, or CMML, as defined by World Health Organization (WHO) criteria, who have relapsed from or are refractory to previous therapy and have failed all (or are not eligible for\u002Fintolerant to) available approved therapies for their disease.\n8. Patients with relapsed\u002Frefractory FLT3 WT AML.\n9. Previous exposure to venetoclax, hypomethylating agent and\u002For FLT3 inhibitors (gilteritinib, midostaurin, quizartinib, sorafenib) is allowed for relapsed\u002Frefractory AML patients.\n10. Mutation status of patients must be known at trial entry.\n11. Female patients should either be of non-child-bearing potential or must agree to use highly effective methods of contraception from Screening until 6 months following administration of the last dose of study drug\n12. Male patients must use double barrier contraception from enrolment through treatment and for 6 months following administration of the last dose of study drug\n\n5.2 Core Exclusion Criteria\n\nPatients with any of the following will not be included in the study:\n\nDisease Under Study and Prior Anticancer Treatment\n\n1. Suspected brain and\u002For leptomeningeal metastases that are symptomatic or untreated or that require current therapy\n2. Acute promyelocytic leukemia (FAB:M3)\n3. Systemic anti-cancer therapy for the disease under study within 2 weeks of the first dose of study treatment. If the previous anti-cancer therapy has a very long half-life and may interact with EP0042, e.g. a strong CYP3A4 inhibitor, the washout period may need to be increased for safety reasons but will be no longer than 3 weeks (Concomitant hydroxyurea is acceptable and will be permitted throughout the screening period and during first 6 cycles of study treatment)\n4. Ongoing toxic manifestations of previous treatments that have not reduced to at least CTCAE Grade 1. Exceptions to this are alopecia or certain Grade 2 treatment related toxicities, which in the opinion of the Investigator should not exclude the patient.\n5. Transplantation (allogeneic or autologous) within last 90 days, or on active immunosuppressive therapy for graft versus host disease in last 2 weeks\n\n   Laboratory Parameters\n6. Patient with any out-of-range laboratory values defined as shown below.\n\n   • Creatinine clearance (calculated using Cockcroft-Gault formula, or measured) \\\u003C 50 mL\u002F min\n7. Inadequate liver function as demonstrated by\n\n   * total serum bilirubin ≥ 1.5 times the upper limits of normal range (ULN) or\n   * ALT ≥3 times the ULN or\n   * AST ≥3 times the ULN or\n   * AST or ALT ≥5 times the ULN in the presence of liver involvement by leukemia",{"count":552,"type":22},70,[25,26],"A research study looking at a new treatment for patients with advanced cancer, to investigate different doses of the experimental study drug, EP0042, in order to determine a dose, which is safe, well-tolerated and likely to be effective in treating AML (acute myeloid leukaemia).",[66,556,29],"Chronic Myelomonocytic Leukemia",{"date":538,"type":44},{"date":559,"type":44},"2020-11-02",{"date":165,"type":22},{"name":562,"class":82},"Ellipses Pharma",6,{"id":565,"slug":566,"hasResults":12,"nctId":567,"briefTitle":568,"officialTitle":569,"acronym":4,"eligibilityCriteria":570,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":571,"targetDuration":4,"studyType":23,"phases":573,"briefSummary":574,"conditions":575,"keywords":578,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":580,"lastUpdatePostDateStruct":581,"startDateStruct":583,"completionDateStruct":585,"leadSponsor":587,"locationsCount":589},"100526733","phase-1-preemptive-ciml-nk-cell-therapy-after-hematopoietic-stem-cell-transplantation-100526733","NCT06138587","Preemptive CIML NK Cell Therapy After Hematopoietic Stem Cell Transplantation","Cytokine-induced Memory-like Natural Killer Cell Therapy After Hematopoietic Stem Cell Transplantation for Eradication of Measurable Residual Disease, a Phase I\u002FIb Clinical Trial","Inclusion Criteria for Trial Enrollment:\n\n* Histologically or cytologically confirmed diagnosis of AML, MDS, or MDS\u002FMPN that is at high risk for post-transplant relapse and that has measurable disease prior to transplant. Patients at high risk for post-transplant relapse include:\n\n  * De novo AML diagnosed at or after age 60, except CBF AML\n  * De novo AML in CR1 AND MRD+ by Hematologics Inc. flow cytometry pretransplant (this would be on the most recent pre-transplant bone marrow)\n  * Secondary AML\n  * AML with 5 - \\> 10% blasts at the time of transplant who are judged by their treating clinician and study PI to have no benefit from additional pre-transplant chemotherapy\n  * Any AML transplanted in CR2 or greater\n  * TP53-mutated MDS or AML\n  * Therapy-related MDS or AML\n  * MDS with monosomy 7\n  * MDS with \\>= 5% blasts at the time of transplant\n  * MDS\u002FMPN or CMML\n* Adequate organ function within 2 weeks of NK cell infusion as defined below (should correspond with admission for SCT):\n\n  * Total bilirubin: ≤1.5 x institutional upper limit of normal (ULN) (except Gilbert's or disease related hemolysis, then \\\u003C 3 x ULN)\n  * AST(SGOT)\u002FALT(SGPT): ≤3 x institutional ULN\n  * Serum creatinine \\\u003C\u002F= 2.0mg\u002FdL\n  * O2 saturation: ≥90% on room air\n  * LVEF \\>40%. If there is no clinical evidence of a change in cardiovascular function from the time of pre-transplantation ECHO (per FACT standards should be performed within 6 weeks of stem cell infusion), then there is no need to repeat it. Otherwise, an ECHO will need to be repeated.\n* Adult patients (age ≥ 18) eligible for and planned to undergo a standard-of-care reduced intensity conditioning (RIC) HLA-matched related or related haploidentical allogeneic stem cell transplant using PTCY-based GVHD prophylaxis. All eligibility criteria and workups for undergoing SOC allogeneic SCT for the recipient and donor will be based on institutional standards and SOPs.\n* For patients with AML, the disease must meet criteria for CR\u002FCri according to 2017 ELN guidelines, or have 5-10% blasts at the time of the transplant and in the judgement of their primary BMT clinician and study PI to have no benefit from additional pre-transplant chemotherapy. For patients with MDS or MDS\u002FMPN, the blast percentage on the bone marrow aspirate and biopsy must be less than 10%.\n* The same related donor is available to provide a non-mobilized apheresis product after the stem-cell donation.\n* ECOG performance status \\\u003C= 2 (Karnofsky \\>= 60%, see Appendix C).\n* Negative pregnancy test for women of childbearing age\n* The effects of CIML NK cells combined with IL-2 on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after IL-2 dose administration.\n* No laboratory evidence of ongoing hemolysis in opinion of investigator\n\nExclusion Criteria Trial Enrollment:\n\n* Adult participants who are eligible for and who would be expected to have a greater benefit from myeloablative conditioning in their SOC allo HSCT as judged by their treating physician\n* Participants with mutations such as FLT3-ITD, IDH, or BCR-ABL mutations who are planned to receive targeted agent maintenance therapy to prevent relapse post-transplant are excluded.\n* Extramedullary leukemia involving sanctuary sites not readily accessible to immune surveillance, such as CNS or testis. Other sites of extramedullary relapse (e.g., leukemia cutis, granulocytic sarcoma) are acceptable.\n* The planned use of sirolimus for GVHD prophylaxis would result in exclusion of the patient from the study. Consideration of the addition of sirolimus to the GVHD prophylaxis regimen within the first 100 days after transplant must be reviewed with the study PI.\n* Prior history of allogeneic stem cell transplant other than SOC alloHSCT referred to in this study taking place 7 days prior to CIML NK infusion.\n* Prior history of solid organ (allograft) transplantation\n* Prior history of allergic reactions to cellular products\n* Uncontrolled concurrent illness such as ongoing or active infection, symptomatic congestive heart failure, unstable angina, cardiac arrhythmia, or psychiatric\u002Fsocial illness that would limit compliance with study requirements.\n* Pregnant women are excluded from this study because of the unknown teratogenic risk of CIML NK cells and IL-2 and with the potential for teratogenic or abortifacient effects by Flu\u002FCy chemotherapy regimen. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with CIML NK cells and IL-2, breastfeeding should be discontinued if the mother is treated on this study.\n* HIV-positive patients are excluded due to the potential for interaction between antiretroviral therapy as well as the risk for lethal infection in the context of marrow-suppressive therapy.\n* Patients with active and uncontrolled Hepatitis B or C are ineligible due to the high risk of treatment-related hepatotoxicity after cellular therapy.\n* Individuals with a history of a different malignancy are ineligible except for the following circumstances: 1. History of other malignancy and have had complete remission of disease for at least 2 years; 2. Diagnosed and treated within the past 2 years for: nonmetastatic melanoma, surgically resected (not needing systemic chemotherapy) squamous cell carcinoma of skin and nonmetastatic prostate cancer not needing systemic chemotherapy.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to IL-2 or other agents used in study. 3. Diagnosed with MGUS or smoldering myeloma, and\u002For treated for multiple myeloma or plasmacytoma as long as attainment of complete remission by IMWG criteria following therapy.\n* Prior history of Grade 2 or higher hemolytic anemia (\\>\u002F= 2g decrease in hemoglobin plus laboratory evidence of hemolysis) from any cause.\n\nInclusion Criteria to Receive CIML NK Infusion\n\n* Adequate organ function within 24 hours of NK cell infusion as defined below:\n\n  * Total bilirubin: ≤1.5 x institutional upper limit of normal (ULN) (except Gilbert's or disease-related hemolysis, then \\\u003C 3 x ULN)\n  * AST(SGOT)\u002FALT(SGPT): ≤3 x institutional ULN\n  * Grade ≥3 non-hematologic toxicities of cyclophosphamide and fludarabine conditioning (except for Grade 3 nausea, vomiting, diarrhea, or constipation).\n* No significant change in clinical status that would, in the opinion of the investigator, increase the risk of adverse events associated with CIML NK infusion, (e.g., symptomatic congestive heart failure, unstable angina, cardiac arrhythmia)\n* No evidence of ongoing hemolysis in opinion of investigator\n\nExclusion Criteria to Receive CIML NK Infusion:\n\n* Systemic steroid therapy (oral or IV) on the day of NK cell infusion\n* Uncontrolled concurrent illness such as ongoing or active infection, symptomatic congestive heart failure, unstable angina, cardiac arrhythmia, or psychiatric\u002Fsocial illness that would limit compliance with study requirements.\n* Participants who have had other investigational agents within 4 weeks prior to CIML NK cell infusion (6 weeks for nitrosoureas or mitomycin C), or immunotherapy within 8 weeks prior, or those who have not recovered from adverse events due to agents administered more than 4 weeks prior. Therapy with BCR-ABL inhibitors must be stopped at least 2 weeks before CIML NK cell infusion and may not be resumed during the DLT period. The use of tocilizumab for cytokine release syndrome after stem cell infusion does not exclude patients, but the use of steroids for the treatment of CRS excludes patients if they are still on steroids by the day of planned NK cell infusion.\n* Systemic steroid use of \\>10mg\u002Fday of prednisone equivalent is an exclusion criteria unless there is a plan for dose to be tapered below this limit within 4 weeks prior to NK cell infusion.\n\nPatients must be off systemic steroid therapy on the day of planned NK cell infusion.\n\n-The presence of donor-specific antibodies (DSAs) with mean fluorescence intensity (MFI) \\>1000 using a standard assay who do not receive a desensitization protocol prior to and during stem cell transplant, or else who do receive a desensitization protocol and have detectable DSAs +1 day after stem cell infusion.\n\nIf inclusion\u002Fexclusion criteria are not met on planned day of CIML NK cell infusion, the NK cell infusion may be delayed for up to 48 hours to enable inclusion criteria to be met.\n\nHowever, patient may still receive CIML NK infusion if relevant parameters are reviewed and both PI and IND holder are in agreement with proceeding.",{"count":572,"type":22},15,[25],"The purpose of this research study is to test the safety and efficacy of cytokine induced memory-like (CIML) natural killer (NK) cells expanded with Interleukin-2 (IL-2) at preventing relapse in acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or MDS and myeloproliferative neoplasm (MPN) overlap syndrome after a standard-of-care stem cell transplant.\n\nNames of the study therapies involved in this study are:\n\n* CIML NK cells intravenous infusion (cellular therapy)\n* Subcutaneous Interleukin-2 (recombinant, human glycoprotein)",[66,96,576,29,440,577],"Leukemia, Myeloid","Myeloproliferative Disorders",[66,96,576,31,579],"MDS and Myeloproliferative Neoplasm Overlap Syndrome","2026-06-15",{"date":582,"type":44},"2026-06-17",{"date":584,"type":44},"2024-01-24",{"date":586,"type":22},"2027-11-30",{"name":588,"class":108},"Dana-Farber Cancer Institute",2,{"id":591,"slug":592,"hasResults":12,"nctId":593,"briefTitle":594,"officialTitle":594,"acronym":4,"eligibilityCriteria":595,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":596,"targetDuration":4,"studyType":23,"phases":598,"briefSummary":599,"conditions":600,"keywords":602,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":580,"lastUpdatePostDateStruct":603,"startDateStruct":604,"completionDateStruct":606,"leadSponsor":608,"locationsCount":589},"100364402","phase-1-a-phase-1-trial-of-ciml-nk-cell-infusion-for-myeloid-disease-relapse-after-hematopoietic-cell-transplantation-100364402","NCT04024761","A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation","1. Inclusion Criteria:\n\n   1.1 Relapse or post-transplant persistence of AML, MDS (including JMML) or MPN (CMML, myelofibrosis or MDS\u002FMPN). Disease relapse or persistence will be defined as any measurable disease by morphology, flow-cytometry, validated tests for minimal residual disease or disease-defining mutations in the bone marrow, or non-immune privileged extramedullary sites.\n\n   1.2 Persistence of disease within 4 weeks before planned NK cell infusion and at least 2 weeks after completion of immune suppression taper as long as it is \\> 2 months after stem cell transplantation for both adult and pediatric patients. If 2 weeks after completion of the immune suppression taper is still within 2 months of the most recent stem cell transplant, then chemotherapy with Fludarabine\u002FCyclophosphamide would need to start no earlier than at least 2 months after the transplant. For adults, disease persistence after a second transplant is allowed as long as the most recent transplant was a haploidentical or HLA matched stem cell transplant. In the pediatric cohort, disease persistence or recurrence after a second transplant is allowed as long as the most recent transplant was a haploidentical or matched related donor SCT.\n\n   1.3 Available original donor (same donor as used for the most recent haploidentical or HLA matched stem cell transplant for adults, or for the most recent matched related donor or related haploidentical donor for pediatrics) that is willing and eligible for non-mobilized collection.\n\n   1.4 Age ≥12 years.\n\n   1.5 ECOG performance status ≤2. For For patients in the pediatric cohort, this corresponds to a Lansky (patients \\\u003C16 years) or Karnofsky (≥16years) performance status of ≥50.\n\n   1.6 T cell chimerism ≥20% donor-derived within the 4 weeks prior to cell infusion.\n\n   1.7 Patient with ≤80% bone marrow involvement within 4 weeks prior to cell infusion. Medications like hydroxyurea, decitabine or cytarabine are allowed to control rising blasts between study enrollment and cell infusion.\n\n   1.8 No systemic corticosteroid therapy for GVHD (≤ 5mg of prednisone or equivalent dose of systemic steroids for non-GVHD, non-autoimmune indications are allowed) for at least 4 weeks prior to cell infusion. Patients on systemic GVHD prophylaxis medications such as tacrolimus or sirolimus need to be off these medications for at least 4 weeks prior to cell infusion.\n\n   1.9 No other systemic medications\u002Ftreatments (e.g. ECP) for GVHD for at least 4 weeks prior to cell infusion.\n\n   1.10 Ability of the patient or legal guardian to understand and the willingness to sign a written informed consent document.\n\n   1.11 Adequate organ function within 2 weeks of NK cell infusion as defined below:\n   * Total bilirubin: ≤1.5 x institutional upper limit of normal (ULN) (except Gilbert's or disease-related hemolysis, then \\\u003C3 x ULN)\n   * AST(SGOT)\u002FALT(SGPT): ≤3 x institutional ULN\n   * Serum creatinine ≤2.0mg\u002FdL\n   * O2 saturation: ≥90% on room air\n   * LVEF \\>40%. If there is no clinical evidence of a change in cardiovascular function from the time of pre-transplantation ECHO, then there is no need to repeat it. Otherwise, an ECHO will need to be repeated within 2 weeks of NK cell infusion.\n\n   1.12 Negative pregnancy test for women of childbearing potential only.\n\n   1.13 The effects of CIML NK cells and IL-2 on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after the last IL-2 dose administration.\n2. Exclusion Criteria:\n\n2.1 Extramedullary relapse involving immuno-privileged sites (e.g. CNS, testes, eyes). Other sites of extramedullary relapse (e.g. leukemia cutis, granulocytic sarcoma) are acceptable.\n\n2.2 Participants who have had investigational agents within 4 weeks prior to cell infusion (6 weeks for nitrosoureas or mitomycin C), or immunotherapy within 8 weeks prior, or those who have not recovered from adverse events due to agents administered more than 4 weeks prior or standard chemotherapy administered more than 14 days ago. Use of hydroxyurea, hypomethylating agents, low-dose cytarabine or venetoclax to control counts within 4 weeks prior to cell infusion is permitted with study PI approval but would need to be stopped 1 day prior to administration of Fludarabine and Cyclophosphamide preceding the NK cell infusion (provided that there are no ongoing AEs attributed to these agents that would preclude start of lymphodepletion in the view of the investigator). Patients on standard of care FLT-3, IDH1, and IDH2 inhibitors can stay on this treatment. Therapy with BCR-ABL inhibitors or bcl-2 inhibitors must be stopped 2 weeks before NK cell infusion and may be resumed after the end of the DLT period.\n\n2.3 Prior history of Donor Lymphocyte Infusion (DLI) within 8 weeks of CIML NK infusion. DLI that was given before this time period and that did not result in any GVHD requiring systemic treatment is not an exclusion criterion.\n\n2.4 Prior history of severe (grade 3 or 4) acute GVHD, or ongoing active GVHD requiring systemic treatment.\n\n2.5 Solid organ transplant recipient. Prior allogeneic HLA matched or mismatched stem cell transplant is allowed in the pediatric cohort. Prior HLA matched related donor or HLA matched unrelated donor stem cell transplant is allowed in the adult cohort.\n\n2.6 History of allergic reactions attributed to compounds of similar chemical or biologic composition to IL-2 or other agents used in study.\n\n2.7 Autoimmune disease: Patients with a history of inflammatory bowel disease, including ulcerative colitis and Crohn's Disease, are excluded from this study, as are patients with a history of symptomatic disease (e.g., rheumatoid arthritis, systemic progressive sclerosis \\[scleroderma\\], systemic lupus erythematosus, autoimmune vasculitis \\[e.g., Wegener's Granulomatosis\\]) and motor neuropathy considered of autoimmune origin (e.g. Guillain-Barre Syndrome and Myasthenia Gravis). Patients with Hashimoto's thyroiditis are eligible to go on study.\n\n2.8 Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n\n2.9 Patients who develop a critical illness prior to NK cell infusion that would contraindicate the administration of Fludarabine and Cyclophosphamide conditioning. Patients who recover from such illness may still be eligible, but this must be reviewed with the study PI. A repeat bone marrow examination may be required depending on the timing of recovery. Patients who become critically ill on the planned day of NK cell infusion are excluded if the NK cell infusion cannot be given within 48 hours of the planned day 0.\n\n2.10 Pregnant women are excluded from this study because of the unknown teratogenic risk of CIML NK cells and IL-2 and with the potential for teratogenic or abortifacient effects by Flu\u002FCy chemotherapy regimen. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with CIML NK cells and IL-2, breastfeeding should be discontinued if the mother is treated on this study.\n\n2.11 HIV-positive participants are ineligible because of the potential for pharmacokinetic interactions with anti-retroviral agents used in this study. In addition, these participants are at increased risk of lethal infections when treated with marrow-suppressive therapy.\n\n2.12 Individuals with active uncontrolled hepatitis B or C, HIV, or HTLV-1 are ineligible as they are at high risk of lethal treatment-related hepatotoxicity after HSCT.\n\n2.13 Individuals with a history of a different malignancy are ineligible except for the following circumstances: 1. History of other malignancy and have had complete remission of disease for at least 2 years; 2. Diagnosed and treated within the past 2 years for: nonmetastatic melanoma, surgically resected (not needing systemic chemotherapy) squamous cell carcinoma of skin and nonmetastatic prostate cancer not needing systemic chemotherapy.",{"count":597,"type":22},50,[25],"This research study is studying cytokine induced memory-like natural killer (CIML NK) cells combined with IL-2 in adult patients (18 years of age or older) with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) and Myeloproliferative Neoplasms (MPN) who relapse after haploidentical hematopoietic cell transplantation (haplo-HCT) or HLA matched stem cells. This study will also study CIML NK cell infusion combined with IL-2 in pediatric patients (12 years of age or older) with AML, MDS, JMML who relapse after stem cell transplantation using HLA-matched related donor or related donor haploidentical stem cells.",[66,29,440,601],"Juvenile Myelomonocytic Leukemia",[96],{"date":582,"type":44},{"date":605,"type":44},"2019-08-31",{"date":607,"type":22},"2026-12-01",{"name":588,"class":108},{"id":610,"slug":611,"hasResults":12,"nctId":612,"briefTitle":613,"officialTitle":614,"acronym":4,"eligibilityCriteria":615,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":616,"targetDuration":4,"studyType":23,"phases":618,"briefSummary":619,"conditions":620,"keywords":624,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":632,"lastUpdatePostDateStruct":633,"startDateStruct":634,"completionDateStruct":636,"leadSponsor":638,"locationsCount":640},"100214912","phase-1-study-of-orally-administered-ag-120-in-subjects-with-advanced-hematologic-malignancies-with-an-idh1-mutation-100214912","NCT02074839","Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation","A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation","Key Inclusion Criteria:\n\n* Subject must be ≥18 years of age.\n* Subjects must have documented IDH1 R132 gene-mutated advanced hematologic malignancy based on local or central evaluation.\n* Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study.\n* Subjects must have ECOG PS of 0 to 2.\n* Platelet count ≥20,000\u002FµL (Transfusions to achieve this level are allowed).\n* Subjects must have adequate hepatic function as evidenced by: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic disease\n* Subjects must have adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance \\>40mL\u002Fmin based on Cockroft-Gault glomerular filtration rate (GFR)\n* Subjects must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or other therapy intended for the treatment of cancer.\n* Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy and on the first day of study drug administration.\n\nKey Exclusion Criteria:\n\n* Subjects who have undergone hematopoietic stem cell transplant (HSCT) within 60 days of the first dose of AG-120, or subjects on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). (The use of a stable dose of oral steroids post HSCT and\u002For topical for ongoing skin GVHD is permitted.)\n* Subjects who received systemic anticancer therapy or radiotherapy \\\u003C14 days prior to their first day of study drug administration. (Hydroxyurea is allowed prior to enrollment and after the start of AG-120).\n* Subjects who received an investigational agent \\\u003C14 days prior to their first day of study drug administration.\n* Subjects who are pregnant or breastfeeding.\n* Subjects with an active severe infection or with an unexplained fever \\>38.5°C during screening visits or on their first day of study drug administration (at the discretion of the Investigator, subjects with tumor fever may be enrolled).\n* Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \\\u003C40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within approximately 28 days of C1D1.\n* Subjects with a history of myocardial infarction within the last 6 months of screening.\n* Subjects with a known unstable or uncontrolled angina pectoris.\n* Subjects with a known history of severe and\u002For uncontrolled ventricular arrhythmias.\n* Subjects with known unstable or uncontrolled angina pectoris.\n* Subjects with heart-rate corrected QT (QTc) interval ≥450 ms or other factors that increase the risk of QT prolongation or arrhythmic events.\n* Patients taking medications that are known to prolong the QT interval\n* Subjects with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C.\n* Subjects with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is a clinical suspicion of CNS involvement by leukemia during screening.\n* Subjects with immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and\u002For disseminated intravascular coagulation.",{"count":617,"type":22},291,[25],"The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and\u002For the recommended Phase II dose. The second portion of the study is a dose expansion phase where four cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Additionally, the study includes a substudy evaluating the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1 mutation. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.",[621,622,623,29],"Relapsed or Refractory Acute Myeloid Leukemia (AML)","Untreated AML","Other IDH1-mutated Positive Hematologic Malignancies",[625,72,626,73,627,628,622,629,630,631],"acute myeloid leukemia","myelodysplastic syndrome","hematologic malignancies","IDH","IDH1","relapsed AML","refractory AML","2026-06-11",{"date":580,"type":44},{"date":635,"type":44},"2014-03-01",{"date":637,"type":22},"2026-12-31",{"name":639,"class":108},"Institut de Recherches Internationales Servier",30,{"id":642,"slug":643,"hasResults":12,"nctId":644,"briefTitle":645,"officialTitle":646,"acronym":73,"eligibilityCriteria":647,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":648,"targetDuration":4,"studyType":23,"phases":649,"briefSummary":650,"conditions":651,"keywords":654,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":660,"lastUpdatePostDateStruct":661,"startDateStruct":663,"completionDateStruct":665,"leadSponsor":667,"locationsCount":4},"100622725","phase-1-pacritinib-with-aza-for-upfront-myelodysplastic-syndrome-100622725","NCT07387354","Pacritinib With Aza for Upfront Myelodysplastic Syndrome","A Phase 1\u002F2 Study of Pacritinib in Combination With Azacitidine for the Treatment of IPSS-M Moderate Low to Very High Risk Myelodysplastic Syndrome","Inclusion Criteria:\n\n* Individuals must meet all of the following inclusion criteria to be eligible to participate in the study:\n\n  1. Patients must have histologic evidence of intermediate to high-risk myelodysplastic syndrome defined as having an IPSS-M score of moderate low, moderate high, high or very high risk. This will be assessed based on evaluations performed prior to screening for trial. Of note, the most recent evaluation pre-trial may be used which does not have to necessarily be at diagnosis.\n  2. Subjects must have recovered from the toxic effects of any prior chemotherapy to ≤ Grade 1 (except alopecia).\n  3. Required screening visit laboratory values: CrCL ≥45; total bilirubin \\\u003C2xULN except for patients with known Gilbert's disease; SGPT (ALT) ≤2xULN, PTT ≤1.5xULN.\n  4. Negative pregnancy test for women with child-bearing potential at screening visit.\n  5. Initial screening baseline QTc ≤480ms.\n  6. Patients must be able to sign consent and be willing and able to comply with scheduled visits, treatment plan and laboratory testing.\n  7. Patients must have an absolute neutrophil count of ≥750 to enroll in study, this must be achieved without the addition of growth factor medication.\n\nExclusion Criteria:\n\n* An individual who meets any of the following criteria will be excluded from participation in this study:\n\n  1. Any prior exposure to a hypomethylating agent (azacitidine or decitabine)\n  2. Any prior exposure to JAK2 inhibitor therapy (ie ruxolitinib or prior pacritinib therapy)\n  3. Any exposure within the past seven days of initiation of study treatment to a strong CYP3A inhibitor\u002Finducer.\n  4. Subjects must not be receiving any chemotherapy agents (except hydroxyurea) within the past thirty days.\n  5. Subjects must not be receiving growth factors (erythropoietin mimetics, granulocyte stimulating factor mimetics, thrombopoietin mimetics) for two weeks prior to enrollment bone marrow. Subjects may not receive growth factors for the duration of this study.\n  6. Subjects with a \"currently active\" second malignancy, other than curatively treated non-melanoma skin cancer, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and postoperative PSA \\\u003C0.5 ng\u002FmL), or other adequately treated carcinoma-in-situ are eligible. Patients are not considered to have a \"currently active\" malignancy if they have completed therapy and are free of disease for ≥ 1 year.\n  7. Subjects with uncontrolled high blood pressure, unstable angina, symptomatic congestive heart failure (NYHA class 2), myocardial infarction within the past 6 months or serious uncontrolled cardiac arrhythmia are not eligible.\n  8. Bleeding event grade \\>=2 (CTCAE 5.0) within prior three months unless provoked (e.g., by surgery or trauma)\n  9. Use of anticoagulant or antiplatelet agents within fourteen days prior to day one with the exception of low dose aspirin (81mg daily).\n  10. Subjects with other severe concurrent disease which in the judgment of the investigator would make the patient inappropriate for entry into this study are ineligible.\n  11. Active or uncontrolled diarrhea or constipation.\n  12. Subjects must not have evidence of active disease in the CNS.\n  13. Subjects must not have received any investigational agents within fourteen days or five half-lives (whichever is longer) of study entry.\n  14. Subjects must not be pregnant or breastfeeding. Pregnancy tests must be obtained for all females of child-bearing potential. Pregnant or lactating patients are ineligible for this study due to the unknown human fetal or teratogenic toxicities of pacritinib. Males or women of childbearing potential may not participate unless they have agreed to use a highly effective contraceptive method (defined in section 10.4.4).\n  15. Subjects who have uncontrolled infection are not eligible. Patients must have any active infections under control. Fungal disease must be stable for at least two weeks before study entry.\n  16. Subjects with bacteremia must have documented negative blood cultures prior to study entry.\n  17. Subjects who are currently candidates for allogeneic transplantation, have a suitable donor, and are willing to undergo transplantation prior to study start.\n  18. Subjects who cannot hold a medication, over the counter, or supplemental product that in the investigator's opinion may put the patient at increased harm.",{"count":7,"type":22},[25,26],"This study will be conducted as a phase 1\u002F2 study of safety and preliminary efficacy of pacritinib in combination with azacitidine for IPSS-M moderate low to very high risk MDS. Phase one will be a 3 + 3 design to assess the dose for the phase two portion. The phase two portion will employ a simon min-max two-stage design whereby fifteen patients will be enrolled in the first stage then ten more if at least two patients in stage one have a response. The dosing of pacritinib for the phase two study will be based on the phase one findings. Standard dosing of azacitidine will be used. A correlative study will be conducted in conjunction with the trial where the investigators will measure whole blood collected pre-treatment and at four days post-treatment to measure intracellular flow and phosflow to detect JAK\u002FSTAT, NF-κβ, and AKT\u002FmTOR signaling in patient samples and how treatment affects these pathways.",[29,73,652,476,653,440],"Myelodysplastic Syndrome, Unclassifiable","Bone Marrow Disease",[655,70,656,657,658,659,31],"Pacritinib","IPSS-M","JAK\u002FSTAT","JAK\u002FIRAK1","NF-κβ","2026-06-08",{"date":662,"type":44},"2026-06-10",{"date":664,"type":22},"2026-07",{"date":666,"type":22},"2027-01",{"name":668,"class":108},"Thomas Jefferson University",{"id":670,"slug":671,"hasResults":12,"nctId":672,"briefTitle":673,"officialTitle":674,"acronym":675,"eligibilityCriteria":676,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":677,"targetDuration":4,"studyType":23,"phases":678,"briefSummary":679,"conditions":680,"keywords":687,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":660,"lastUpdatePostDateStruct":694,"startDateStruct":696,"completionDateStruct":698,"leadSponsor":700,"locationsCount":702},"100434327","phase-2-faecal-microbiota-transplantation-after-allogeneic-stem-cell-transplantation-100434327","NCT04935684","Faecal Microbiota Transplantation After Allogeneic Stem Cell Transplantation","Faecal Microbiota Transplantation for Prevention of Graft-versus-host Sisease After Allogeneic Stem Cell Transplantation for Haematological Malignancies","TMF-Allo","Inclusion Criteria:\n\n* Patient aged 18 or over\n* Men and women\n* Patients affiliated with a social-security organization\n* Patients undergoing a myelo-ablative allo-HSCT for a controlled haematologic malignant disease, with peripheral stem cells, whatever the type of donor (except cord blood)\n* Signed and dated informed consent\n\nExclusion Criteria:\n\n* Status of tumor progression at the time of allo-HSCT\n* Inability to understand the protocol (linguistic barrier, cognitive difficulties)\n* Medical history of another progressive cancer or occurrence in the 3 previous years (excluding basal cell carcinoma)\n* Presence of a simultaneous serious and uncontrolled disease (severe cardiac, renal, hepatic or respiratory failure, severe sepsis)\n* Fecal incontinence\n* Participation in another clinical trial studying an allograft procedure including the type of graft, the type of immunosuppression, a preventive or a curative treatment of GvHD, or studying the effectiveness of a FMT in another indication.\n* Pregnant women\n* Patient under guardianship, curatorship or protection of justice",{"count":5,"type":22},[26],"The aim of this study is to assess the Fecal Microbiota Transplantation (FMT) efficacy in the prevention of allogeneic hematopoietic stem cell transplantation (allo-HSCT) complications and particularly Graft versus Host Disease (GvHD).\n\nThe hypothesis of this study is that allogeneic FMT may improve outcomes of these patients.",[681,29,682,683,684,685,686],"Acute Leukemia in Remission","Myeloproliferative Syndrome","Hodgkin Lymphoma","Lymphoma, Non-Hodgkin","Myeloma","Chronic Lymphocytic Leukemia",[688,689,690,691,692,693],"Allogeneic hematopoietic stem cell transplantation","Hematologic malignancies","Graft versus Host Disease","Intestinal microbiota","Intestinal dysbiosis","Fecal Microbiota Transplantation",{"date":695,"type":44},"2026-06-09",{"date":697,"type":44},"2022-12-20",{"date":699,"type":22},"2030-05",{"name":701,"class":108},"University Hospital, Clermont-Ferrand",20,{"id":704,"slug":705,"hasResults":12,"nctId":706,"briefTitle":707,"officialTitle":707,"acronym":4,"eligibilityCriteria":708,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":709,"targetDuration":711,"studyType":130,"phases":4,"briefSummary":712,"conditions":713,"keywords":726,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":730,"lastUpdatePostDateStruct":731,"startDateStruct":732,"completionDateStruct":734,"leadSponsor":736,"locationsCount":52},"100264360","investigation-of-the-genetics-of-hematologic-diseases-100264360","NCT02720679","Investigation of the Genetics of Hematologic Diseases","Inclusion Criteria:\n\n* An individual (proband) receiving therapy or expert consultation regarding a non-malignant hematologic disorder, MDS or MPN.\n* A biologically-related individual to the identified proband to include: first, second or third degree relatives.\n\nExclusion Criteria:\n\n* None",{"count":710,"type":22},1716,"10 Years","The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings, urine, saliva and buccal swabs, left over tissue, as well as health information will be used to study and learn about blood diseases by using genetic and\u002For genomic research. In general, genetic research studies specific genes of an individual; genomic research studies the complete genetic makeup of an individual.\n\nIt is not known why many people have blood diseases, because not all genes causing these diseases have been found. It is also not known why some people with the same disease are sicker than others, but this may be related to their genes. By studying the genomes in individuals with blood diseases and their family members, the investigators hope to learn more about how diseases develop and respond to treatment which may provide new and better ways to diagnose and treat blood diseases.\n\nPrimary Objective:\n\n* Establish a repository of DNA and cryopreserved blood cells with linked clinical information from individuals with non-malignant blood diseases and biologically-related family members, in conjunction with the existing St. Jude biorepository, to conduct genomic and functional studies to facilitate secondary objectives.\n\nSecondary Objectives:\n\n* Utilize next generation genomic sequencing technologies to Identify novel genetic alternations that associate with disease status in individuals with unexplained non-malignant blood diseases.\n* Use genomic approaches to identify modifier genes in individuals with defined monogenic non-malignant blood diseases.\n* Use genomic approaches to identify genetic variants associated with treatment outcomes and toxicities for individuals with non-malignant blood disease.\n* Use single cell genomics, transcriptomics, proteomics and metabolomics to investigate biomarkers for disease progression, sickle cell disease (SCD) pain events and the long-term cellular and molecular effects of hydroxyurea therapy.\n* Using longitudinal assessment of clinical and genetic, study the long-term outcomes and evolving genetic changes in non-malignant blood diseases.\n\nExploratory Objectives\n\n* Determine whether analysis of select patient-derived bone marrow hematopoietic progenitor\u002Fstem (HSPC) cells or induced pluripotent stem (iPS) cells can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms.\n* Determine whether analysis of circulating mature blood cells and their progenitors from selected patients with suspected or proven genetic hematological disorders can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms.",[714,715,716,717,718,719,720,721,722,723,724,29,725],"Bone Marrow Failure Syndromes","Erythrocyte Disorder","Leukocyte Disorder","Hemostasis","Blood Coagulation Disorder","Sickle Cell Disease","Dyskeratosis Congenita","Diamond-Blackfan Anemia","Congenital Thrombocytopenia","Severe Congenital Neutropenia","Fanconi Anemia","Myeloproliferative Diseases",[727,728,729],"Genetics","Whole genome sequencing","Pediatrics and hematology","2026-06-05",{"date":695,"type":44},{"date":733,"type":44},"2016-06-17",{"date":735,"type":22},"2050-07",{"name":737,"class":108},"St. Jude Children's Research Hospital",{"id":739,"slug":740,"hasResults":12,"nctId":741,"briefTitle":742,"officialTitle":742,"acronym":4,"eligibilityCriteria":743,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":502,"enrollmentInfo":744,"targetDuration":4,"studyType":23,"phases":745,"briefSummary":746,"conditions":747,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":748,"lastUpdatePostDateStruct":749,"startDateStruct":750,"completionDateStruct":752,"leadSponsor":754,"locationsCount":52},"100643551","phase-2-automated-total-marrow-and-lymphoid-irradiation-for-allogeneic-hematopoietic-cell-transplant-100643551","NCT07634536","Automated Total Marrow and Lymphoid Irradiation for Allogeneic Hematopoietic Cell Transplant","Inclusion Criteria for 20 Gy Arm (Cohort A)\n\n1. Age, Performance Status, and Graft Criteria require all of the following bullet points:\n\n   * Age 18 to 60 years (inclusive)\n   * HCT Co-Morbidity score (HCT-CI) \\\u003C 5 (http:\u002F\u002Fwww.qxmd.com\u002Fcalculate-online\u002Fhematology\u002Fhct-ci)(31)\n   * Adequate performance status is defined as Karnofsky score ≥ 70%\n   * Patients must be receiving an allogeneic peripheral blood stem cell graft\n   * Patients and selected donor must be HLA typed at high resolution using DNA based typing at the following HLA-loci: HLA-A, -B, -C and DRB1. Donors may be an 8\u002F8 matched sibling donor, 8\u002F8 matched unrelated donor, haploidentical related donor, or 7\u002F8 mismatched unrelated donor.\n2. Eligible Diseases (Any one of the following)\n\n   Acute Myeloid Leukemia (AML) Must have at least one of the following characteristics:\n   * Blasts \\>5% in the peripheral blood and\u002For bone marrow after \\>2 prior lines of AML directed therapy, present during the trial screening window\n   * Adverse plus risk by AlloHCT Refined ELN Criteria: defined as having complex cytogenetics, TP53 mutation, or MECOM rearrangement confirmed at any time point.(32)\n\n   Myelodysplastic syndrome Must have at least one of the following characteristics at the time of conditioning:\n   * Blasts \\>10% in the peripheral blood and\u002For bone marrow after \\>1 prior line of therapy.\n   * TP53 mutation confirmed at any time point\n\n   Myeloproliferative neoplasms (MPN) or MDS\u002FMPN overlap. Must have at least one of the following characteristics:\n   * Blasts \\>10% in the peripheral blood and\u002For bone marrow during the trial screening window\n   * TP53 mutation confirmed at any time point\n3. Adequate organ function is defined as all of the following:\n\n   Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 45% confirmed by MUGA or echocardiography Pulmonary: DLCO, FEV1, FVC \\> 50% predicted, and absence of O2 requirements. Liver: Transaminases \\\u003C 3 x upper limit of normal (ULN) and total bilirubin ≤ 2 mg\u002FdL except for patients with Gilbert's syndrome or hemolysis (as indicated by provider documentation).\n\n   Renal: Creatinine \\\u003C 2.0 mg\u002FdL (adults) and creatinine clearance \\> 40 mL\u002Fmin.\n4. Must be FIRST allogeneic HCT\n5. Sexually active females of childbearing potential and males with partners of child-bearing potential must agree to use adequate birth control during study treatment.\n6. Voluntary written consent\n\nInclusion Criteria for 12 Gy Arm (Cohort B)\n\n1. Age, Performance Status, and Graft Criteria require all of the following bullet points:\n\n   Age 18 to 70 years (inclusive) Adequate performance status is defined as Karnofsky score ≥ 70% Patients must be receiving an allogeneic peripheral blood stem cell graft Patients and selected donor must be HLA typed at high resolution using DNA based typing at the following HLA-loci: HLA-A, -B, -C and DRB1. Donors may be an 8\u002F8 matched sibling donor, 8\u002F8 matched unrelated donor, haploidentical related donor, or 7\u002F8 mismatched unrelated donor.\n2. Eligible Diseases (Any of the following) Acute Myeloid Leukemia (AML) Myelodysplastic syndrome Myeloproliferative neoplasm MDS\u002FMPN overlap\n3. Must have relapse after prior allo HCT\n4. Adequate organ function is defined as all of the following:\n\n   Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 40% confirmed by MUGA or echocardiography Pulmonary: DLCO, FEV1, FVC \\> 40% predicted, and absence of O2 requirements. Liver: Transaminases \\\u003C 3 x upper limit of normal (ULN) and total bilirubin ≤ 2 mg\u002FdL except for patients with Gilbert's syndrome or hemolysis (as indicated by provider documentation).\n\n   Renal: Creatinine \\\u003C 2.0 mg\u002FdL (adults) and creatinine clearance \\> 40 mL\u002Fmin. Sexually active females of childbearing potential and males with partners of child-bearing potential must agree to use adequate birth control during study treatment.\n5. Voluntary written consent\n\nExclusion Criteria:\n\n1. Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.\n2. Untreated active infection. Controlled or asymptomatic infections requiring continued antimicrobial therapy are permissible.\n3. Active HIV infection, defined as HIV infection with detectable viral load\n4. Active central nervous system malignancy\n5. GVHD requiring systemic therapy including \\> 0.25 mg\u002Fkg prednisone (or equivalent) or other systemic therapy for GVHD (e.g., tacrolimus, sirolimus, ruxolitinib, belumosodil, ibrutinib, axatilimab).\n6. Any other medical or psychological condition that is deemed serious and unsafe for clinical trial participation.\n7. Exposure to prior radiation that is deemed unsafe for clinical trial participation.",{"count":640,"type":22},[26],"Intensive conditioning regimens used in allogeneic hematopoietic cell transplant (HCT) help to eliminate hematologic tumors and reduce the risk of relapse, but are also characterized by high toxicity. Total marrow and lymphoid irradiation (TMLI) is a specialized radiation technique that specifically targets marrow and lymphoid tissue to maximize antitumor efficacy while reducing off target toxicity. Despite these benefits, TMLI is technically challenging and time consuming. The radiation oncology team at Stanford has developed an automated TMLI platform to overcome these challenges. In this phase II trial, automation will be incorporated into a previously validated conditioning regimen of fludarabine\u002Fcyclophosphamide\u002FTMLI HCT with post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis to confirm the feasibility and safety of automation in patients receiving allogeneic HCT for high-risk myeloid malignancies.",[66,440,29],"2026-06-04",{"date":695,"type":44},{"date":751,"type":22},"2026-08",{"date":753,"type":22},"2028-08",{"name":519,"class":108},{"id":756,"slug":757,"hasResults":12,"nctId":758,"briefTitle":759,"officialTitle":759,"acronym":4,"eligibilityCriteria":760,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":4,"enrollmentInfo":761,"targetDuration":4,"studyType":23,"phases":762,"briefSummary":763,"conditions":764,"keywords":4,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":766,"lastUpdatePostDateStruct":767,"startDateStruct":768,"completionDateStruct":770,"leadSponsor":772,"locationsCount":52},"100467305","phase-2-a-phase-ii-study-of-cladribine-and-low-dose-cytarabine-in-combination-with-venetoclax-alternating-with-azacitidine-and-venetoclax-in-patients-with-higher-risk-myeloproliferative-chronic-myelomonocytic-leukemia-or-higher-risk-myelodysplastic-syndromes-with-excess-blasts-100467305","NCT05365035","A Phase II Study of Cladribine and Low Dose Cytarabine in Combination With Venetoclax, Alternating With Azacitidine and Venetoclax, in Patients With Higher-risk Myeloproliferative Chronic Myelomonocytic Leukemia or Higher-risk Myelodysplastic Syndromes With Excess Blasts","Inclusion Criteria:\n\n1. Age \\>\u002F= 18 years.\n2. Diagnosis of MDS or CMML by WHO and:\n\n   * MDS relapsed cohort (Cohort A): MDS with IPSS-R score \\>3.5 and \\>5% blasts with no response after 6 cycles of azacitidine, decitabine, guadecitabine or ASTX727 (decitabine\u002Fcedazuridine) or relapse or progression after any number of cycles\n   * CMML relapsed cohort (Cohort B): CMML 1 or 2 with no response after 6 cycles of azacitidine, decitabine, guadecitabine or ASTX727 (decitabine\u002Fcedazuridine) or relapse or progression after any number of cycles\n   * MDS HMA-naïve cohort (Cohort C): MDS with IPSS-R score \\>3.5 and \\>\u002F= 10% blasts\n   * CMML HMA-naïve cohort (Cohort D): CMML-2; OR CMML-1 with at least one of the following high-risk features: extramedullary disease, splenomegaly of \\>5cm below costal margin or by sonographic volumetric assessment, platelets \\\u003C100x109\u002FL, Hgb level \\\u003C10g\u002FdL, WBC \\>13x109\u002FL, clonal cytogenetic abnormality (other than monosomy Y) or high risk mutations (ASXL1, RUNX1, SETBP1, BRAF, NRAS, KRAS, PTPN11, NF1, CBL).\n   * MDS\u002FMPN relapsed cohort (Cohort E): MDS\u002FMPN-NOS, MDS\u002FMPN with neutrophilia (atypical CML) or MDS\u002FMPN-RS-T with \\>5% blasts with no response after 6 cycles of azacitidine, decitabine, guadecitabine or ASTX727 (decitabine\u002Fcedazuridine) or relapse or progression after any number of cycles\n   * MDS\u002FMPN HMA-naïve cohort (Cohort F): MDS\u002FMPN-NOS or MDS\u002FMPN with neutrophilia (atypical CML) with\n\n     * \\>\u002F=10% blasts or\n     * with \\>5% blasts at least one of the following high-risk features: splenomegaly \\>5cm below costal margin, WBC \\>13x109\u002FL, high risk cytogenetic or molecular features (ASXL1, SETBP1, i(17q), TP53) or\n     * who might not be deemed to benefit from HMA therapy due to proliferative or extramedullary disease.\n3. Eastern Cooperative Oncology Group (ECOG) performance status of \\\u003C\u002F= 2\n4. Creatinine clearance \\> 30 ml\u002Fmin no end\u002Fstage renal disease (using Cockcroft-Gault)\n5. Adequate hepatic function with total bilirubin 2x ULN, AST or ALT 2.5 xULN unless deemed to be due to underlying disease involvement.\n6. Willing to adhere to and comply with all prohibitions and restrictions specified in the protocol.\n7. Patient must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.\n8. English and Non-English speaking patients will be allowed\n\nExclusion Criteria:\n\n1. Uncontrolled infection not adequately responding to appropriate antibiotics\n2. New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \\\u003C50% by echocardiogram or multigated acquisition (MUGA) scan.\n3. History of myocardial infarction within the last 6 months or unstable\u002Funcontrolled angina pectoris or history of severe and\u002For uncontrolled ventricular arrhythmias.\n4. Female patients who are pregnant or lactating.\n5. Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \\[birth control pills\\], contraceptive injections, intrauterine devices \\[IUD\\], double-barrier method \\[spermidical jelly or foam with condoms or diaphragm\\], contraceptive patch, or surgical sterilization) throughout the study.\n6. Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.\n7. Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy.",{"count":92,"type":22},[26],"To learn if the combination of cladribine, cytarabine, venetoclax, and azacitidine can help to control higher-risk myelodysplastic syndrome (MDS) with excess blasts and\u002For higher-risk chronic myelomonocytic leukemia (CMML).",[29,765],"Myeloproliferative Chronic Myelomonocytic Leukemia","2026-06-03",{"date":730,"type":44},{"date":769,"type":44},"2022-09-23",{"date":771,"type":22},"2027-12-31",{"name":773,"class":108},"M.D. Anderson Cancer Center",{"id":775,"slug":776,"hasResults":12,"nctId":777,"briefTitle":778,"officialTitle":778,"acronym":4,"eligibilityCriteria":779,"healthyVolunteers":12,"sex":17,"minAge":780,"maxAge":781,"enrollmentInfo":782,"targetDuration":4,"studyType":23,"phases":783,"briefSummary":784,"conditions":785,"keywords":788,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":793,"lastUpdatePostDateStruct":794,"startDateStruct":795,"completionDateStruct":797,"leadSponsor":799,"locationsCount":52},"100500742","phase-2-alphabeta-t-cell-and-cd19-b-cell-depletion-in-allogeneic-stem-cell-transplantation-in-patients-with-malignant-diseases-100500742","NCT05800210","Alpha\u002FBeta T Cell and CD19+ B Cell Depletion in Allogeneic Stem Cell Transplantation in Patients With Malignant Diseases","Inclusion Criteria:\n\nA. Children, Adolescents, Young adults (ages 6 months to ≤39 years) with the following diseases may be eligible:\n\ni. ALL\n\n1. ALL high risk including one or more of the following: (t(9;22) or 11q23 chromosomal abnormality, primary induction failure (≤15% blasts at time of registration), mixed phenotype acute leukemia (MPAL), persistent MRD (≥0.01% by flow or persistent abnormal karyotype detected by cytogenetics) or hypodiploidy (≤44 chromosomes)) in first remission\n2. ALL in second remission and beyond\n\nii. AML\n\n1. History of AML induction\u002Freinduction Failure (≤15% blasts at time of registration)\n2. AML in CR1 with poor cytogenetics (i.e., 12p, 5a, -7, FLT3 mutation\u002Fduplication, t(9;11) and others)\n3. AML with persistent minimal residual disease (MRD) in CR1(≥0.01% on flow or persistent abnormal karyotype detected by cytogenetics)\n4. AML CR2 or beyond\n5. AML in refractory relapse but ≤15% bone marrow leukemia blasts\n6. Therapy-related AML\n\niii. Juvenile MyeloMonocytic Leukemia (JMML)\n\n1. JMML in CR1 without CBL mutation\n2. JMML with recurrence of disease with or without CBL mutation\n3. JMML CR2 or beyond\n\niv. Chronic Myeloid Leukemia (CML)\n\n1\\. CML in CR with regard to blast crisis\n\nv. High Risk Myelodysplastic syndrome (MDS)\n\nvi. Lymphoma: Hodgkin (HL) or Non-Hodgkin (NHL)\n\n1. HL or NHL with a history of induction failure\n2. HL or NHL in PR1 or PR2\n3. HL or NHL in CR2 or subsequent remission\n\nB. Subjects must not have more than one active malignancy at the time of enrollment (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen \\[as determined by the treating physician and approved by the PI\\] may be included).\n\nC. HLA-matched (5-6\u002F6) sibling donor, matched (8-10\u002F10) unrelated donor available for stem cell donation, haplo-identical related donor (at least one full haplotype must be matched).\n\nD. Karnofsky or Lansky score ≥60% at the time of enrollment. Karnofsky scores must be used for patients \\>16 years of age and Lansky scores for patients ≤16 years of age.\n\nE. Adequate organ function (within 4 weeks of initiation of preparative regimen), defined as:\n\ni. Pulmonary: FEV1, FVC, and corrected DLCO must all be ≥ 60% of predicted by pulmonary function tests (PFTs). For children who are unable to perform for PFTs due to age, the criteria are: no evidence of dyspnea at rest and no need for supplemental oxygen.\n\nii. Renal: Creatinine clearance or radioisotope GFR ≥60 mL\u002Fmin\u002F1.73 m2 or a serum creatinine based on age\u002Fgender\n\niii. Cardiac: Shortening fraction of ≥ 27% by echocardiogram) or ejection fraction of ≥ 50% by echocardiogram or radionuclide scan (MUGA).\n\niv. Hepatic: SGOT (AST) or SGPT (ALT) \\\u003C 5 x upper limit of normal (ULN) for age. Conjugated bilirubin \\\u003C 2.5 mg\u002FdL, unless attributable to Gilbert's Syndrome.\n\nF. Written informed consent obtained from the subject or guardian and the subject agrees to comply with all the study-related procedures.\n\nG. Subjects of childbearing potential (SOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for at least 8 weeks after the last dose of study drug to minimize the risk of pregnancy.\n\nH. Subjects with partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 8 weeks following the last dose of study drug.\n\nExclusion Criteria:\n\nA. Patients with documented uncontrolled infection\n\nB. Patients who have received allogeneic hematopoietic stem cell transplantation within 6 months, unless being done as a boost.\n\nC. Patients with active ≥Grade 2 aGVHD.\n\nD. Demonstrated lack of compliance with medical care.\n\nE. Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least 8 weeks after the last dose of study drug.\n\nF. Females who are known to be pregnant or breastfeeding.\n\nG. History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.\n\nH. Prisoners or subjects who are incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.","6 Months","39 Years",{"count":702,"type":22},[26],"This study will assess the safety, efficacy, and feasibility of ⍺\u002Fβ CD3+ T-cell and CD19+ B-cell depletion in allogeneic stem cell transplantation in patients with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), juvenile myelomonocytic leukemia (JMML), high risk myelodysplastic syndrome (MDS), chronic myeloid leukemia (CML) and lymphoma. Subjects will receive an allogeneic stem cell transplant that has been depleted of ⍺\u002Fβ CD3+ T-cells and CD19+ B-cells using the Miltenyi CliniMACS Prodigy® system.",[159,66,601,29,786,684,787],"Chronic Myeloid Leukemia","Lymphoma, Hodgkin",[789,790,791,792],"hematologic malignancy","GVHD","stem cell transplantation","graft manipulation","2026-06-02",{"date":748,"type":44},{"date":796,"type":44},"2024-05-03",{"date":798,"type":22},"2028-05",{"name":800,"class":108},"University of Florida",{"id":802,"slug":803,"hasResults":12,"nctId":804,"briefTitle":805,"officialTitle":806,"acronym":4,"eligibilityCriteria":807,"healthyVolunteers":12,"sex":17,"minAge":60,"maxAge":808,"enrollmentInfo":809,"targetDuration":4,"studyType":23,"phases":810,"briefSummary":812,"conditions":813,"keywords":817,"overallStatus":40,"whyStopped":4,"lastUpdateSubmitDate":820,"lastUpdatePostDateStruct":821,"startDateStruct":822,"completionDateStruct":824,"leadSponsor":826,"locationsCount":52},"100359777","early-phase-1-hyperbaric-oxygen-therapy-and-allogeneic-peripheral-blood-stem-cell-pbsc-transplant-100359777","NCT03964506","Hyperbaric Oxygen Therapy and Allogeneic Peripheral Blood Stem Cell (PBSC) Transplant","A Pilot Study to Determine the Safety and Efficacy of Incorporating Hyperbaric Oxygen Therapy Into RIC Fludarabine and Melphalan and Allogeneic Hematopoietic Stem\u002FProgenitor Transplantation","Inclusion Criteria:\n\n* Voluntary written informed consent\n* Men or women, age ≥ 18 years of age, with upper limit of 75 years old.\n* Subjects with acute myeloid leukemia (AML) or Myelodysplastic Syndrome (MDS) for cohort 1.\n* Subjects with chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), CML, chronic neutrophilic leukemia (CNL), myelofibrosis, and myelodysplastic\u002Fmyeloproliferative (MDS\u002FMPN) overlap syndrome for cohort 2.\n* Karnofsky performance status (KPS) of ≥ 70%\n* Patients should have New York Heart Association (NYHA) Functional Classification, Class I (ordinary physical activity does not cause undue fatigue, palpitation, dyspnea, or anginal pain) or Class II (ordinary physical activity results in fatigue, palpitation, dyspnea, or anginal pain).\n* Adequate hepatic, renal, cardiac and pulmonary function to be eligible for transplant. Minimum criteria include: Hepatic: ALT, AST \\\u003C 4x IULN and serum total bilirubin ≤ 2.0 mg\u002FdL; Renal: serum creatinine: ≤ 2.0 mg\u002FdL; Left ventricular ejection fraction ≥ 45% measured by 2D-ECHO or MUGA scan; EKG with no clinically significant arrhythmia; FEV1, FVC and DLCO ≥ 50% of predicted value (corrected to serum hemoglobin)\n* Women of child-bearing potential and men with partners of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 30 days following completion of therapy. Should a woman or partner become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician and the investigator immediately.\n* A woman of child-bearing potential is any female (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: Has not undergone a hysterectomy or bilateral oophorectomy; or Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months)\n* Women of child-bearing potential should have a negative urine or serum pregnancy test within 4 weeks of starting preparative regimen\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding\n* Severe chronic obstructive pulmonary disease requiring oxygen supplementation\n* History of spontaneous pneumothorax, prior chest surgery requiring thoracotomy or direct chest irradiation to the lungs\n* Evidence of pneumothorax or significant pulmonary fibrosis on chest imaging within 60 days of transplant.\n* Active malignancy excluding AML, MDS, CMML, aCML CML, CNL, MF and MDS\u002FMPN overlap syndrome.\n* Active ear\u002Fsinus infection. Patients with chronic sinusitis or sinus headaches are excluded unless cleared by ear, nose, and throat specialist.\n* Recent sinus surgery (within the last 5 years).\n* Ear surgery excluding myringotomy or ear tubes\n* Subjects must agree to refrain from active tobacco or e-cigarette use 72 hours prior to transplant until complete transplant recovery. Nicotine replacement therapy is allowed.\n* Claustrophobia\n* History of recurrent seizures within 5 years of study enrollment.\n* Uncontrolled asthma\n* Uncontrolled viral or bacterial infection at the time of study enrollment\n* Active or recent (prior 6 months) invasive fungal infection without interdisciplinary (ID) consult and approval\n* Patients who had intrathecal chemotherapy within 2 weeks of starting preparative regimen or cranial irradiation within 4 weeks of starting preparative regimen","75 Years",{"count":504,"type":22},[811],"EARLY_PHASE1","The purpose of this study is to determine if hyperbaric oxygen therapy is safe in the setting of stem cell transplantation. This study will also determine if hyperbaric oxygen therapy improves engraftment, graft versus host disease, neutrophil count, and incidence and severity of mucositis (inflammation of the mouth or gut) and infection. This study has two cohorts. The first cohort is subjects with acute myeloid leukemia (AML) or Myelodysplastic Syndrome (MDS). The second cohort is subjects with chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), chronic monocytic leukemia, chronic neutrophilic leukemia (CNL), myelofibrosis, and myelodysplastic\u002Fmyeloproliferative (MDS\u002FMPN) overlap syndrome. The first cohort has completed the recruitment so only the second cohort will be recruited.",[66,29,556,814,815,816,67],"Atypical Chronic Myeloid Leukemia","Chronic Monocytic Leukemia","Myelofibrosis",[818,819],"Allogeneic transplant","Hyperbaric Oxygen","2026-06-01",{"date":766,"type":44},{"date":823,"type":44},"2020-07-01",{"date":825,"type":22},"2028-03-01",{"name":827,"class":108},"Omar Aljitawi"]