[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"mds\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:mds":28},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,39,0,25,[9,62,105,138,212,241,272,292,314,338,359,387,424,451,480,513,535,556,583,605,627,658,684,710,739],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":54,"completionDateStruct":56,"leadSponsor":58,"locationsCount":61},"100053848","phase-1-a-phase-13-study-of-t-cell-receptor-engineered-donor-t-cells-in-subjects-undergoing-allogeneic-peripheral-blood-stem-cell-transplantation-alloha-2-100053848",false,"NCT05473910","A Phase 1\u002F3 Study of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation (ALLOHA-2)","A Phase 1\u002F3 Study Evaluating the Efficacy and Safety of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation (ALLOHA-2)","Inclusion Criteria:\n\n* Male or female aged ≥ 18 years at the time of signing the informed consent.\n* Eastern Cooperative Oncology Group (ECOG)-PS ≤ 2 at the time of the screening visit.\n* Contraceptive use by male and female participants must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Male Participants:\n* A male participant must agree to use a highly effective contraceptive as detailed in Appendix 4 of this protocol during the intervention period and for at least 12 months after the last dose of study intervention and refrain from donating sperm during this period.\n* Female Participants:\n* A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:\n* Not a woman of childbearing potential (WOCBP) OR\n* A WOCBP who agrees to follow the contraceptive guidance during the intervention period and for at least 12 months after the last dose of study intervention.\n* Preparing to undergo allogeneic HCT for either of the following:\n* AML, MDS, ALL\n* Participants in the treatment arms must express HLA-A\\*0201. Participants in the control arm may express any HLA type.\n* Having the HA1+\u002F- or HA-1+\u002F+ (HA-1 positive) genotype to be eligible for TSC-100 treatment.\n* Having the HA2+\u002F- HA-2+\u002F+ (HA-2 positive) genotype to be eligible for TSC-101 treatment.\n* Having a haploidentical donor, MMUD, or MUD for HCT who is adequately HLA-matched by institutional standards and meets the donor inclusion criteria.\n\nConsidered to be clinically indicated for haploidentical donor, MMUD, or MUD transplantation at the discretion of the treating investigator.\n\nConsidered to be clinically indicated for RIC at the discretion of the treating investigator.\n\nConsidered to be clinically indicated for peripheral blood stem cell transplantation at the discretion of the treating investigator.\n\nOrgan function parameters for transplant eligibility are met per institutional standards.\n\nCapable of giving signed informed consent - which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nParticipants must provide consent for mandatory study procedures including bone marrow biopsy and blood sampling for research analyses in the ICF.\n\nParticipants must agree to participate in long-term follow-up for up to 15 years post initial product treatment if they are enrolled in the study and receive the investigational Tcell infusion.\n\nDonor Inclusion Criteria :\n\nMale or female aged ≥ 18 years at the time of signing the informed consent. Able to undergo peripheral blood stem cell (PBSC) collection and up to 2 rounds of leukapheresis (for TSC-100 or TSC101 manufacturing for treatment arms only, and f for stem cell collection for both treatment arms and the control arm).\n\nDonors matched to TSC-100 participants should be HA-1-\u002F- (negative) and\u002For negative for all HLA-A\\*02 alleles Donors matched to TSC-101 participants should be negative for all HLA-A\\*02 alleles Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nExclusion Criteria:\n\nMedical or psychological conditions that would make the participant an unsuitable candidate for cell therapy including another concurrent uncontrolled malignancy or active CNS disease.\n\nThe presence of organ toxicities will not necessarily exclude participants from enrolling on the protocol at the discretion of the PI; however, a delay in the infusion of HA1\u002FHA2 TCRT cells may be required at the discretion of the treating investigator Participants with levels of donor-specific HLA antibodies that are considered by the treating investigator to be high enough to warrant desensitization protocols and who have no alternate donors.\n\nParticipants who meet inclusion criteria for TSC-101 but who are also positive for HLAA\\*02:07.\n\nParticipants with evidence of clinically significant infection or uncontrolled viral r reactivation of cytomegalovirus (CMV), Epstein-Barr virus (EBV), Adenovirus, BK virus (BKV), or human herpesvirus 6 (HHV-6).\n\nParticipants with active cardiac disease, defined as:\n\nUncontrolled or symptomatic angina within the past 3 months. History of clinically significant arrhythmias (such as ventricular tachycardia, ventricular fibrillation, torsades de pointes). Atrial fibrillation with controlled ventricular response on treatment is not an exclusion.\n\nMyocardial infarction \\\u003C 3 months from study entry. Uncontrolled or symptomatic congestive heart failure. Prior allogeneic HCT. Participants who have a history of hypersensitivity to murine proteins. Enrollment on a concomitant trial with a novel investigational agent. Use of anti-thymocyte globulin, alemtuzumab, or other in vivo T-cell depleting agents from Day -14 through end of study.\n\nDonor Exclusion Criteria :\n\nDonors for TSC-100 positive for any HLA-A\\*02 allele would be excluded unless they are HA-1 negative. If donors with any HLA-A\\*02 allele are considered for patients eligible for TSC-100, the donor would undergo HA-1 testing to ensure that the donor is HA-1 negative (40% probability).\n\nDonors for TSC-101 positive for any HLA-A\\*02 allele are excluded regardless of HA- 2 status.\n\nDonors who test positive for any of the following: HIV-1, HIV-2, human T-lymphotropic virus (HTLV)-1, HTLV-2 seropositive or with active hepatitis B or hepatitis C virus infection, syphilis, West Nile virus through central lab testing. Donors who screen positive for risk of CreutzfeldtJakob disease or Zika virus infection using donor history questionnaires will also be excluded. Donors with evidence of past CMV or EBV infections will be allowed.\n\nRelated donor residing outside of the United States of America (USA). If the donor screening, testing and leukapheresis can be performed at the same site where the participant is being treated, the donor is considered eligible.","ALL","18 Years",{"count":20,"type":21},310,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This is a multi-center, non-randomized, concurrent controlled, multi-arm, Phase 1 interventional, open-label, biologic assignment-based umbrella study evaluating the feasibility, safety and preliminary efficacy of an escalating dose regimen of up to 2 doses of TSC-100 and TSC-101 in patients with AML, MDS, or ALL following HCT from a haploidentical donor, MMUD, or MUD",[27,28],"AML","MDS",[30,31,27,28,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,17,48],"HA-2","TSC-101","Adoptive Cell Therapy","T-cell receptor","T lymphocyte","TCR-engineered T cells","bone marrow transplant","haploidentical","allogeneic stem cell transplant","BMT","RIC","HSCT","Hematopoietic stem cell transplantation","ALLOHA-2","Mismatched unrelated donors MMUD","ALLOHA","HA-1","TSC-100","Reduced Intensity Conditioning","RECRUITING","2026-07-10",{"date":52,"type":53},"2026-07-13","ACTUAL",{"date":55,"type":53},"2022-11-01",{"date":57,"type":21},"2028-06",{"name":59,"class":60},"TScan Therapeutics, Inc.","INDUSTRY",21,{"id":63,"slug":64,"hasResults":12,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":68,"eligibilityCriteria":69,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":70,"targetDuration":72,"studyType":73,"phases":4,"briefSummary":74,"conditions":75,"keywords":93,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":104},"100053513","global-registry-and-natural-history-study-for-mitochondrial-disorders-100053513","NCT05554835","Global Registry and Natural History Study for Mitochondrial Disorders","Global Mitochondrial Registry to Define Natural History and Outcome Measures to Achieve Definite Trial Readiness for Mitochondrial Disorders","GENOMIT","Inclusion Criteria:\n\n* suspected or confirmed mitochondrial disease\n* willingness to participate\n\nExclusion Criteria:\n\n* unwillingness to participate",{"count":71,"type":21},6000,"30 Years","OBSERVATIONAL","The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.",[76,77,78,79,80,81,82,83,84,85,86,87,88,28,89,90,91,92],"Mitochondrial Diseases","Kearns-Sayre Syndrome","MIDD","SANDO","SCAE","NARP Syndrome","MELAS Syndrome","MERRF Syndrome","Coenzyme Q10 Deficiency","LHON","MNGIE","MIRAS","Barth Syndrome","Mitochondrial Myopathies","Leigh Syndrome","Pearson Syndrome","CPEO",[94],"Patient Registry","2026-07-09",{"date":52,"type":53},{"date":98,"type":53},"2009-02-01",{"date":100,"type":21},"2040-12",{"name":102,"class":103},"LMU Klinikum","OTHER",33,{"id":106,"slug":107,"hasResults":12,"nctId":108,"briefTitle":109,"officialTitle":110,"acronym":4,"eligibilityCriteria":111,"healthyVolunteers":12,"sex":17,"minAge":112,"maxAge":113,"enrollmentInfo":114,"targetDuration":4,"studyType":22,"phases":116,"briefSummary":118,"conditions":119,"keywords":122,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":130,"completionDateStruct":132,"leadSponsor":134,"locationsCount":137},"100198569","phase-2-allogeneic-hematopoietic-stem-cell-transplant-for-gata2-mutations-100198569","NCT01861106","Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations","Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome","* ELIGIBILITY CRITERIA:\n\nINCLUSION CRITERIA- Recipient\n\n1. Patient age of 6-70 years.\n2. Mutation in the GATA2 gene, or evidence of loss of expression of one allele of GATA2, by cDNA analysis performed by a CLIA certified laboratory, or the clinical syndrome of MonoMAC.\n3. Clinical history of at least one serious or disfiguring infection and\u002For GATA2 bone marrow immunodeficiency disorder with lose of one or more immune populations in the bone marrow including monocytes, Natural Killer (NK) cells, and B-lymphocytes, with or without additional cytopenias involving the red blood cell, neutrophil, or platelet compartment.\n4. Availability of a 10\u002F10 or 9\u002F10 or 8\u002F10 HLA-matched related or unrelated donor, or a haploidentical related donor.\n5. Patients may have evidence of MDS with one or more peripheral blood cytopenias and greater than 5% blasts but must have less than 10% blasts in the bone marrow in the absence of filgrastim in order to proceed directly to transplant. The majority of patients with MDS will have less than 5% blasts.\n6. Disease status: Patients are to be referred in remission for evaluation. Should a patient have progressive disease with \\>10% blasts on screening\u002Fbaseline bone marrow biopsy, the patient may receive standard treatment under the current study prior to proceeding with transplant. Once the patient has \\\u003C10% blasts, they may proceed to transplant. The patient may also be referred back to their primary hematologist or oncologist for treatment. If this course of action is not in the best interest of the patient according to the clinical judgment of the PI\u002FLAI, then the patient may receive standard treatment for the malignant disease or hematological disorder under the current study. If under either of these settings, it becomes apparent that the participant will not be able to proceed to transplant, then he\u002Fshe must come off study. Recipient-Subjects receiving a standard therapy will be told about the therapy, associated risks, benefits and alternatives of the proposed therapy, and availability of receiving the same treatment elsewhere, outside of a research protocol.\n7. Left ventricular ejection fraction \\> 40%, preferably by 2-D echocardiogram obtained within 90 days prior to initiation of conditioning therapy.\n8. Creatinine: Adult patients: \\\u003C= 2.0 mg\u002Fdl and creatinine clearance \\>= 30 ml\u002Fmin; Pediatric patients (\\\u003C18 years old): creatinine \\\u003C1.5 mg\u002FdL and a creatinine clearance, using the Schwartz Formula, \\> 30 mL\u002Fmin\u002F1.73m\\^2.\n9. Serum conjugated bilirubin \\\u003C 2.5 mg\u002Fdl; serum ALT and AST \\\u003C= 5 times upper limit of normal.\n10. Pulmonary function tests: FEV1 and DLCO \\>30% Note: For children who are unable to cooperate for PFTs, the criterion is: No evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen therapy\n11. Ability of patient or Legally Authorized Representative (LAR) (if the patient is deemed by the treating physician to be cognitively impaired or questionably impaired in such a way that the ability of the patient to give informed consent is questionable) to understand and the willingness to sign a written informed consent document indicating that they are aware of the investigational nature of this study or written informed consent obtained from parent or legal guardian if subject is a minor.\n12. As therapeutic agents used in this trial may be harmful to a fetus, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least one-year post-allo HSCT. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.\n13. All transplant patients remain in the NIH hospital or, if discharged, stay close to the NIH for a minimum of 100 days after transplant or longer, if there are complications. An adult caregiver must be with the patient at all times from discharge to day 100.\n\nEXCLUSION CRITERIA- Recipient\n\n1. Patients who are receiving any other investigational agents with the exception of virus- specific cytotoxic T-cells for the treatment of viral infection\u002Freactivation prior to allo HSCT\n2. HIV-positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.\n3. History of allergic reactions attributed to compounds of similar chemical or biological composition to agents (steroids, cyclophosphamide, busulfan) used in the study\n4. Chronic active hepatitis B. Patient may be hepatitis B core antibody positive. For patients with a concomitant positive hepatitis B surface antigen, patients will require a hepatology consultation. The risk-benefit profile of transplant and hepatitis B will be discussed with the patient, and eligibility determined by the PI or Lead Associate Investigator.\n5. History of psychiatric disorder which may compromise compliance with transplant protocol, or which does not allow for appropriate informed consent.\n6. Active infection refractory to antimicrobial therapy.\n7. Active CNS involvement by malignancy (patients with known positive CSF cytology or parenchymal lesions visible by prior CT or MRI).\n8. Pregnant or lactating.\n9. The effects on breast-milk are unknown and may be harmful to the infant; therefore, women should not breast feed during the interval from study entry to one year post-transplant.\n10. Presence of active malignancy in another organ system other than the hematopoietic, except when driven by viruses in which case the immune reconstitution after transplant may control the malignancy. This includes solid tumors not in remission.\n11. No available 10\u002F10 or 9\u002F10 or 8\u002F10 HLA-matched related or unrelated donor, or haploidentical related donor.","6 Years","70 Years",{"count":115,"type":21},144,[117],"PHASE2","Background:\n\n\\- GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition. A stem cell transplant will give stem cells from a matching donor (related or unrelated) to a recipient. It will allow the donor stem cells to produce healthy bone marrow and blood cells that will attack the recipient s cancer cells.\n\nObjectives:\n\n\\- To see if stem cell transplants are successful at treating GATA2 mutations and related conditions.\n\nEligibility:\n\n\\- Recipients who are between 6 and 70 years of age and have GATA2 deficiency.\n\nDesign:\n\n* All participants will be screened with a physical exam and medical history. Blood samples will be collected. Recipients will have imaging studies and other tests.\n* Recipients will have chemotherapy or radiation to prepare for the transplant. On the day of the transplant, they will receive the donated stem cells.\n* Recipients will stay in the hospital until their condition is stable after transplant.\n* Frequent blood tests and scans will be required for the first 6 months after the transplant, followed by less frequent visits over time.",[120,121,28],"GATA2","Immunodeficiency",[123,124,121,125,126],"Allogeneic Donors","Peripheral Blood Stem Cell","Myelodysplasia","Haploidentical","2026-06-23",{"date":129,"type":53},"2026-06-24",{"date":131,"type":53},"2013-07-24",{"date":133,"type":21},"2028-12-31",{"name":135,"class":136},"National Cancer Institute (NCI)","NIH",1,{"id":139,"slug":140,"hasResults":12,"nctId":141,"briefTitle":142,"officialTitle":142,"acronym":143,"eligibilityCriteria":144,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":145,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":147,"conditions":148,"keywords":193,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":203,"lastUpdatePostDateStruct":204,"startDateStruct":206,"completionDateStruct":208,"leadSponsor":210,"locationsCount":137},"100289631","familial-investigations-of-childhood-cancer-predisposition-100289631","NCT03050268","Familial Investigations of Childhood Cancer Predisposition","SJFAMILY","NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown below, you may enroll regardless of the results of your clinical genetic testing.\n\nDEFINITION OF FAMILIAR CANCER FOR THIS PROTOCOL:\n\nIn this protocol, the definition of \"Familial Cancer\" is met if any of the following is present:\n\n* An individual with a history of cancer diagnosed under 26 years of age who has at least one first, second or third degree relative with a history of cancer diagnosed under 51 years of age; OR\n* An individual who has been diagnosed with more than one cancer, at least one of which was diagnosed under 26 years of age; OR\n* An individual with a clinical or molecular diagnosis of a known cancer predisposition syndrome; OR\n* An individual with a congenital cancer diagnosed before 6 months of age; OR\n* An individual with a rare pediatric cancer or tumor diagnosed before 26 years of age\n\nº Excluding human papilloma virus-associated cervical cancer and non-melanoma skin cancer occurring in adults.\n\nINCLUSION CRITERIA:\n\n* An individual who meets this protocol's definition of \"Familial Cancer,\" as above.\n* Biologic relatives of an individual meeting this protocol's definition of \"Familial Cancer,\" who are either affected or unaffected by cancer.\n\nEXCLUSION CRITERIA:\n\n* An inability or unwillingness of the research participant or his\u002Fher legally authorized representative (LAR) to provide written informed consent.\n* The participant has received allogeneic bone marrow transplantation and has NO pre-transplant germline (cancer-unaffected) DNA available AND is unwilling to provide a skin sample.",{"count":146,"type":21},1500,"NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown in the Eligibility Section, you may enroll regardless of the results of your clinical genetic testing.\n\nWhile it is well recognized that hereditary factors contribute to the development of a subset of human cancers, the cause for many cancers remains unknown. The application of next generation sequencing (NGS) technologies has expanded knowledge in the field of hereditary cancer predisposition. Currently, more than 100 cancer predisposing genes have been identified, and it is now estimated that approximately 10% of all cancer patients have an underlying genetic predisposition.\n\nThe purpose of this protocol is to identify novel cancer predisposing genes and\u002For genetic variants. For this study, the investigators will establish a Data Registry linked to a Repository of biological samples. Health information, blood samples and occasionally leftover tumor samples will be collected from individuals with familial cancer. The investigators will use NGS approaches to find changes in genes that may be important in the development of familial cancer. The information gained from this study may provide new and better ways to diagnose and care for people with hereditary cancer.\n\nPRIMARY OBJECTIVE:\n\n* Establish a registry of families with clustering of cancer in which clinical data are linked to a repository of cryopreserved blood cells, germline DNA, and tumor tissues from the proband and other family members.\n\nSECONDARY OBJECTIVE:\n\n* Identify novel cancer predisposing genes and\u002For genetic variants in families with clustering of cancer for which the underlying genetic basis is unknown.",[149,150,151,27,152,153,154,155,156,157,158,159,160,161,162,163,164,165,166,167,168,169,170,171,172,28,173,174,175,176,177,178,179,180,181,182,183,184,185,186,187,188,189,190,191,192],"Acute Leukemia","Adenomatous Polyposis","Adrenocortical Carcinoma","BAP1 Tumor Predisposition Syndrome","Carney Complex","Choroid Plexus Carcinoma","Constitutional Mismatch Repair Deficiency Syndrome","Diamond-Blackfan Anemia","DICER1 Syndrome","Dyskeratosis Congenita","Emberger Syndrome","Familial Acute Myeloid Leukemia","Familial Adenomatous Polyposis","Fanconi Anemia","Familial Cancer","Familial Wilms Tumor","Familial Neuroblastoma","GIST","Hereditary Breast and Ovarian Cancer","Hereditary Paraganglioma-Pheochromocytoma Syndrome","Hodgkin Lymphoma","Juvenile Polyposis","Li-Fraumeni Syndrome","Lynch Syndrome","Melanoma Syndrome","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Neuroblastoma","Neurofibromatosis Type 1","Neurofibromatosis Type II","Nevoid Basal Cell Carcinoma Syndrome","Non Hodgkin Lymphoma","Noonan Syndrome and Other Rasopathy","Overgrowth Syndromes","Pancreatic Cancer","Peutz-Jeghers Syndrome","Pheochromocytoma\u002FParaganglioma","PTEN Hamartoma Tumor Syndrome","Retinoblastoma","Rhabdoid Tumor Predisposition Syndrome","Rhabdomyosarcoma","Rothmund-Thomson Syndrome","Tuberous Sclerosis","Von Hippel-Lindau Disease",[194,195,196,197,198,199,200,201,202],"Familial cancer","Genetic predisposition","Heritable disease","Cancer risk","Genome analysis","Genetic modifiers","Next generation sequencing (NGS)","Genetic counseling","DNA","2026-06-15",{"date":205,"type":53},"2026-06-17",{"date":207,"type":53},"2017-04-06",{"date":209,"type":21},"2037-03-31",{"name":211,"class":103},"St. Jude Children's Research Hospital",{"id":213,"slug":214,"hasResults":12,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":4,"eligibilityCriteria":218,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":219,"enrollmentInfo":220,"targetDuration":4,"studyType":22,"phases":222,"briefSummary":223,"conditions":224,"keywords":4,"overallStatus":231,"whyStopped":4,"lastUpdateSubmitDate":232,"lastUpdatePostDateStruct":233,"startDateStruct":235,"completionDateStruct":237,"leadSponsor":239,"locationsCount":137},"100642605","phase-2-shr2554aza--overlapped-modified-bucy-for-high-riskrelapsed-leukemiamds-100642605","NCT07575412","SHR2554\u002FAZA + Overlapped Modified BUCY for High-risk\u002FRelapsed Leukemia\u002FMDS","A Prospective, Multicenter, Open-label, Randomized Controlled Trial of SHR2554 Plus Azacitidine in Overlapped Sequential Combination With Modified BUCY Conditioning Regimen in Patients With High-risk or Relapsed\u002FRefractory Acute Leukemia and Myelodysplastic Neoplasms Secondary IDs","Inclusion Criteria:\n\n1. Age 15-60 years, of either sex.\n2. Diagnosis of AML or ALL according to the WHO 2022 criteria, with an indication for allogeneic hematopoietic stem cell transplantation:\n\n   AML with high-risk genetics at diagnosis (risk stratification per ELN 2022) or relapsed\u002Frefractory AML (meeting any of the following: refractory-failure to achieve complete remission (CR) after two cycles of induction chemotherapy; relapse-reappearance of blasts in peripheral blood or bone marrow (≥5%) after first CR, or extramedullary relapse (EMR)).\n\n   High-risk B-ALL at diagnosis (risk stratification per ELN 2022) or pre-transplant MRD-positive B-ALL.\n\n   Confirmed T-ALL. History of central nervous system leukemia (CNSL) or pathologically confirmed extramedullary disease (EMD) during AML or ALL.\n\n   Myelodysplastic neoplasms (MDS): IPSS score intermediate-2 or high; IPSS-R score high or very high; IPSS-M score high or very high.\n3. Availability of an appropriate HLA-matched donor.4: ECOG performance status 0-2.5: Adequate major organ function, defined as: Left ventricular ejection fraction ≥50%. Pulmonary function: DLCO ≥50% of predicted value. Liver function: ALT\u002FAST ≤3×ULN, total bilirubin ≤2×ULN. Renal function: estimated creatinine clearance (CrCl) ≥60 mL\u002Fmin.6: Ability to understand the study and voluntary signed informed consent.\n\nExclusion Criteria:\n\n1: Acute promyelocytic leukemia (APL);2: Active central nervous system leukemia;3: Prior allogeneic hematopoietic stem cell transplantation;4: Prior treatment with any EZH2 inhibitor;5: Uncontrolled active infection as assessed by the investigator;6: Myocardial infarction or unstable angina within the previous 6 months;7: Known hypersensitivity to SHR2554, azacitidine, or any excipient of the mBuCy regimen;8: Pregnant or breastfeeding women;9: Any other medical condition that, in the investigator's judgment, would preclude study enrollment.","65 Years",{"count":221,"type":21},180,[117],"This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-65 years with high risk or relapsed\u002Frefractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the mBuCy conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint is 1-year event-free survival (EFS). Secondary endpoints include 2-year overall survival, 2-year cumulative incidence of relapse, transplant-related mortality, incidence of acute\u002Fchronic GVHD, and safety profiles.",[225,226,28,227,228,229,230],"Acute Myeloid Leukemia","Acute Lymphoblastic Leukemia","Transplantation, Stem Cell","Conditioning Therapy","SHR2554","Azacytidine","NOT_YET_RECRUITING","2026-06-08",{"date":234,"type":53},"2026-06-10",{"date":236,"type":21},"2026-06",{"date":238,"type":21},"2030-06",{"name":240,"class":103},"The First Affiliated Hospital of Soochow University",{"id":242,"slug":243,"hasResults":12,"nctId":244,"briefTitle":245,"officialTitle":246,"acronym":28,"eligibilityCriteria":247,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":248,"targetDuration":4,"studyType":22,"phases":249,"briefSummary":250,"conditions":251,"keywords":257,"overallStatus":231,"whyStopped":4,"lastUpdateSubmitDate":232,"lastUpdatePostDateStruct":265,"startDateStruct":266,"completionDateStruct":268,"leadSponsor":270,"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":21},[24,117],"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.",[252,28,253,254,255,256],"Myelodysplastic Syndromes","Myelodysplastic Syndrome, Unclassifiable","Hematologic Diseases","Bone Marrow Disease","Myeloproliferative Neoplasm",[258,259,260,261,262,263,264],"Pacritinib","Azacitidine","IPSS-M","JAK\u002FSTAT","JAK\u002FIRAK1","NF-κβ","Myelodysplastic Syndrome",{"date":234,"type":53},{"date":267,"type":21},"2026-07",{"date":269,"type":21},"2027-01",{"name":271,"class":103},"Thomas Jefferson University",{"id":273,"slug":274,"hasResults":12,"nctId":275,"briefTitle":276,"officialTitle":277,"acronym":4,"eligibilityCriteria":278,"healthyVolunteers":12,"sex":17,"minAge":279,"maxAge":280,"enrollmentInfo":281,"targetDuration":4,"studyType":22,"phases":283,"briefSummary":284,"conditions":285,"keywords":4,"overallStatus":231,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":290,"leadSponsor":291,"locationsCount":137},"100636198","phase-2-shr2554aza--overlapped-tbf-for-high-riskrelapsed-leukemiamds-100636198","NCT07562568","SHR2554\u002FAZA + Overlapped TBF for High-risk\u002FRelapsed Leukemia\u002FMDS","A Prospective, Multicenter, Open-label, Randomized Controlled Trial of SHR2554 Plus Azacitidine in Overlapped Sequential Combination With TBF Conditioning Regimen in Patients With High-risk or Relapsed\u002FRefractory Acute Leukemia and Myelodysplastic Neoplasms","Inclusion Criteria:\n\n1. Age 15-60 years, of either sex.\n2. Diagnosis of AML or ALL according to the WHO 2022 criteria, with an indication for allogeneic hematopoietic stem cell transplantation: AML with high-risk genetics at diagnosis (risk stratification per ELN 2022) or relapsed\u002Frefractory AML (meeting any of the following: refractory-failure to achieve complete remission (CR) after two cycles of induction chemotherapy; relapse-reappearance of blasts in peripheral blood or bone marrow (≥5%) after first CR, or extramedullary relapse (EMR)). High-risk B-ALL at diagnosis (risk stratification per ELN 2022) or pre-transplant MRD-positive B-ALL. Confirmed T-ALL. History of central nervous system leukemia (CNSL) or pathologically confirmed extramedullary disease (EMD) during AML or ALL. Myelodysplastic neoplasms (MDS): IPSS score intermediate-2 or high; IPSS-R score high or very high; IPSS-M score high or very high.\n3. Availability of an appropriate HLA-matched donor.\n4. ECOG performance status 0-2.\n5. Adequate major organ function, defined as: Left ventricular ejection fraction ≥50%. Pulmonary function: DLCO ≥50% of predicted value. Liver function: ALT\u002FAST ≤3×ULN, total bilirubin ≤2×ULN. Renal function: estimated creatinine clearance (CrCl) ≥60 mL\u002Fmin.\n6. Ability to understand the study and voluntary signed informed consent.\n\nExclusion Criteria:\n\n1. Acute promyelocytic leukemia (APL);\n2. Active central nervous system leukemia;\n3. Prior allogeneic hematopoietic stem cell transplantation;\n4. Prior treatment with any EZH2 inhibitor;\n5. Uncontrolled active infection as assessed by the investigator;\n6. Myocardial infarction or unstable angina within the previous 6 months;\n7. Known hypersensitivity to Zeprumetostat, azacitidine, or any excipient of the mBuCy regimen;\n8. Pregnant or breastfeeding women;\n9. Any other medical condition that, in the investigator's judgment, would preclude study enrollment.","15 Years","60 Years",{"count":282,"type":21},160,[117],"This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-60 years with high-risk or relapsed\u002Frefractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the TBF conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint was the 2-year cumulative incidence of relapse (CIR) after allo-HSCT. Secondary endpoints included 2-year overall survival (OS), 2-year disease-free survival (DFS), transplant-related mortality (TRM), the incidence of acute and chronic graft-versus-host disease (GVHD), and safety profiles.",[225,226,28,227,228,229,230],"2026-04-26",{"date":288,"type":53},"2026-05-01",{"date":236,"type":21},{"date":238,"type":21},{"name":240,"class":103},{"id":293,"slug":294,"hasResults":12,"nctId":295,"briefTitle":296,"officialTitle":296,"acronym":4,"eligibilityCriteria":297,"healthyVolunteers":12,"sex":17,"minAge":298,"maxAge":299,"enrollmentInfo":300,"targetDuration":4,"studyType":22,"phases":302,"briefSummary":303,"conditions":304,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":305,"lastUpdatePostDateStruct":306,"startDateStruct":308,"completionDateStruct":310,"leadSponsor":312,"locationsCount":137},"100630889","phase-2-mt2025-35-allogeneic-hematopoietic-stem-cell-transplantation-using-reduced-intensity-conditioning-treosulfan-and-fludarabine-with-post-transplant-cytoxan-ptcy-for-the-treatment-of-hematological-diseases-100630889","NCT07493538","MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases","Inclusion Criteria:\n\n* Patients 2-75 years of age\n* ≤7 5 years of age: Karnofsky score ≥ 70% (≥ 16 years) or Lansky play score ≥ 50 (\\\u003C 16 years) with appropriate organ criteria as below (in other inclusion criteria)\n* 5\u002F6 or 6\u002F6 related donor, OR a 5-8\u002F8 HLA-A, B, C, DRB1 allele match unrelated donor, OR a haplotype (at least 5\u002F10) related donor\n* adequate liver (no decompensated liver failure, Child Pugh A, AST\u002FALT \\\u003C5X ULN) and renal function (creatinine \\\u003C2.0)\n* absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%\n* DLCO FEV1, FVC ≥ 40% predicted, and absence of O2 requirement\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding\n* Evidence of untreated\u002Funcontrolled HIV infection\n* Untreated active serious infection\n* Active CNS malignancy\n* CML in blast crisis not in a complete remission by abnormal blast count.\n* Less than 3 months since prior myeloablative transplant","2 Years","75 Years",{"count":301,"type":21},132,[117],"This is a Phase II study following subjects proceeding with Treosulfan (36g\u002Fm2) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion, with post-transplant cyclophosphamide (PTCy) at 40mg\u002Fkg, tacrolimus and MMF for GVHD prophylaxis.",[27,28,149,225,252],"2026-04-13",{"date":307,"type":53},"2026-04-16",{"date":309,"type":53},"2026-04-10",{"date":311,"type":21},"2035-03",{"name":313,"class":103},"Masonic Cancer Center, University of Minnesota",{"id":315,"slug":316,"hasResults":12,"nctId":317,"briefTitle":318,"officialTitle":319,"acronym":4,"eligibilityCriteria":320,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":299,"enrollmentInfo":321,"targetDuration":4,"studyType":22,"phases":323,"briefSummary":324,"conditions":325,"keywords":326,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":329,"lastUpdatePostDateStruct":330,"startDateStruct":332,"completionDateStruct":334,"leadSponsor":336,"locationsCount":137},"100376867","phase-2-bmt-06-study-of-intensity-modulated-total-marrow-irradiation-im-tmi-100376867","NCT04187105","BMT-06: Study of Intensity Modulated Total Marrow Irradiation (IM-TMI)","BMT-06: Phase II Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine\u002FCyclophosphamide and Post-Transplant Cyclophosphamide Conditioning for Partially HLA Mismatched Allogeneic Transplantation in Patients With Acute Leukemia and Myelodysplastic Syndrome (MDS)","Inclusion Criteria:\n\n1. Patient age 18-75 years\n2. Related donor who is, at minimum, Human Leukocyte Antigen (HLA) haploidentical or mismatched unrelated donor.\n\n   * Haploidentical: The donor and recipient must be identical in at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1. A minimum match of 4\u002F8 if using HLA-A,-B,-DRB1,-Cw, or 5\u002F10 if using HLA-A,-B,-Cw ,-DRB1, and -DQB1, will be considered evidence that the donor and recipient share one HLA haplotype.\n   * Unrelated donors: unrelated donors who are mismatched in one or more of the following loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1,HLA-DQB1- can be included with a maximum of 4\u002F8 or 5\u002F10 mismatches.\n3. Eligible diagnoses are listed below. Patient must have one of the following:\n\n   1. Relapsed or refractory acute leukemia (including AML or ALL in CR2 and primary refractory leukemia).\n   2. Poor-risk AML in first remission:\n\n      * AML arising from MDS or a myeloproliferative disorder, or secondary AML\n      * Poor risk molecular features including but not limited to presence of FLT3 internal tandem duplication mutation.\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\n   3. Poor risk ALL in first remission:\n\n      * Poor risk cytogenetics: Philadelphia Chromosome, t(4;11), KMT2A translocation, t(8;14), complex karyotype (⩾ 5 chromosomal abnormalities) and low hypodiploidy (30-39 chromosomes)\u002Fnear triploidy (60-78 chromosomes)\n      * Philadelphia-like ALL\n      * Presentation WBC \\>30 × 109 for B-ALL or \\>100 109 for T-ALL\n      * Age\\>35\n      * Poor MRD clearance, defined as levels \\>1 × 10-3 after induction and levels \\>5 × 10-4 after early consolidation by flow cytometry\n   4. Myelodysplastic syndromes (MDS) with at least one of the following poor-risk features:\n\n      * i. Poor-risk cytogenetics (including but not limited to 7\u002F7q minus or complex cytogenetics)\n      * ii. IPSS score of INT-2 or greater\n      * iii. Treatment-related or Secondary MDS\n      * iv. MDS diagnosed before age 21 years\n      * v. Progression on or lack of response to standard DNA-methyltransferase inhibitor therapy\n      * vi. Life-threatening cytopenias, including those generally requiring greater than weekly transfusions\n      * vii. Poor risk molecular features including but not limited to the presence of BCOR, ASXL1, p53 or RUNX1 mutations\n   5. Mixed lineage and biphenotypic leukemia\n4. Adequate end-organ function as measured by:\n\n   * a. Left ventricular ejection fraction ≥ 40%\n   * b. Bilirubin ≤ 2.0 mg\u002FdL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST \\\u003C 5 x ULN\n   * c. FEV1 and FVC \\> 50% of predicted\n\nExclusion Criteria:\n\n1. Presence of significant co morbidity as shown by:\n\n   * a. Left ventricular ejection fraction \\\u003C 40%\n   * b. Bilirubin \\> 2.0 mg\u002FdL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST \\> 5 x ULN\n   * c. FEV1 and FVC \\\u003C 50% of predicted or DLCO \\\u003C50% of predicted once corrected for anemia\n   * d. Karnofsky score \\\u003C70\n   * e. History of cirrhosis\n2. Patients unable to sign informed consent\n3. Patient who have previously received radiation to \\>20% of bone marrow containing areas (assessed by radiation oncology physician)",{"count":322,"type":21},27,[117],"This study is being done to see if the addition of a targeted form of radiation to standard conditioning regimen will increase the amount of cancer cells that are killed off in the bone marrow and reduce the chances that your disease may return. This description is called Intensity Modulated Total Marrow Irradiation (IM-TMI).",[149,28],[327,328],"Stem Cell Transplant","Half-matched (haploidentical) stem cell transplant","2026-03-31",{"date":331,"type":53},"2026-04-06",{"date":333,"type":53},"2020-01-27",{"date":335,"type":21},"2026-12",{"name":337,"class":103},"University of Illinois at Chicago",{"id":339,"slug":340,"hasResults":12,"nctId":341,"briefTitle":342,"officialTitle":343,"acronym":4,"eligibilityCriteria":344,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":345,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":346,"conditions":347,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":349,"lastUpdatePostDateStruct":350,"startDateStruct":352,"completionDateStruct":354,"leadSponsor":356,"locationsCount":358},"100599176","replacing-bone-marrow-diagnostics-with-peripheral-blood-analysis-in-cytopenia-patients-100599176","NCT07081087","Replacing Bone Marrow Diagnostics With Peripheral Blood Analysis in Cytopenia Patients","Development of a Peripheral Blood Assay to Replace BM Evaluation in Cytopenia - a Multi-Center Observational Study","Inclusion Criteria:\n\n1. Patients aged 18 and up with suspected\u002Fconfirmed MDS cases referred to BM evaluation either for diagnosis or risk assessment due to cytopenia\n2. Platelets \\\u003C 150 × 10E9\u002FL or\n3. Absolute neutrophil count \\\u003C 1.8 × 10E9\u002FL or\n4. Hemoglobin (Hgb) \\\u003C 13 g\u002FdL (males) and \\\u003C 12 g\u002FdL (female) and\n5. For both sexes, no evidence of Iron, folinic acid, or B12 deficiency\n\nExclusion Criteria:\n\n1. Women who are pregnant\n2. Previous diagnosis of leukemia; AML, MPN, ALL, CLL, MGUS\u002FMM or any other gammopathy\n3. Lymphocytes \\> 5000\u002Ful\n4. Patients who are on disease-related therapy are excluded, unless they are treated with Erythropoietin or Prednisone. See Appendix 2 for the list of excluded treatments.\n5. Patients who have undergone a bone marrow transplant.",{"count":146,"type":21},"This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing.",[348,28],"Cytopenia","2026-03-22",{"date":351,"type":53},"2026-03-25",{"date":353,"type":53},"2025-06-01",{"date":355,"type":21},"2028-12",{"name":357,"class":103},"Weizmann Institute of Science",2,{"id":360,"slug":361,"hasResults":12,"nctId":362,"briefTitle":363,"officialTitle":363,"acronym":364,"eligibilityCriteria":365,"healthyVolunteers":12,"sex":17,"minAge":366,"maxAge":367,"enrollmentInfo":368,"targetDuration":4,"studyType":22,"phases":370,"briefSummary":371,"conditions":372,"keywords":377,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":378,"lastUpdatePostDateStruct":379,"startDateStruct":381,"completionDateStruct":383,"leadSponsor":385,"locationsCount":358},"100500462","phase-2-a-pilot-study-to-evaluate-the-feasibility-of-post-hematopoietic-stem-cell-transplant-prophylaxis-with-decitabine-combined-with-filgrastim-for-children-and-young-adults-with-aml-mds-and-related-myeloid-malignancies-100500462","NCT05796570","A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies","MORE","Inclusion Criteria:\n\n* Disease Criteria: Participants must have a histologically confirmed diagnosis of one of the following hematologic malignancies for eligibility, as defined by the criteria below:\n\n  * AML (relapsed, de-novo or secondary) based on WHO classification\n  * MDS (relapsed, de-novo or secondary) based on WHO classification\n  * Treatment myeloid neoplasm (tMDS\u002FAML; relapsed disease included)\n  * Myeloid Sarcoma\n  * Acute Undifferentiated Leukemia (MPAL and acute leukemia of ambiguous lineage\u002FNOS not eligible)\n* Note: MDS, AML, MDS\u002FAML, or tMDS\u002FAML as defined above may be idiopathic\u002Fde novo or derived from a germline predisposition to myeloid malignancy. For patients with an underlying germline disorder, those conditions that are not associated with increased risk for toxicity to treatment, including patients with known germline ANKRD26, DDX41, ELANE and other congenital neutropenia disorders, ETV6, GATA-2, Li-Fraumeni, RUNX1, SAMD9\u002FSAMD9L, or Shwachman-Diamond Syndrome, will be analyzed within the general treatment cohort (Cohort A, see Table 1) along with patients with idiopathic disease (Cohort B, see Table 2).\n\nMDS, AML, MDS\u002FAML, or tMDS\u002FAML derived from the following germline disorders will be enrolled in a separate cohort (B) and adverse events monitored closely for higher rates compared to cohort A:\n\n* Dyskeratosis Congenita or associated telomeropathies as defined by telomere length \\\u003C1st percentile on 3 out of 4 lymphocyte subsets and\u002For corresponding pathogenic genetic mutation.\n* Fanconi Anemia as defined by positive chromosomal breakage test to DEB\u002FMMC and\u002For corresponding pathogenic genetic mutation.\n* Nijmegen Breakage Syndrome as defined by positive chromosomal breakage test to DEB\u002FMMC and\u002For corresponding pathogenic genetic mutation\n* ERCC6L2 by genomic testing.\n\nTable 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)\n\n-iBMF with Standard risk for Treatment Related toxicities:\n\n* germline mutations in ANKRD26\n* germline mutations in DDX41\n* ELANE and other Congenital Neutropenia Disorders\n* germline mutations in ETV6\n* germline mutations in GATA-2\n* Li-Fraumeni\n* germline mutations in RUNX1\n* SAMD9\u002FSAMD9L\n* Shwachman-Diamond Syndrome\n* Familial MDS with thrombocytopenia\n* Diamond-Blackfan Anemia\n\nTable 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)\n\n* iBMF with Increased Risk for Treatment Related Toxicities:\n\n  * Fanconi Anemia\n  * Dyskeratosis Congenita and associated Telomere Disorders\n  * Nijmegen Breakage Syndrome\n  * ERCC6L2\n* Patients must be receiving an allogeneic hematopoietic stem cell transplant. All donor types and graft sources are permitted. All conditioning regimens are permitted. All GVHD prophylaxis regimens are permitted.\n* Timing of Enrollment: Registration can occur from day - 40 to day - 1 prior to stem cell infusion.\n* Disease Status: Study enrollment will occur pre HCT. Any disease status is acceptable at the time of enrollment; however, patients must be in a MRD negative remission (as defined by multidimensional flow cytometry (MDF) post HCT prior to protocol treatment start). Post HCT\u002F pretreatment disease status will be performed by Hematologics.\n* No limitations on prior therapy.\n* Age ≥1 year and ≤ 39 year of age.\n* ECOG performance status ≤2 (Lansky, Karnofsky ≥60%).\n* Participants must have adequate organ function to be eligible for allogenic HCT as per institutional standard.\n* Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial. Anti- retroviral therapy must not have a non-acceptable drug interaction with protocol treatment.\n* For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. Suppressive therapy must not have a non-acceptable drug interaction with protocol treatment.\n* Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. Ongoing antiviral therapy must not have a non-acceptable drug interaction with protocol treatment.\n* Participants with a malignancy in remission are eligible for this trial.\n* Participants with known history or current symptoms of cardiac disease should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better.\n* The effects of filgrastim on the developing human fetus are unknown. For this reason and because decitabine is known to be teratogenic, 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 completion of decitabine administration.\n* Ability to understand and the willingness to sign a written informed consent document.\n\nExclusion Criteria:\n\n* Participants who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities \\> Grade 2) except for bone marrow suppression.\n* Participants should not be enrolled on another study that prohibits initiation of maintenance therapy.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to decitabine or filgrastim.\n* Participants with uncontrolled intercurrent illness.\n* Participant who are not able to present for clinic visits for at least 7 months after study treatment initiation.\n* Participant with FLT3\u002FITD mutations are excluded as maintenance therapy with tyrosine kinase therapy should be considered in this context. However, if a participant has a co-occurring NUP28 mutation, they will be considered eligible.\n* Participants with a concurrent active malignancy are not eligible for this trial.","1 Year","39 Years",{"count":369,"type":21},37,[117],"The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT.\n\nThe names of the study drugs involved in this study are:\n\n* Decitabine (a nucleoside metabolic inhibitor)\n* Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)",[225,252,373,28,374,375,376],"Myeloid Malignancies","Inherited Bone Marrow Failure Syndrome","Myeloid Neoplasm","Aml",[225,252,373,28,374,375,27],"2026-03-17",{"date":380,"type":53},"2026-03-19",{"date":382,"type":53},"2023-04-19",{"date":384,"type":21},"2029-09-01",{"name":386,"class":103},"Franziska Wachter",{"id":388,"slug":389,"hasResults":12,"nctId":390,"briefTitle":391,"officialTitle":392,"acronym":4,"eligibilityCriteria":393,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":394,"enrollmentInfo":395,"targetDuration":4,"studyType":22,"phases":397,"briefSummary":398,"conditions":399,"keywords":406,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":416,"lastUpdatePostDateStruct":417,"startDateStruct":419,"completionDateStruct":421,"leadSponsor":423,"locationsCount":137},"100411935","phase-2-cord-blood-transplant-in-children-and-young-adults-with-blood-cancers-and-non-malignant-disorders-100411935","NCT04644016","Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders","Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders","Participant Inclusion Criteria:\n\nAge and Donor Status:\n\nPatients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period.\n\nDiagnoses :\n\nI. Acute myelogenous leukemia (AML) :\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).\n  * Therapy-related AML (t-AML).\n  * White cell count at presentation \\> 100,000.\n  * Presence of extramedullary leukemia at diagnosis.\n  * Any unfavorable subtype by FAB or WHO classification.\n  * High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.\n  * Requirement for 2 or more inductions to achieve CR1.\n  * Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.\n  * Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.\n  * Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in ≤ 10% of cells are eligible.\n\nII. Acute lymphoblastic leukemia (ALL):\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * White cell count at presentation \\> 30,000 for B-cell lineage and \\> 100,000 for T-cell lineage.\n  * Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.\n  * Failure to achieve complete remission (CR) after four weeks of induction therapy.\n  * Persistence or recurrence of MRD on therapy.\n  * Any patient unable to tolerate consolidation and\u002For maintenance chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and\u002For CAR-T cell therapy.\n\nIII. Other acute leukemias:\n\nLeukemias of ambiguous lineage or of other types (e.g. blastic plasmacytoid dendritic cell neoplasm) with less than 5% blasts by BM morphology. Patients with persistent\u002Frelapsed disease with cytogenetic, flow cytometric or molecular aberrations in ≤ 5% of cells are eligible.\n\nIV. Myelodysplastic Syndrome (MDS) \u002F Myeloproliferative Disorders (MPD) other than myelofibrosis:\n\n* International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.\n* Any IPSS risk category if life-threatening cytopenia(s) exists.\n* Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.\n* MDS\u002F myeloproliferative disorder overlap syndromes without myelofibrosis.\n* MDS\u002F MPD patients must have less than 10% bone marrow myeloblasts and ANC ≥ 0.2 (growth factor supported if necessary) at transplant work-up.\n\nV. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission:\n\n* Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.\n* Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease\u002F CR\u002F PR with no single lesion equal to or more than 5 cm.\n* Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion ≥ 5 cm.\n\nVI. Inherited Metabolic Disorders \\[also see EBMT Handbook for discussion on patient eligibility for allogeneic transplant; in general, patients are considered early in the disease course, before they develop neurologic symptoms (46)\\]:\n\n* Hurler Syndrome\n* Hunter (MPS 2 - early disease)\n* Sly syndrome (MPSVIII)\n* α-Mannosidosis\n* X- ALD\n* Osteopetrosis\n* Metachromatic Leukodystrophy\n* Globoid (GLD)\n\nVII. Non-Malignant disorders (other) \\[also see EBMT Handbook for criteria for transplant (46)\\]\n\n* Hemoglobinopathies\n* Bone Marrow Failure syndromes\n* Immunodeficiencies, including HLH\n\nOrgan Function and Performance Status Criteria:\n\n* Karnofsky or Lansky score ≥ 70% (see Appendix)\n* Bilirubin ≤ 1.5 mg\u002FdL (unless benign congenital hyperbilirubinemia).\n* ALT ≤ 3 x upper limit of normal.\n* Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .\n* Left ventricular ejection fraction ≥ 50%.\n* Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.\n* Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \\> 50 mL\u002Fmin\u002F1.73m2 (calculated or estimated) or GFR (mL\u002Fmin\u002F1.72m2) \\>30% of predicted normal for age.\n\nNormal GFR in Children and Young Adults (Age) : Mean GFR +- SD (mL\u002Fmin\u002F1.73 m2)\n\n1 week: 40.6 + \u002F - 14.8 2-8 weeks: 65.8 + \u002F - 24.8 \\>8 weeks: 95.7 + \u002F - 21.7 2-12 years: 133.0 + \u002F - 27.0 13-21 years (males): 140.0 + \u002F - 30.0 13-21 years (females: 126.0 + \u002F - 22.0\n\nGFR, glomerular filtration rate; SD, standard deviation greater than 2 years old: Normal GFR is 100 mL\u002F min. Infants: GFR must be corrected for body surf ace area.\n\nFor metabolic diseases: disease status to be evaluated according to EBMT Handbook \\[45\\].\n\nGraft Criteria\n\nCB units will be selected according to the current MSKCC unit selection algorithm. High resolution 8 allele HLA typing and recipient HLA antibody profile will be performed. Cord unit selection will occur based on HLA-match, total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body weight. The cord bank of origin will also be considered. Donor specific HLA antibodies, if present, will also be taken into consideration and may influence the selection of the graft. CB graft will consist of one or two CB units (CBU) based on MSKCC selection algorithm.\n\n* Each CB unit must be at least 3\u002F8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.\n* For malignant diseases follow MSKCC CBU selection algorithm\n* For non-malignant diseases, CBU will be required to have \\> 5 x 107 TNC\u002Fkg; high HLA allele level match is preferable\n\nParticipant Exclusion Criteria:\n\n* Inadequate performance status\u002F organ function.\n* Advanced metabolic disease (EBMT handbook).\n* Active CNS leukemic involvement.\n* Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.\n* Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.\n* Autologous stem cell transplant within the preceding 6 months.\n* Any prior allogeneic stem cell transplant.\n* Active and uncontrolled infection (bacterial\u002Ffungal\u002Fviral) at time of transplantation.\n* HIV infection.\n* Seropositivity for HTLV-1.\n* Pregnancy or breast feeding.\n* Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.","21 Years",{"count":396,"type":21},31,[117],"This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.",[27,17,28,400,401,402,403,404,405],"MPD Withou Myelofibrosis","NHL or HL","Inherited Metabolic Disorders","Hemoglobinopathies","Bone Marrow Failure","HLH",[407,27,125,28,408,409,410,411,412,413,414,415],"Acute myelogenous leukemia","Myeloproliferative Disorder","Therapy-Related AML and MDS","Therapy-Related Acute Myeloid Leukemia","T-AML","High-risk cytogenetics","Cord Blood Transplantation","20-480","Memorial Sloan Kettering Cancer Center","2026-03-12",{"date":418,"type":53},"2026-03-13",{"date":420,"type":53},"2020-11-20",{"date":422,"type":21},"2026-12-20",{"name":415,"class":103},{"id":425,"slug":426,"hasResults":12,"nctId":427,"briefTitle":428,"officialTitle":429,"acronym":4,"eligibilityCriteria":430,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":431,"targetDuration":4,"studyType":22,"phases":433,"briefSummary":434,"conditions":435,"keywords":436,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":441,"lastUpdatePostDateStruct":442,"startDateStruct":444,"completionDateStruct":446,"leadSponsor":448,"locationsCount":450},"100453222","phase-1-study-evaluating-combination-of-luspatercept-in-lr-mds-without-rs-having-failed-or-being-ineligible-to-esa-100453222","NCT05181735","Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA","A Randomized Phase I\u002F II Multicenter Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA","Inclusion Criteria:\n\nPatients must meet all of the following criteria to participate in the study:\n\n* Myelodysplastic syndrome according to current WHO classification\n* Age ≥ 18 years\n* Patients with lower risk MDS according to IPSS classification (LOW, INT-1) without RS who failed to achieved a response or who subsequently relapse after ESA (at least 60000 U EPO-a over at least 12weeks or equivalent), without disease progression (or ineligible to ESA defined by EPO \\> 500 UI\u002Fl)\n* Hemoglobin \\\u003C 9 gr\u002Fdl or Transfusion dependant (at least 3 RBCs in 16 wk in at least 2 transfusion episodes)\n* Non del(5q) syndrome\n* Adequat renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance ≥ 40 mL\u002Fmin (MDRD formula).\n* Adequat liver function, defined by total bilirubin and transaminases less than 1.5 times the upper limit of normal.\n* Patient is not known to be refractory to platelet transfusions.\n* Written informed consent.\n* Patient must understand and voluntarily sign consent form.\n* Patient must be able to adhere to the visit schedule as outlined in the study and follow protocol requirements.\n* ECOG performance status 0-2 at the time of screening.\n* A FCBP (female of childbearing potential) for this study was defined as a sexually mature woman who: (1) had not undergone a hysterectomy or bilateral oophorectomy; or (2) had not been naturally postmenopausal (amenorrhea following cancer therapy did not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). A FCBP participating in the study must:\n\n  * Have had 2 negative pregnancy tests as verified by the investigator prior to starting IP (unless the screening pregnancy test was done within 72 hours of Cycle 1 Day 1). She must have had agreed to ongoing a monthly pregnancy testing during the course of the study and after EOT\n  * If sexually active, agreed to have used, and been able to comply with, highly effective contraception\\*\\* without interruption, 5 weeks prior to starting IP, during treatment with IP (including dose interruptions), and for 12 weeks after discontinuation of IP.\n  * \\*\\* Highly effective contraception was defined in this protocol as the following (information also appeared in the ICF): Hormonal contraception (eg, birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device, tubal ligation (tying your tubes), or a partner with a vasectomy\n* Male subjects must: Have agreed to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (eg, polyurethane), during sexual contact with a pregnant female or a FCBP while participating in the study, during dose interruptions, and for at least 12 weeks following IP discontinuation, even if he had undergone a successful vasectomy\n\nExclusion Criteria:\n\nA patient meeting any of the following criteria is not eligible to participate in the study:\n\n* Severe infection or any other uncontrolled severe condition.\n* Uncontrolled hypertension\n* Significant cardiac disease - NYHA Class III or IV or having suffered a myocardial infarction in the last 6 months.\n* del(5q) syndrome\n* Use of investigational agents within 30 days or any anticancer therapy (including IMiD) within 2 weeks before the study entry with the exception of hydroxyurea. The patient must have recovered at least a grade 1 from all acute toxicity from any previous therapy.\n* Use of EPO within 4 weeks before the study entry\n* Active cancer, or cancer during the year prior to trial entry other than basal cell carcinoma, or carcinoma in situ of the cervix or breast.\n* Patient already enrolled in another therapeutic trial of an investigational drug.\n* Known HIV infection or active hepatitis B or C.\n* Women who are or could become pregnant or who are currently breastfeeding.\n* Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form.\n* Patient eligible for allogeneic stem cell transplantation.\n* Known allergies to luspatercept or EPO or any of its excipients.\n* No affiliation to a health insurance system.",{"count":432,"type":21},150,[24,117],"Study of the combination of luspatercept in low-risk myelodysplastic syndrom (LR-MDS) without ring sideroblasts (RS) having failed or being ineligible to ESA",[28,252],[28,437,438,439,440],"LR-MDS","Luspatercept","Eprex","ESA","2026-03-03",{"date":443,"type":53},"2026-03-05",{"date":445,"type":53},"2022-05-18",{"date":447,"type":21},"2029-06-19",{"name":449,"class":103},"Groupe Francophone des Myelodysplasies",40,{"id":452,"slug":453,"hasResults":12,"nctId":454,"briefTitle":455,"officialTitle":456,"acronym":457,"eligibilityCriteria":458,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":459,"targetDuration":4,"studyType":22,"phases":461,"briefSummary":463,"conditions":464,"keywords":467,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":471,"lastUpdatePostDateStruct":472,"startDateStruct":474,"completionDateStruct":476,"leadSponsor":478,"locationsCount":137},"100618174","quality-of-life-guided-transfusion-in-refractory-mds-or-aml-100618174","NCT07328191","Quality of Life-Guided Transfusion in Refractory MDS or AML","Quality of Life-based Transfusion in Refractory MDS or AML Under Advanced Palliative Care and Supportive Treatment.","Q-TRANS","Inclusion Criteria:\n\n* Adults aged 18 years and older.\n* Diagnosis of refractory MDS or AML, receiving palliative care only.\n* Anaemia with haemoglobin ≤ 8 g\u002Fdl at inclusion (or ≤ 9 g\u002Fdl if associated with cardiovascular disease).\n* Transfusion dependency, defined as requiring more than 2 GR transfusions every 8 weeks.\n* No vitamin B9, vitamin B12 or iron deficiency.\n* Ability to understand and sign the informed consent form.\n* Ability to comply with the schedule of visits and other protocol requirements.\n\nNon inclusion Criteria:\n\n* Previous malignant disease other than MDS or AML (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast).\n* Active uncontrolled infection (hepatitis B or C, HIV).\n* Use of G-CSF.\n* Confirmed neurocognitive disorders (based on prior diagnosis or clinical assessment by the investigator) impairing comprehension, consent or the ability to reliably complete the EQ-5D-5L questionnaire.\n* Active uncontrolled heart disease.\n* Active haemolytic anaemia.\n* Recent major surgery.\n* Life-threatening complications of MDS\u002FAML.\n* Presence of another serious or unstable disease which, in the investigators' opinion, would make participation in the study inappropriate or risky for the patient's safety.\n* Vulnerable individuals.\n\nExclusion Criteria:\n\n* Immediate severe complications related to MDS\u002FAML, such as uncontrolled bleeding, pneumonia with hypoxia or shock, or severe disseminated intravascular coagulopathy.\n* Withdrawal of the patient's voluntary informed consent.",{"count":460,"type":21},52,[462],"NA","Patients with refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) in exclusive palliative care frequently receive red blood cell transfusions based on hemoglobin thresholds, despite limited evidence of clinical benefit in this setting.\n\nThis prospective randomized study compares a standard hemoglobin-based transfusion strategy to a quality-of-life-guided strategy using the EQ-5D-5L questionnaire, with the aim of reducing transfusion burden while maintaining patient safety and quality of life.",[28,465,466],"AML, Adult","Anemia",[468,469,470,28,27],"palliative care","Chronic Transfusion-Dependent Anemia","Transfusion-dependent anemia","2026-02-10",{"date":473,"type":53},"2026-02-12",{"date":475,"type":53},"2026-02-09",{"date":477,"type":21},"2028-02-09",{"name":479,"class":103},"Centre Hospitalier Universitaire de Nice",{"id":481,"slug":482,"hasResults":12,"nctId":483,"briefTitle":484,"officialTitle":485,"acronym":486,"eligibilityCriteria":487,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":488,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":490,"conditions":491,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":475,"lastUpdatePostDateStruct":506,"startDateStruct":507,"completionDateStruct":509,"leadSponsor":511,"locationsCount":137},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":489,"type":21},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[492,225,493,494,495,264,28,496,497,498,499,500,501,502,503,504,505],"Multiple Myeloma","Hemophilia","Hemophilia A","Hemophilia B","Lymphoma","Leukemia","Aplastic Anemia","Bleeding Disorder","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","Paroxysmal Nocturnal Hemoglobinuria, PNH",{"date":473,"type":53},{"date":508,"type":53},"2020-12-01",{"date":510,"type":21},"2030-12-01",{"name":512,"class":103},"Institute of Hematology & Blood Diseases Hospital, China",{"id":514,"slug":515,"hasResults":12,"nctId":516,"briefTitle":517,"officialTitle":517,"acronym":4,"eligibilityCriteria":518,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":394,"enrollmentInfo":519,"targetDuration":4,"studyType":22,"phases":521,"briefSummary":522,"conditions":523,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":527,"lastUpdatePostDateStruct":528,"startDateStruct":530,"completionDateStruct":532,"leadSponsor":534,"locationsCount":137},"100541177","phase-1-car-t-cell-therapy-directed-to-cd70-for-pediatric-patients-with-hematological-malignancies-100541177","NCT06326463","CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies","Inclusion Criteria\n\nAge ≤21 years old\n\nRelapsed\u002Frefractory CD70+ hematological malignancy\n\nRelapsed disease: Patients developing recurrent disease after a prior complete remission (CR)\n\nRefractory disease: Patients with persistent disease despite 3 cycles of induction chemotherapy.\n\n* Relapsed\u002Frefractory CD70+ AML or MDS:\n\n  * Relapsed disease that is CD70 positive\n  * Refractory disease that is persistent despite 3 cycles of chemotherapy\n* Relapsed\u002Frefractory CD70+ B-cell ALL:\n\n  * Relapsed disease that is CD70 positive and CD19 negative\u002Fdim or patients otherwise ineligible for CD19-directed therapies including:\n  * Patients in 2nd or greater relapse\n  * Patients with relapse after allogeneic HSCT\n* Relapsed\u002Frefractory CD70+ T-cell ALL:\n\n  * Relapsed \u002Frefractory disease that is CD70 positive\n* Mixed Phenotype Acute Leukemia (MPAL):\n\n  * Relapsed\u002Frefractory that is CD70 positive\n* Relapsed\u002Frefractory CD70+ lymphoma:\n\n  * Relapsed disease that is CD70 positive and CD19 negative\u002Fdim or patients otherwise ineligible for CD19-directed therapies including:\n  * Patients in 2nd or greater relapse\n  * Patients with relapse after allogeneic HSCT\n\nEstimated life expectancy of \\>12 weeks\n\nKarnofsky or Lansky (age- dependent) performance score ≥50\n\nPatients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis\n\nPatient must have an identified HCT donor\n\nFor females of childbearing age:\n\ni. Not lactating with intent to breastfeed\n\nii. Not pregnant with negative serum or urine pregnancy test within 7 days prior to enrollment\n\nExclusion Criteria\n\n* Known primary immunodeficiency\n* Known history of HIV positivity\n* Severe intercurrent bacterial, viral or fungal infection\n* History of hypersensitivity to cornstarch or hydroxyethyl starch\n* Patients with acute promyelocytic leukemia (APL)\n* Known contraindication to protocol defined lymphodepleting\n* chemotherapy regimen of Fludarabine\u002Fcyclophosphamide",{"count":520,"type":21},18,[24],"The study participant has one of the following blood cancers: acute myelogenous leukemia (AML)\u002Fmyelodysplastic syndrome (MDS), acute lymphoblastic leukemia (B-ALL, T-ALL) or Lymphoma. Your cancer has been difficult to treat (refractory) or has come back after treatment (relapse).\n\nPrimary Objective\n\nTo determine the safety and maximum tolerated dose of intravenous infusions of escalating doses of CD70-CAR T cells in patients (≤21 years) with recurrent\u002Frefractory CD70+ hematological malignancies after lymphodepleting chemotherapy.\n\nSecondary Objectives\n\nTo evaluate the antileukemic activity of CD70-CAR T cells. We will determine the anti- leukemic activity of the CD70-CAR T cells in the bone marrow and in the treatment of extramedullary disease.",[524,525,526,496,28],"Hematologic Malignancy","ALL, Childhood","AML, Childhood","2026-01-22",{"date":529,"type":53},"2026-01-23",{"date":531,"type":53},"2024-10-16",{"date":533,"type":21},"2031-07-01",{"name":211,"class":103},{"id":536,"slug":537,"hasResults":12,"nctId":538,"briefTitle":539,"officialTitle":540,"acronym":4,"eligibilityCriteria":541,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":542,"targetDuration":4,"studyType":22,"phases":544,"briefSummary":545,"conditions":546,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":547,"lastUpdatePostDateStruct":548,"startDateStruct":550,"completionDateStruct":552,"leadSponsor":554,"locationsCount":137},"100593406","phase-1-a-study-of-oral-tetrahydrouridine-decitabine-in-relapsed-or-refractory-myelodysplastic-syndromes-mds-100593406","NCT07006025","A Study of Oral Tetrahydrouridine-Decitabine in Relapsed or Refractory Myelodysplastic Syndromes (MDS)","Phase 1 Study of Oral Tetrahydrouridine-Decitabine to Treat Relapsed or Refractory Myelodysplastic Syndromes","Inclusion Criteria:\n\n* Patients must have a diagnosis of MDS that has received one or more prior standard therapies and is relapsed or refractory\n* Patients must be 18 years of age or older\n* Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 3\n* Patients must have adequate end-organ function\n* Patient's body weight must be ≥ 41 kg\n* Subjects must be able to understand and willing to sign a written informed consent document and complete study-related procedures.\n\nExclusion Criteria:\n\n* Diagnosis of acute promyelocytic leukemia (APL)\n* Prior treatment with ≥4 28-day cycles of parenteral or oral decitabine\n* No other disease-directed therapy, save for hydroxyurea, including experimental or investigational drug therapy for 14 days prior to study entry (hydroxyurea should be discontinued ≥24 hours prior to initiation of study drug)\n* Requiring concomitant treatment with drugs that are cytidine deaminase (CDA) substrates and\u002For inhibitors, e.g., cytarabine, 5-azacytidine, gemcitabine\n* Currently pregnant or breastfeeding. Females of childbearing potential must have a negative serum pregnancy test within 72 hours of treatment start.\n* Uncontrolled intercurrent illness that could limit life expectancy or ability to complete study correlates\n* Women of Childbearing Potential (WOCBP) who are unwilling to agree to use dual contraceptive measures (i.e., hormonal or barrier method of birth control; abstinence, condom) prior to study entry, for the duration of study participation, and until 6 months after taking the last dose of THU\u002Fdecitabine\n* Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a WOCBP, beginning at the screening visit and continuing until 6 months after taking the last dose of THU\u002Fdecitabine\n* 9\\. Patients with uncontrolled active human retrovirus (HIV) infection, as this will further increase the risk for opportunistic infections. However, patients with HIV with undetectable viral load by polymerase chain reaction (PCR), without opportunistic infection, and on a stable regimen of antiretroviral therapy are eligible",{"count":543,"type":21},20,[24],"The goal of this clinical trial is to learn about the safety and effectiveness of the combination drug Tetrahydrouridine (THU) and decitabine (DEC) to treat patients with relapsed or refractory myelodysplastic syndrome. The main questions it aims to answer are:\n\n* Does the combination drug exhibit hematological and nonhematological toxicity?\n* Does the combination drug improve health status and reduce the number of days of hospitalization?\n\nParticipants will:\n\n* Take tetrahydrouridine and decitabine once a week for 24 weeks\n* Visit the clinic once every 4 weeks for checkups and tests\n* Keep a diary of their symptoms",[28],"2026-01-13",{"date":549,"type":53},"2026-01-15",{"date":551,"type":53},"2025-06-03",{"date":553,"type":21},"2027-03-30",{"name":555,"class":60},"Treebough Therapies",{"id":557,"slug":558,"hasResults":12,"nctId":559,"briefTitle":560,"officialTitle":561,"acronym":4,"eligibilityCriteria":562,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":299,"enrollmentInfo":563,"targetDuration":4,"studyType":22,"phases":565,"briefSummary":566,"conditions":567,"keywords":570,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":573,"lastUpdatePostDateStruct":574,"startDateStruct":576,"completionDateStruct":578,"leadSponsor":580,"locationsCount":582},"100619634","phase-1-a-phase-1-study-of-cg009301-for-injection-in-adult-subjects-with-recurrent-or-refractory-haematological-malignancies-100619634","NCT07347171","A Phase 1 Study of CG009301 for Injection in Adult Subjects With Recurrent or Refractory Haematological Malignancies","A Phase 1, Open-label, Multicentre Study Evaluating the Safety, Tolerability, Pharmacokinetic\u002FPharmacodynamic Characteristics, and Preliminary Efficacy of CG009301 for Injection in Adult Subjects With Relapsed or Refractory Haematological Malignancies","Inclusion Criteria:\n\n1. Age ≥18 years and \\\u003C75 years at the time of signing the informed consent form; no gender restrictions;\n2. Patients with relapsed\u002Frefractory haematological malignancies who have received a definitive diagnosis by pathology and\u002For cytology, confirmed histologically, and who have failed prior standard treatment regimens. The investigator must deem that no standard treatment is available or that the patient cannot tolerate existing therapies. Dose-escalation phase: unrestricted haematological tumour types. Dose-expansion phase: must meet one of the following criteria: a. Subjects meeting AML diagnostic criteria based on WHO 2022 5th edition classification, confirmed by bone marrow cytomorphology, including AML evolving from early-stage MDS or MPN. Criteria for recurrent AML: Leukaemic cells reappearing in peripheral blood after CR, or \\>5% blast\u002Fimmature cells in bone marrow, or extramedullary leukaemic infiltration. Criteria for refractory AML: - Treatment-naïve cases unresponsive to two standard-regimen cycles; - Relapse within 12 months after consolidation\u002Fintensification therapy following CR; - Relapse after 12 months unresponsive to conventional chemotherapy; Patients with two or more relapses; Persistent extramedullary leukaemia; b. Patients diagnosed with high-risk or very high-risk MDS according to the WHO 2022 5th edition classification, with a percentage of blasts in bone marrow smear or biopsy pathology \\\u003C 20%, and deemed by the investigator to have no other appropriate treatment options. Diagnostic criteria for recurrent MDS: Following achievement of complete remission, partial remission, or haematological improvement, at least one of the following must occur: - Bone marrow blastic count returns to pre-treatment levels; - ANC or PLT decreases by ≥50% from best response; - HGB decreases by ≥15 g\u002FL or becomes transfusion-dependent. Diagnostic criteria for refractory MDS: Following adequate treatment (at least four cycles of demethylating agent therapy), meeting the IWG 2023 response criteria for \"stable disease\", \"failure\", or \"disease progression\"; progression after demethylating agent or other drug therapy, or patient intolerance to toxicity (e.g., treatment-related grade 3 or higher hepatic or renal toxicity during therapy leading to permanent discontinuation); c. Subjects meeting ALL diagnostic criteria based on WHO 2022 5th edition classification, with ≥20% primitive\u002Fimmature lymphocytes in bone marrow. Relapsed ALL diagnostic criteria: Patients who, after achieving CR following induction therapy, exhibit recurrence of leukaemic cells in peripheral blood, \\>5% primitive\u002Fimmature lymphocytes in bone marrow, or development of extramedullary disease; Criteria for refractory ALL: Patients failing to achieve CR following standard induction therapy;\n3. ECOG performance status score of 0-1;\n4. Investigator-assessed expected survival ≥3 months;\n5. Recovery of toxicities from prior treatment to ≤Grade 1 according to NCI-CTCAE v5.0 (excluding alopecia and long-term stable chronic conditions);\n6. No prior autologous haematopoietic stem cell transplantation, or transplantation more than 2 months prior with toxicities resolved to ≤ Grade 1;\n7. Adequate organ function support, with screening laboratory tests meeting all criteria: a. Coagulation function prior to study drug administration: INR ≤ 1.5 × ULN or aPTT ≤ 1.5 × ULN; b. Hepatic function: serum total bilirubin ≤ 2× ULN; AST and\u002For ALT ≤ 2.5× ULN; c. Cr ≤ 2× ULN or CrCL \\> 30 mL\u002Fmin (calculated using Cockcroft-Gault formula); d. LVEF ≥ 40%; and QTc ≤ 480 milliseconds; e. White blood cell count may decrease below 50.0 × 10⁹\u002FL at baseline or following hydroxyurea administration\n8. Non-pregnant and non-lactating: Infertile subjects; or subjects with potential for conception who agree to use effective contraception (hormonal, barrier, or abstinence). Male subjects must also abstain from sperm donation during study participation and for 90 days after the last dose of CG009301 injection. Women of childbearing potential must have a negative serum pregnancy test (serum-β-hCG) during the screening period;\n9. Understand the study's purpose, process, nature, significance, potential benefits, and risks, and voluntarily sign the written informed consent form. Be able to comply with scheduled visits, treatment plans, laboratory tests, and other study instructions or procedures.\n\nExclusion Criteria:\n\n1. Central nervous system leukaemia presenting with neurological and\u002For psychiatric symptoms;\n2. Receipt of antitumour therapy (excluding hydroxyurea and prophylactic intrathecal injections) such as chemotherapy, immunotherapy, targeted therapy, or biological therapy within 4 weeks or 5 half-lives (whichever is shorter) prior to the first study drug administration; receipt of radiotherapy within 2 weeks; receipt of traditional Chinese herbal medicine within 2 weeks;\n3. Major surgery within 4 weeks prior to the first study dose, or anticipated need for major surgery during the study period;\n4. Active infection deemed uncontrolled by the investigator following treatment with antibiotics, antiviral agents, or antifungal medications;\n5. Severe or uncontrolled underlying medical conditions deemed ineligible for inclusion by the investigator, including but not limited to respiratory disorders (e.g., chronic obstructive pulmonary disease requiring oxygen therapy, moderate or higher asthma, moderate or higher pulmonary fibrosis, recurrent pulmonary oedema), cardiovascular disorders (e.g., prior coronary artery bypass grafting or coronary stent implantation, myocardial infarction within the past 6 months, NYHA Class III-IV heart failure), unstable angina within the past 6 months, uncontrolled hypertension (systolic \\>160 mmHg or diastolic \\>100 mmHg), arrhythmias requiring ongoing medical or interventional management), endocrine disorders (severe hyperthyroidism\u002Fhypothyroidism, uncontrolled diabetes mellitus), and neurological\u002Fpsychiatric conditions affecting cognition, compliance, or personal safety (e.g., unstable epilepsy, dementia, schizophrenia, depression); psychiatric disorders (e.g., unstable epilepsy, dementia, schizophrenia, depression);\n6. Active autoimmune diseases (e.g., inflammatory bowel disease, idiopathic thrombocytopenic purpura, lupus erythematosus, autoimmune haemolytic anaemia, scleroderma, severe psoriasis, rheumatoid arthritis), or allergy to the study drug or excipients;\n7. Significant non-leukaemia-related bleeding risk (e.g., anticoagulant or antiplatelet therapy, arteriovenous malformation), or recent history of major bleeding (e.g., gastrointestinal haemorrhage, intracranial haemorrhage, disseminated intravascular coagulation);\n8. Grade 2 or higher central nervous system or peripheral neuropathy (excluding stable Grade 3 conditions lasting over 6 months that do not impair daily functioning);\n9. Allogeneic haematopoietic stem cell transplantation within 12 months prior to initial administration;\n10. History of deep vein thrombosis, pulmonary embolism, or any other severe thromboembolic event within 6 months prior to initial administration (thrombosis originating from implanted venous access ports or catheters, superficial vein thrombosis, or lacunar cerebral infarction are not considered \"severe\" thromboembolic events); Known familial and\u002For acquired thrombotic predisposition, such as hereditary or acquired defects in anticoagulant proteins, coagulation factors, fibrinolytic proteins, or presence of acquired risk factors conferring high thrombotic propensity;\n11. HIV, HBV, and HCV infection: positive HIV antibody and PCR tests; HBsAg positive or viral DNA ≥100 IU\u002FmL; positive HCV antibody with HCV-RNA quantification exceeding the upper limit of normal;\n12. Individuals who received (attenuated) live virus vaccination within 4 weeks prior to first dosing;\n13. Individuals with a documented history of alcohol or substance abuse;\n14. Any past or current medical condition, treatment, or laboratory abnormality that may interfere with study results or affect the subject's ability to complete the study, or if the investigator deems the subject unsuitable for participation in this study.",{"count":564,"type":21},45,[24],"The goal of this clinical trial is to learn about the safety of drug CG009301. It also learns if drug CG009301 works to treat in Participants with relapsed or refractory adult haematological malignancies.\n\nThe main question\\[s\\] it aims to answer are:\n\n1. To determine the maximum tolerated dose (MTD) and\u002For objective best dose (OBD) of CG009301 for injection in subjects with relapsed or refractory adult haematological malignancies.\n2. To establish subsequent dosing regimens for CG009301 for injection.\n3. To characterise the safety profile and tolerability of CG009301 for injection. Participants will Receive treatment with CG009301 until disease progression.",[497,27,28,568,569],"AML (Acute Myelogenous Leukemia)","MDS (Myelodysplastic Syndrome)",[497,571,572,27,28],"GSPT1","CG009301","2026-01-08",{"date":575,"type":53},"2026-01-16",{"date":577,"type":53},"2025-04-17",{"date":579,"type":21},"2027-12-30",{"name":581,"class":103},"Cullgen (Shanghai),Inc",3,{"id":584,"slug":585,"hasResults":12,"nctId":586,"briefTitle":587,"officialTitle":588,"acronym":589,"eligibilityCriteria":590,"healthyVolunteers":12,"sex":17,"minAge":219,"maxAge":4,"enrollmentInfo":591,"targetDuration":4,"studyType":22,"phases":592,"briefSummary":593,"conditions":594,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":596,"lastUpdatePostDateStruct":597,"startDateStruct":599,"completionDateStruct":601,"leadSponsor":603,"locationsCount":582},"100558538","phase-1-safety-of-mt-401-ots-in-patients-with-relapsed-aml-or-mds-100558538","NCT06552416","Safety of MT-401-OTS in Patients With Relapsed AML or MDS","A Phase 1 Study of Allogenic Off-the-Shelf Multi-Tumor-Associated Antigen-Specific T Cell Products (MT-401-OTS) Administered to Patients With Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndromes (RAPID)","RAPID","Inclusion Criteria:\n\n* General\n\n  1. Must be ≥ 65 years of age and capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in the protocol, at the time of signing the ICF\n  2. Must have a life expectancy ≥ 12 weeks\n  3. Must have an ECOG performance status of 0-2\n  4. Must have available MT-401-OTS product with a ≥ 2\u002F8 HLA match Disease Characteristics\n  5. For participants with AML:\n\n     1. Must have a confirmed diagnosis of AML or MDS\u002FAML per 2022 WHO Classification of Haematolymphoid Tumours: Myeloid and Histiocytic\u002FDendritic Neoplasms or 2022 International Consensus Criteria\n     2. Must have intermediate or high-risk disease based on ELN 2022 criteria.\n     3. If no targetable mutation is present, must have received 1 prior standard regimen with at least 4 cycles of standard therapy containing an HMA or a standard cytarabine-containing induction therapy\n     4. If targetable mutation is present, must have received a regimen that includes commercially available targeted therapy unless unable to tolerate or the participant declines (must be documented in the informed consent). If targeted therapy was not administered as part of first-line of therapy, a second regimen is allowed.\n     5. Must have either: ≤ 10% bone marrow blasts and ≤ 5% peripheral blasts during screening and not be considered to have hyperproliferating disease at diagnosis or after treatment OR Evidence of MRD based on evaluation at a local laboratory\n  6. For participants with MDS:\n\n     1. Must have confirmed diagnosis of MDS based on 2022 WHO Classification of Haematolymphoid Tumours: Myeloid and Histiocytic\u002FDendritic Neoplasms or 2022 ICC criteria\n     2. Must have high-risk or very-high-risk disease based on IPSS-M (ie, not evolved to AML)\n     3. Must have received standard treatment with at least 4 cycles of an HMA and have evidence of continued disease, including morphologic disease or MRD-positive\n     4. Must have bone marrow blasts ≤ 10% at screening Health Status\n  7. Must have adequate coagulation, hepatic, renal, and cardiac function:\n\n     1. PT\u002FINR and PTT\u002FaPTT \\\u003C 1.3 × ULN\n     2. AST and ALT \\\u003C 3 × ULN; for participants with leukemic infiltration of the liver (documented by biopsy or imaging), AST and ALT \\\u003C 5 × ULN is permitted.\n     3. Total bilirubin ≤ 1.5 × ULN unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin (2 × ULN is permitted)\n     4. eGFR ≥ 40 mL\u002Fmin by the MDRD formula\n     5. LVEF ≥ 45% (prior to apheresis and lymphodepletion) Sex\n  8. Women of childbearing potential are eligible to participate if they agree to the following during the intervention period and for at least 1 year after the last infusion of MT-401-OTS:\n\n     1. Must use a contraceptive method that is highly effective (ie, with a failure rate of \\\u003C 1% per year; see Section 10.3), preferably with low user dependency PLUS\n     2. Must agree not to donate eggs (ie, ova and oocytes) for the purpose of reproduction\n  9. Male participants are eligible to participate if they agree to the following during the intervention period and for at least 6 months after the last infusion of MT-401-OTS:\n\n     1. Must refrain from donating sperm\n\n        PLUS either:\n     2. Must be abstinent from intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent OR\n     3. Must agree to use a male condom AND should also be advised of the benefit for a nonpregnant female partner to use a highly effective method of contraception (see Section 10.3) as a condom may break or leak\n\nExclusion Criteria:\n\n* Disease-Related\n\n  1. Have leukemic involvement in the CNS\n  2. Have other extramedullary disease involvement (except hepatosplenic involvement)\n  3. Have APL Medical Conditions\n  4. Have primary immunodeficiency\n  5. Have severe or uncontrolled autoimmune disorder\n  6. Have a history or presence of clinically relevant CNS pathology, such as epilepsy, seizure, aphasia, stroke, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome or psychosis\n  7. Have active malignancies (ie, those that are progressing or have required treatment change in the last 24 months) other than the disease being treated under study. Exceptions to this inclusion include the following:\n\n     1. Nonmelanoma skin cancer treated within the last 24 months that is considered completely cured\n     2. Adequately treated breast lobular carcinoma in situ and breast ductal carcinoma in situ\n     3. Adequately treated cervical carcinoma in situ without evidence of disease\n     4. History of localized breast cancer and receiving antihormonal agents, or history of localized prostate cancer (N0M0) and receiving androgen-deprivation therapy\n     5. A malignancy that is considered cured with minimal risk of recurrence\n  8. Have any active systemic infection requiring therapy (viral, bacterial, or fungal), including HIV\n  9. Have active hepatitis B or C infection or other clinically active liver diseases, as defined below:\n\n     1. Seropositivity for hepatitis B as defined by a positive test for HbsAg Participants with resolved infection (ie, participants who are HbsAg-negative with antibodies to total anti-HBc with or without the presence of anti-HBs) must be screened using RT-PCR measurement of HBV DNA levels. Those who are RT PCR-positive will be excluded.\n\n        Participants with serologic findings suggestive of HBV vaccination (anti HBs positivity as the only serologic marker) AND a known history of prior HBV vaccination, do not need to be tested for HBV DNA by RT PCR.\n     2. Active hepatitis C infection as defined by being positive for a nucleic acid test for HCV RNA\n  10. Have Class III or IV congestive heart failure per New York Association\n  11. Have unstable angina\n  12. Have a history or evidence of current, uncontrolled, clinically significant, unstable arrhythmias\n  13. Have an oxygen saturation on room air of ≤ 92%\n  14. Have clinically significant reversible nonhematologic toxicities from prior cancer therapy that have not recovered to Grade 1 or baseline Note: Participants with clinically nonsignificant toxicities, such as asymptomatic laboratory values, will be allowed on study.\n\n      Prior\u002FConcomitant Therapies\n  15. Received prior treatments for underlying malignancy, except as specified in the Inclusion Criteria. Participants with AML secondary to MDS may have received prior treatment for MDS.\n  16. Have had prior HSCT\n  17. Are receiving concurrent therapies other than HMA, as delineated in the study design\n  18. Have received hematopoietic growth factors within 2 days of lymphodepleting conditioning regimen\n  19. Have a history of severe allergic reactions\u002Fintolerance to any of the study intervention components, including the conditioning regimen, HMA, or DSMO, or to tocilizumab\n  20. Have had major surgery within 14 days (central line placement allowed)\n  21. Have received systemic steroids (exception: physiological doses of steroids allowed) or other immunosuppressive therapies within 14 days prior to lymphodepleting conditioning regimen Other\n  22. Are unable to be matched with MT-401-OTS product inventory\n  23. Are pregnant or breastfeeding\n  24. Have any other issue that, in the opinion of the treating physician, would make the participant ineligible for the study or unable to comply with its requirements",{"count":450,"type":21},[24],"This study is a Phase 1 multicenter, open-label study evaluating the safety and efficacy of escalating doses of MT-401-OTS in 2 participant populations: 1) Those with intermediate or high-risk AML per 2022 ELN criteria who have evidence of MRD and\u002For \\\u003C\u002F= 10% blast following prior induction therapy or at least 4 cycles of nonintensive therapy and 2) those with high- or very-high-risk MDS per 2023 IWG criteria and who have residual disease with \\\u003C\u002F= 10% blasts following treatment with an HMA-based therapy.",[595,28],"Acute Myeloid Leukemia, in Relapse","2025-12-08",{"date":598,"type":53},"2025-12-16",{"date":600,"type":53},"2025-06-16",{"date":602,"type":21},"2029-09",{"name":604,"class":60},"Marker Therapeutics, Inc.",{"id":606,"slug":607,"hasResults":12,"nctId":608,"briefTitle":609,"officialTitle":610,"acronym":4,"eligibilityCriteria":611,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":612,"targetDuration":4,"studyType":22,"phases":614,"briefSummary":615,"conditions":616,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":617,"lastUpdatePostDateStruct":618,"startDateStruct":620,"completionDateStruct":622,"leadSponsor":624,"locationsCount":626},"100557103","phase-1-eganelisib-as-monotherapy-and-in-combination-with-cytarabine-in-relapsedrefractory-aml-100557103","NCT06533761","Eganelisib as Monotherapy and in Combination With Cytarabine in Relapsed\u002FRefractory AML","A Phase 1b Open-Label Study to Evaluate the Safety and Tolerability of Eganelisib as Monotherapy and in Combination With Cytarabine in Patients With Relapsed\u002FRefractory Acute Myeloid Leukemia","Inclusion Criteria:\n\n* Pathological diagnosis of either: AML according to World Health Organization (WHO) 2022 revised criteria per the local pathology report and with ≥10% bone marrow blasts (acute promyelocytic leukemia is excluded but secondary AML and treatment-related AML can be included); Higher-risk (IPSS-R Intermediate, High or Very High Risk at time of study entry) myelodysplastic syndromes (HR-MDS) according to WHO 2022 revised criteria per the local pathology report and with ≥10% bone marrow blasts.\n* Eastern Cooperative Oncology Group (ECOG) performance status ≤2.\n* Adequate hepatic and renal function measured within 7 days prior to the first dose of eganelisib.\n\nExclusion Criteria:\n\n* Autologous or allogeneic stem cell transplant within 6 months prior to Cycle 1 Day 1.\n* Receiving immunosuppressants (eg, cyclosporin) or systemic steroids (except for steroid use as cortisol replacement therapy in documented adrenal insufficiency).\n* Active fungal disease or uncontrolled infection of any kind; patients receiving antibiotic, antifungal or antiviral treatment must be afebrile and hemodynamically stable for \\>72 hours prior to treatment\n* WBC count \\>25 × 10\\^9\u002FL measured within 7 days prior to the first dose of eganelisib (hydroxyurea is permitted to decrease the WBC count).\n* Presence of a clinically significant non-hematologic toxicity of prior therapy that has not resolved to Grade ≤1 or Baseline, whichever is worst, as determined by NCI CTCAE v 5.0, except alopecia or skin pigmentation. Fatigue and neuropathy must have resolved to Grade ≤2.",{"count":613,"type":21},125,[24],"This is a Phase 1b open-label, multicenter, dose-escalation and dose-optimization study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and anti-tumor efficacy of eganelisib as monotherapy and in combination with cytarabine in patients with relapsed\u002Frefractory (r\u002Fr) acute myeloid leukemia (AML) or r\u002Fr higher-risk myelodysplastic syndromes (HR-MDS).\n\nThe study consists of 2 parts:\n\n* Part 1: Dose Escalation (DE) in both monotherapy and in combination.\n* Part 2: Dose Optimization",[465,28],"2025-10-20",{"date":619,"type":53},"2025-10-22",{"date":621,"type":53},"2025-04-28",{"date":623,"type":21},"2028-03-15",{"name":625,"class":60},"Stelexis BioSciences",13,{"id":628,"slug":629,"hasResults":12,"nctId":630,"briefTitle":631,"officialTitle":632,"acronym":633,"eligibilityCriteria":634,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":635,"targetDuration":637,"studyType":73,"phases":4,"briefSummary":638,"conditions":639,"keywords":647,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":649,"lastUpdatePostDateStruct":650,"startDateStruct":652,"completionDateStruct":654,"leadSponsor":656,"locationsCount":137},"100608743","high-altitude-hematology-observation-stem-cell-transplantation-halo-sct-100608743","NCT07205523","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT): A Prospective Real-World Cohort Study in the Qinghai-Tibet Plateau","HALO-SCT","Inclusion Criteria:\n\n1. Patients diagnosed with hematologic diseases who are admitted to the HSCT center of Qinghai University Affiliated Hospital on or after September 1, 2023.\n2. Planned or actual hematopoietic stem cell transplantation (HSCT).\n3. Provision of signed informed consent.\n\nExclusion Criteria:\n\n1. Inability to provide long-term follow-up data due to severe comorbidities or logistical reasons.\n2. Substance abuse compromising adherence.\n3. Any condition judged by investigators to jeopardize safety or compliance.",{"count":636,"type":21},1000,"100 Years","The High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) study is the first prospective real-world cohort of hematologic diseases and transplantation in the Qinghai-Tibet Plateau. Patients undergoing hematopoietic stem cell transplantation (HSCT) at Qinghai University Affiliated Hospital, together with their donors, are systematically enrolled. The registry collects demographic, diagnostic, treatment, prognosis, and medical expense information, as well as biospecimens for future analyses. Historical data are incorporated, and prospective data collection is ongoing with long-term follow-up planned. The registry is designed as a sustainable research infrastructure to provide comprehensive data on disease incidence, treatment patterns, outcomes, and resource utilization in a high-altitude setting.",[640,641,642,643,493,264,28,496,497,498,644,500,492,645,646],"Hematopoietic Stem Cell Transplantation (HSCT)","Acute Myeloid Leukemia (AML)","Leukemias, Acute Myeloid","Myeloid Leukemias, Acute","Bleeding Disorders","Myeloma, Multiple","Immune Reconstitution",[648],"High-altitude Bone marrow transplantation Allogeneic HSCT Autologous HSCT Immune reconstitution Graft-versus-host disease Relapse Survival Quality of life","2025-09-25",{"date":651,"type":53},"2025-10-03",{"date":653,"type":53},"2023-09-01",{"date":655,"type":21},"2100-12-31",{"name":657,"class":103},"Yigeng Cao,MD,PhD",{"id":659,"slug":660,"hasResults":12,"nctId":661,"briefTitle":662,"officialTitle":663,"acronym":4,"eligibilityCriteria":664,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":113,"enrollmentInfo":665,"targetDuration":4,"studyType":22,"phases":667,"briefSummary":668,"conditions":669,"keywords":672,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":674,"lastUpdatePostDateStruct":675,"startDateStruct":677,"completionDateStruct":679,"leadSponsor":681,"locationsCount":683},"100570200","phase-1-bsb-1001-in-patients-undergoing-hla-matched-allogenic-hematopoietic-stem-cell-transplant-for-aml-all-or-mds-100570200","NCT06704152","BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS","A Phase 1\u002F2a Multicenter Ascending Dose Study to Evaluate the Safety of HA-1 Minor Histocompatibility Antigen-Reactive TCR-Modified T Cells (BSB-1001) in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS","Inclusion Criteria:\n\n1. Male or female patients, ages 18 - 70 years inclusive, undergoing alloHCT.\n2. Any of the following high-risk hematologic malignancies:\n\n   1. AML diagnosed which has been treated with at least two lines of therapy\\* Refractory or relapsed (CR, CRh or CRi,), including myeloblasts up to 25% OR MRD positive OR persistent disease-defining cytogenetic abnormality OR MRD-negative, but with high-risk disease For patients in remission meeting criteria a, consolidation regimens would be considered another line of therapy of eligibility purposes\n   2. ALL which has been with abnormal lymphoblasts ≥5% and up to 25% in bone marrow OR persistent disease-defining cytogenetic abnormality or MRD positive\n   3. MDS after at least one line of therapy, which includes hypomethylating agent(s) and must be high or very high risk by Revised International Prognostic Scoring System (IPSS-R), monosomy, or complex karyotype or TP53 mutation.\n   4. In the expansion phase AML patients diagnosed which has been treated with at least two lines of therapy, and refractory or relapsed (CR, CRh or CRi,), including myeloblasts up to 25% OR MRD positive OR persistent disease-defining cytogenetic abnormality OR MRD-negative, but with high- risk disease\n3. HLA-A\\*02:01 AND HA-1 positive (either H\u002FH or H\u002FR).\n4. Suitable for one of the approved conditioning regimens as defined in the protocol.\n5. Patient must have an identified donor that is HA 1-negative with 10\u002F10 matched related or unrelated donor\n\nExclusion Criteria:\n\n1. Weight \\> 100 kg.\n2. Prior history of allogeneic stem cell transplantation\n3. Prior history of autologous stem cell transplantation within 1 year prior to the planned dosing of BSB-1001 (day 0)\n4. Previous genetically engineered chimeric antigen receptor T Cell therapy (CAR-T), approved or investigational, within 2 years of screening, with the exception of patients with ALL previously treated with an autologous CAR-T product.\n5. Treatment with other investigational agents within 5 half-lives of the planned dosing of BSB-1001 (day 0).\n6. History of treatment with checkpoint inhibitor therapy within 3 months of transplantation.\n7. Other malignancy with life expectancy \\\u003C 1year.\n8. Pregnant or lactating women.\n9. Uncontrolled bacterial, viral, or fungal infections at time of enrollment.\n10. Past or current viral infections as defined in the protocol.\n11. CNS involvement refractory to intrathecal chemotherapy and\u002For standard cranial- spinal radiation. 12 Karnofsky Performance Score \\\u003C 60%.\n\n13\\. Inadequate organ function as defined in protocol.",{"count":666,"type":21},38,[24,117],"The goal of this clinical trial is to test BSB-1001 which is a new type of cellular therapy to treat blood cancers (AML, ALL and MDS). It will evaluate the safety of BSB-1001 and also determine whether it works to prevent relapse of your cancer.",[670,671,28],"AML, Adult Recurrent","ALL, Recurrent, Adult",[673],"TCR, T-cell therapy","2025-09-17",{"date":676,"type":53},"2025-09-18",{"date":678,"type":53},"2025-02-04",{"date":680,"type":21},"2029-03",{"name":682,"class":60},"BlueSphere Bio, Inc",6,{"id":685,"slug":686,"hasResults":12,"nctId":687,"briefTitle":688,"officialTitle":689,"acronym":690,"eligibilityCriteria":691,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":692,"enrollmentInfo":693,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":694,"conditions":695,"keywords":696,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":702,"lastUpdatePostDateStruct":703,"startDateStruct":705,"completionDateStruct":707,"leadSponsor":709,"locationsCount":358},"100591154","a-long-term-follow-up-study-of-tscan-tcr-t-products-100591154","NCT06976736","A Long Term Follow-up Study of TScan TCR-T Products","A Long-term Follow-up Study to Assess Safety in Participants Who Received an Investigational T-Cell Receptor Engineered T-Cell (TCR-T) Product","LTFU","Inclusion Criteria:\n\n* Participants who received a TCR-T cellular therapy in a clinical study sponsored by TScan Therapeutics.\n* Signed informed consent.\n\nExclusion Criteria:\n\n* None","110 Years",{"count":636,"type":21},"The purpose of this Long-Term Follow-Up (LTFU) study is to monitor participants who have previously received TSC-100 or TSC-101 TCR-T therapies in the TSCAN-001 study. Participants will be monitored for 15 years from the date of TCR-T cell therapy administration to assess long-term safety and efficacy.",[27,17,28],[697,698,699,700,31,47,701],"TSCAN","Cell Therapies","TCR-T Cell Therapy","Long Term Follow-Up (LTFU)","TSCAN-001","2025-09-02",{"date":704,"type":53},"2025-09-10",{"date":706,"type":21},"2025-09-09",{"date":708,"type":21},"2040-09",{"name":59,"class":60},{"id":711,"slug":712,"hasResults":12,"nctId":713,"briefTitle":714,"officialTitle":715,"acronym":4,"eligibilityCriteria":716,"healthyVolunteers":12,"sex":17,"minAge":717,"maxAge":4,"enrollmentInfo":718,"targetDuration":4,"studyType":22,"phases":720,"briefSummary":721,"conditions":722,"keywords":723,"overallStatus":231,"whyStopped":4,"lastUpdateSubmitDate":729,"lastUpdatePostDateStruct":730,"startDateStruct":732,"completionDateStruct":734,"leadSponsor":736,"locationsCount":4},"100603020","phase-1-qh101-cell-therapy-relapsedrefractoryrr-acute-myeloid-leukemiaaml-and-myelodysplastic-syndromesmds-100603020","NCT07131085","QH101 Cell Therapy Relapsed\u002FRefractory(R\u002FR) Acute Myeloid Leukemia(AML) and Myelodysplastic Syndromes(MDS)","Clinical Study on the Safety and Efficacy of QH101 in Patients With Relapsed\u002FRefractory Acute Myeloid Leukemia(R\u002FR AML) and Relapsed\u002FRefractory Myelodysplastic Syndromes(R\u002FR MDS)","Selection criteria:\n\n1. Age ≥ 14 years, no gender restrictions;\n2. Diagnosed with AML according to the standards of the NCCN (2024 V2), ELN (2023), and the Chinese \"Guidelines for the Diagnosis and Treatment of AML (2024 Edition)\"; or diagnosed with MDS according to the standards of the NCCN (2024 V2), ELN (2023), and the Chinese \"Expert Consensus on the Diagnosis and Treatment of MDS (2024 Edition)\"; （1） Meets the criteria for R\u002FR AML, including any of the following:\n\n   1. Relapsed: Leukemic cells reappear in the peripheral blood after complete remission, or Leukemic blasts≥5% in the bone marrow (excluding other causes such as bone marrow regeneration after consolidation chemotherapy), or leukemic cell infiltration in extramedullary sites;\n   2. Refractory: Primary cases that fail to respond to two cycles of standard treatment, or cases that relapse within 12 months after consolidation intensive therapy following complete remission (CR), or cases that relapse after 12 months and fail to respond to conventional chemotherapy, or cases with two or more relapses, or cases with persistent extramedullary leukemia; （2）Meets the criteria for R\u002FR MDS, including any of the following conditions:\n\n   \u003C!-- -->\n\n   1. Relapsed: Patients who have achieved hematologic improvement or CR but subsequently experience hematologic decline (e.g., hemoglobin \\\u003C10 g\u002FdL, platelets \\\u003C50×10⁹\u002FL, neutrophils \\\u003C1.0×10⁹\u002FL), or an increase in the proportion of blast cells in the bone marrow (≥5%), or the emergence of new cytogenetic abnormalities or molecular progression (e.g., increased TP53 mutation burden);\n   2. Refractory: No hematologic or bone marrow improvement after ≥4-6 cycles of hypomethylating agent (HMA) therapy (e.g., azacitidine or decitabine), or low-risk MDS resistant to erythropoietin or immunomodulatory agents (e.g., lenalidomide).\n3. Expected survival time exceeds 3 months;\n4. Eastern Cooperative Oncology Group (ECOG) performance status is 0-2;\n5. Organ function meets the following requirements: 1)Liver function must meet: ALT ≤ 3 × ULN; AST ≤ 3 × ULN; Total bilirubin ≤ 3.0 × ULN. 2)Renal function must meet the following criteria: Serum creatinine ≤ 1.5 × upper limit of normal (ULN); 3)Cardiac function: Echocardiogram showing left ventricular ejection fraction ≥ 50%; 4)Pulmonary function: Normal oxygen saturation without oxygen supplementation.\n6. Female participants of childbearing potential and male participants whose partners are of childbearing potential must use medically approved contraceptive measures or abstain from sexual intercourse during the study treatment period and for at least 6 months after the study treatment period. Female participants of childbearing potential must have a negative serum HCG test within 7 days prior to study enrollment and must not be breastfeeding.\n7. No significant genetic disorders;\n8. The subject or their legal guardian voluntarily participates in this study, understands the trial information, objectives, and risks described in the informed consent form, and can provide a signed and dated informed consent form;\n9. The subject or their legal guardian is willing and able to comply with all trial requirements.\n\nExclusion criteria:\n\n1. Patients with a history of severe central nervous system disorders, such as uncontrolled epileptic seizures, stroke, severe brain injury with aphasia, paralysis, dementia, Parkinson's disease, or mental disorders;\n2. New York Heart Association (NYHA) Class III or IV heart failure;\n3. Undergone coronary angioplasty, coronary stent implantation, or coronary artery bypass surgery; or experienced thrombotic or embolic events (e.g., cerebrovascular events \\[including transient ischemic attacks, but excluding lacunar cerebral infarction\\], deep vein thrombosis \\[excluding deep vein thrombosis caused by PICC catheter placement\\], pulmonary embolism, etc.);\n4. Presence of disseminated intravascular coagulation;\n5. Presence of severe autoimmune diseases or immunodeficiency disorders;\n6. Presence of active graft-versus-host disease requiring ongoing systemic treatment;\n7. Subjects currently receiving systemic steroid or other immunosuppressive therapy prior to screening, and who are determined by the investigator to require long-term use of such therapy after enrollment (excluding inhaled or topical use);\n8. Other severe medical conditions deemed inappropriate for enrollment by the investigator (e.g., uncontrolled hypertension or diabetes, severe renal insufficiency, severe pulmonary dysfunction, etc.);\n9. Active HBV or HCV infection (HBV-DNA positive or HCV-RNA positive), HIV-positive, or positive syphilis test results;\n10. Other severe or persistent active infections;\n11. Adverse events related to systemic immunotherapy (including other investigational drugs or medical device interventions) prior to screening have not yet decreased to Grade 1 severity or returned to baseline status;\n12. Discontinuation of immunosuppressive agents for less than 2 weeks;\n13. History of allergy to any component of the cellular product;\n14. Vaccination or any surgical procedure within 4 weeks prior to screening;\n15. Other conditions deemed by the investigator to potentially increase the risk to the subject or interfere with trial results.","14 Years",{"count":719,"type":21},9,[24],"QH101 is an allogeneic TCR-enhanced Vδ2 T cell therapy product engineered to express BTN protein-specific binding elements on the cell surface. This innovative approach harnesses the natural cytotoxic capabilities of Vδ2 T cells while augmenting their ability to recognize BTN proteins, thereby significantly improving tumor cell elimination efficiency. Notably, QH101 is designed without co-stimulatory signal domains or the CD3ζ domain, which prevents T cell exhaustion from overactivation and effectively enhances in vivo persistence.\n\nPatients with R\u002FR AML face particularly poor prognoses, with conventional chemotherapy and targeted therapies achieving suboptimal complete remission rates and long-term survival below 10%. Similarly, R\u002FR MDS patients typically demonstrate median overall survival of less than one year (with TP53-mutated cases showing even poorer outcomes of 3-6 months), making clinical trial participation the most viable therapeutic option.\n\nThe development of effective treatments for R\u002FR AML\u002FMDS presents significant challenges due to:1)The paucity of disease-specific molecular targets;2)The slow progress in drug development. Allogeneic γδ T-cell therapy featuring enhanced TCR functionality and multi-mechanism tumoricidal activity represents a promising investigational approach for addressing R\u002FR AMLMDS. This innovative strategy combines the advantages of: 1)Improved target recognition through TCR enhancement; 2)Multi-faceted tumor-killing mechanisms; 3)Potential for better safety and persistence profiles.",[28,27],[724,725,726,727,728],"TCR","BTN","γδT","allogeneic","cell therapy","2025-08-12",{"date":731,"type":53},"2025-08-20",{"date":733,"type":21},"2025-08-15",{"date":735,"type":21},"2027-12-31",{"name":737,"class":738},"Anhui Provincial Hospital","OTHER_GOV",{"id":740,"slug":741,"hasResults":12,"nctId":742,"briefTitle":743,"officialTitle":744,"acronym":4,"eligibilityCriteria":745,"healthyVolunteers":12,"sex":17,"minAge":280,"maxAge":4,"enrollmentInfo":746,"targetDuration":4,"studyType":73,"phases":4,"briefSummary":747,"conditions":748,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":750,"lastUpdatePostDateStruct":751,"startDateStruct":753,"completionDateStruct":755,"leadSponsor":757,"locationsCount":137},"100337994","frailty-phenotype-assessments-to-optimize-treatment-strategies-for-older-patients-with-hematologic-malignancies-100337994","NCT03680677","Frailty Phenotype Assessments to Optimize Treatment Strategies for Older Patients With Hematologic Malignancies","Prospective Analysis of Frailty Phenotype Assessments to Optimize Treatment Strategies for Older Patients With Hematologic Malignancies","Eligibility Criteria Arm A:\n\n* Age 60 years or older.\n* New diagnosis of Acute Leukemia or MDS, or suspected diagnosis.\n* Able to consent to the study.\n\nEligibility Criteria Arm B:\n\n* Age 60 years or older with a hematologic malignancy.\n* Plan to undergo an allogeneic blood or marrow transplantation or CAR T-cell therapy.\n* Able to consent to the study.",{"count":543,"type":21},"The purpose of this research study is to determine if frailty assessments can be used to predict how well patients aged 60 years and older will do after chemotherapy, CAR T-cell therapy, or allogeneic stem cell transplant.",[749,28],"Leukemia, Acute","2025-07-29",{"date":752,"type":53},"2025-07-31",{"date":754,"type":53},"2018-09-21",{"date":756,"type":21},"2026-11",{"name":758,"class":103},"Abramson Cancer Center at Penn Medicine"]