[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"severe-aplastic-anemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:severe-aplastic-anemia":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,17,0,[8,47,71,98,126,156,193,216,243,264,291,319,351,374,397,422,447],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":31,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100299122","phase-1-unrelated-umbilical-cord-blood-transplantation-for-severe-aplastic-anemia-and-hypo-plastic-mds-using-cordintm-umbilical-cord-blood-derived-ex-vivo-expanded-stem-and-progenitor-cells-to-expedite-engraftment-and-improve-transplant-outcome-100299122",false,"NCT03173937","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome","Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002F uL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions).\n\nOR\n\nHistory of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C 5% myeloblasts and \\\u003C 30% of cellularity in the bone marrow on screening morphologic analysis.\n\n-Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n\nIdentification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>=5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>=9\u002F10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn(TM) unit has been rejected or b) umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the CordIn(TM) unit has been rejected.\n\n-Availability of at least one \\>=4\u002F8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).\n\nThe cord blood unit must contain a minimum TNC of at least 1.8 x 10\\^9 and at least 1.5x10\\^7\u002Fkg TNC and at least 8 x 10\\^6 CD34+ cells (all doses prior to thawing).\n\nException: Cord units containing at least 8 x 10\\^6 CD34+ cells but less than 1.8 x 10\\^9 TNC may be eligible for use on this trial if\n\n1. the pre-expansion CD34\u002Fkg recipient cell number is at least 1.5 x 10\\^5 \u002Fkg\n\n   AND\n2. approval for use of this cord unit for expansion is granted by Gamida Cell.\n3. the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.\n\n   * The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.\n   * Ages 4-60 years inclusive.\n   * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C 40% (evaluated by ECHO).\n* Transaminases \\> 5x upper limit of normal.\n* Serum bilirubin \\>4 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n* Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.\n* Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.","ALL","4 Years","75 Years",{"count":20,"type":21},37,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","Background:\n\nSevere aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective.\n\nObjective:\n\nTo test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.\n\nEligibility:\n\nRecipients ages 4-60 with SAA or MDS\n\nDonors ages 4-75\n\nDesign:\n\nRecipients will be screened with:\n\n* Blood, lung, and heart tests\n* Bone marrow biopsy\n* CT scan\n\nRecipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose.\n\nRecipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:\n\n* First 3-4 months: 1-2 times weekly\n* Then every 6 months for 5 years\n\nDonors will be screened with:\n\n* Physical exam\n* Medical history\n* Blood tests\n\nDonors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein.\n\nDonors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.",[28,29,30],"Severe Aplastic Anemia","Hypo-Plastic MDS","Myelodysplastic Syndrome (MDS)",[32,33],"Haploidentical","Nonmyeloablative","RECRUITING","2026-07-01",{"date":37,"type":38},"2026-07-02","ACTUAL",{"date":40,"type":38},"2017-06-13",{"date":42,"type":21},"2032-03-01",{"name":44,"class":45},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":57,"briefSummary":58,"conditions":59,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":46},"100389867","phase-2-tcr-alpha-beta-t-cell-depleted-haploidentical-hct-in-the-treatment-of-non-malignant-hematological-disorders-in-children-100389867","NCT04356469","TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children","Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children","Inclusion Criteria:\n\n1. Severe sickle cell disease (HbSS, HbSC, HbSB0, HbSB+, HbSD, HbSE) with at least one of the following criteria:\n\n   1. Cerebrovascular accident lasting longer than 24 hours\n   2. Impaired neuropsychological function with abnormal brain MRI\u002FMRA\n   3. Patients with frequent (≥ 3 per year for preceding 2 years) painful vaso-occlusive episodes\n   4. Recurrent (≥ 3 in lifetime) acute chest syndrome events which have necessitated erythrocyte transfusion therapy\n   5. Any combination of ≥ 3 acute chest syndrome episodes and vaso-occlusive pain episodes yearly for 3 years and have failed treatment with hydroxyurea (HU) (at least 6 months on maximum tolerated dose) or who are intolerant to HU therapy\n2. Thalassemia major with at least one of the following criteria:\n\n   1. Transfusion dependency defined as receiving 8 or more transfusions per year\n   2. Thalassemia diagnosis documented by clinical assessment, laboratory evidence with microcytic anemia and absence of HbA (\\\u003C 10%) on electrophoresis and or confirmation by DNA analysis of alpha and beta gene loci\n   3. Genotypically proven thalassemia major for children \\\u003C 2 years of age even in the absence of transfusion dependency\n   4. Lucarelli class 1 or 2 risk status (i.e. with only 0-2 of the following factors: hepatomegaly, portal fibrosis, or poor response to chelation therapy)\n3. Bone marrow failure syndromes and autoimmune cytopenias:\n\n   1. Severe Aplastic Anemia refractory to immunosuppressive therapy\n   2. Diamond Blackfan Anemia refractory to conventional therapy\n   3. Inherited Bone Marrow Failure Syndromes such as Fanconi anemia and Shwachman-Diamond syndrome with progressive marrow failure (without cytogenetic evidence of MDS\u002FAML)\n   4. Severe Congenital Neutropenia\n   5. Congenital Amegakaryocytic Thrombocytopenia\n   6. Glanzmann Thrombasthenia\n   7. Autoimmune Cytopenias refractory to conventional treatment (including Pure red cell aplasia, Evan's syndrome, Immune thrombocytopenia, autoimmune hemolytic anemia)\n   8. Other marrow failure disorders not otherwise specified\n\nInclusion Criteria:\n\n1. Patient has a suitable genotypic identical match of 5\u002F10. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1 and HLA-DQB1.\n2. Patients must have adequate organ function measured by:\n\n   1. Cardiac: asymptomatic or if symptomatic then LVEF at rest must be ≥ 40% or SF ≥ 26%\n   2. Pulmonary: asymptomatic or if symptomatic DLCO ≥ 40% of predicted (corrected for hemoglobin) or pulse oximetry ≥ 92% on room air if the patient is unable to perform pulmonary function testing.\n   3. Renal: Creatinine clearance (CrCl) or glomerular filtration rate (GFR) must be \\> 50 mL\u002Fmin\u002F1.73 m2.\n   4. Hepatic: Serum conjugated (direct) bilirubin \\\u003C 2.0 x ULN for age as per local laboratory unless attributable to Gilbert's syndrome; AST and ALT \\\u003C 5.0 x ULN for age as per local laboratory. Patients with hyperbilirubinemia as a consequence of hyperhemolysis, or a profound change in serum hemoglobin post blood transfusion, are not excluded.\n   5. Karnofsky or Lansky (age-dependent) performance score ≥ 50\n3. Signed written informed consent\n\nExclusion Criteria:\n\n1. Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donors are not excluded.\n2. Pregnant or breastfeeding females.\n3. Patient has HIV or uncontrolled fungal, bacterial or viral infections.\n4. Patient has received prior solid organ transplant.\n5. Patient has active GVHD (\\> grade II) or chronic extensive GVHD due to a previous allograft at the time of inclusion.\n6. For patients with hemoglobinopathy, liver biopsy is necessary if the patient has received chronic transfusions for over a year and has two ferritin levels of ≥ 1000 ng\u002Fml. Patients with cirrhosis, extensive bridging hepatic fibrosis, or active hepatitis are excluded from enrollment.","0 Years","21 Years",{"count":5,"type":21},[25],"This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.",[60,28,61],"Hemoglobinopathy (Disorder)","Bone Marrow Failure Syndrome","2026-06-29",{"date":35,"type":38},{"date":65,"type":38},"2020-07-22",{"date":67,"type":21},"2027-06-30",{"name":69,"class":70},"Johns Hopkins All Children's Hospital","OTHER",{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":4,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":78,"enrollmentInfo":79,"targetDuration":4,"studyType":22,"phases":81,"briefSummary":82,"conditions":83,"keywords":85,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":97},"100146179","phase-2-allogeneic-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-and-other-bone-marrow-failure-syndromes-using-g-csf-mobilized-cd34-selected-hematopoietic-precursor-cells-co-infused-with-a-reduced-dose-of-non-mobilized-donor-t-cells-100146179","NCT01174108","Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells","Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-Cells","* INCLUSION CRITERIA:\n* Recipient:\n\n  * Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional BMT may delay or prevent such treatment:\n\n    * 1\\) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and\u002For cytopenia, and\u002For transfusion dependence and\u002For recurrent and debilitating hemolytic crisis\n    * 2\\) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA \\[acquired or congenital\\]) with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count \\\u003C=1000 cells\u002FuL or for patients receiving granulocyte transfusions, absolute neutrophil count \\\u003C=1000 cells\u002F uL before beginning granulocyte transfusions). in newly diagnosed patients and\u002For in patients who have failed immunosuppressive therapy.\n    * 3\\) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and\u002For neutropenia.\n  * Ages 4 to 80 (both inclusive), and weight \\>15 kg\n  * Availability of HLA identical or single HLA locus mismatched family donor or 10\u002F10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ).\n  * 9\u002F10 donors where all the HLA sequences have the same antigen\u002Fpeptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g groups can be considered acceptable due to the exact matching which exists in the binding domains.\n  * Telomere Length Testing\n  * Germline\u002FInherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exist, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed elsewhere prior to enrolling on 04-H-0012.\n\nEXCLUSION CRITERIA:\n\n\\- Recipient: any of the following\n\n* Major anticipated illness or organ failure incompatible with survival from PBSC transplant\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% predicted (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed) using DL Adj and DL\u002FVA\u002FAdj.\n* Left ventricular ejection fraction \\\u003C40% (evaluated by ECHO)\n* Serum creatinine greater than 2.5mg\u002Fdl or creatinine clearance less than 50 ml\u002Fmin by 24 hr urine collection\n* Serum bilirubin greater than 4 mg\u002Fdl, transaminases greater than 5 times the upper limit of normal\n* Pregnant or lactating\n* Fanconi s anemia (test to be performed at a CLIA-certified laboratory)\n* ECOG performance status of 3 or more (See NIH Bone \\& Marrow Transplant Consortium Supportive Care Guidelines for HSCT Recipients or Institutional Guidelines for bone and marrow transplants)\n* Other malignant diseases liable to relapse or progress within 5 years, with the exception of a separate hematologic malignancy where allogeneic stem cell transplant has been shown to be potentially curative.\n* Presence of an active infection not adequately responding to appropriate therapy.\n* Inability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects age 8 -17 years with formal consent being obtained from parents or legal guardian.\n\nINCLUSION CRITERIA:\n\n-Related Donor:\n\n* Related donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood samples for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all related donors, but study participation is not required for a donor to make a stem cell donation, so it is possible that not all related donors will enroll onto this study\n* Age greater than or equal to 4 and less than or equal to 80 years old\n\nEXCLUSION CRITERIA:\n\n-Related Donor: None\n\nINCLUSION CRITERIA \\& EXCLUSION CRITERIA: Unrelated Donor\n\n\\- The NMDP unrelated donor inclusion criteria will be used as outlined in document (http:\u002F\u002Fbethematch.org\u002FWorkArea\u002FDownloadAsset.aspx?id=1960). Donor eligibility will be completed per NMDP standards and in accordance with most recent and stringent FDA guidelines.","80 Years",{"count":80,"type":21},120,[25],"Background:\n\n* Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications.\n* By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications.\n\nObjectives:\n\n\\- To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient.\n\nEligibility:\n\n* Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants.\n* Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program.\n\nDesign:\n\n* All participants will be screened with a physical examination and medical history.\n* DONORS:\n* Donors will undergo an initial apheresis procedure to donate white blood cells.\n* After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood.\n* After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood.\n* RECIPIENTS:\n* Recipients will provide an initial donation of white blood cells to be used for research purposes only.\n* From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant.\n* After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion.\n* After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant.\n* Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.",[28,84],"MDS (Myelodysplastic Syndrome)",[30,28,86,87,88],"Pure Red Cell Aplasia","Paroxysmal Nocturnal Hemoglobinuria (PNH)","Miltenyi CD34 Reagent System","2026-06-27",{"date":91,"type":38},"2026-06-30",{"date":93,"type":38},"2010-12-10",{"date":95,"type":21},"2028-06-30",{"name":44,"class":45},2,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":4,"eligibilityCriteria":104,"healthyVolunteers":11,"sex":16,"minAge":105,"maxAge":106,"enrollmentInfo":107,"targetDuration":4,"studyType":109,"phases":4,"briefSummary":110,"conditions":111,"keywords":114,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":118,"lastUpdatePostDateStruct":119,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":46},"100440193","natural-history-of-acquired-and-inherited-bone-marrow-failure-syndromes-100440193","NCT05012111","Natural History of Acquired and Inherited Bone Marrow Failure Syndromes","The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes","* INCLUSION CRITERIA:\n\nTo be eligible to participate in this study, an individual must meet all of the following criteria. Subjects and their family members who undergo screening but ultimately do not meet criteria for cohorts 1-5 will be removed from the study. Subjects may forgo screening and sign directly onto cohorts 1-5 if they meet criteria based on either prior NIH testing or external examinations. Family members will only be asked to be screened for participation onto this study after confirmation of eligibility by an affected participant.\n\nCohorts 1-4\n\n* Age \\>=2 years\n* Diagnosis of acquired or inherited bone marrow failure or ineffective hematopoiesis or TBD (see below for cohort 3 specific criteria)\n* Ability and willingness to come to the NIH CC for consultation and testing\n* Ability of subject or Legally Authorized Representative (LAR) to understand the investigational nature of the protocol and their willingness to sign a written informed consent document.\n* For cohort 3 - TBD:\n\nPresence of a pathogenic, likely pathogenic, or known family mutation in a telomere maintenance gene\n\nOR\n\nIf mutation negative or VUS, telomere length \\\u003C10th percentile in lymphocytes with at least two clinical features: 1) cytopenia (Hb \\\u003C10g\u002FdL or ANC \\\u003C1.5x10\\^9 or platelets \\\u003C100), 2) documented liver fibrosis by histology OR abnormal liver US \u002F fibro scan consistent with fatty liver or fibrosis), 3) documented pulmonary fibrosis by histology \u002F radiology OR PFTs showing FEV1 \\\u003C80% or DLCO \\\u003C60%, 4) family history of: marrow failure, myeloid malignancy, liver fibrosis or lung fibrosis.\n\nCohort 5\n\n* Age \\>= 2 years\n* First degree family member with a known or suspected inherited bone marrow failure syndrome from a patient enrolled on this or another NIH protocol as determined by a PI or AI\n* Ability and willingness to safely provide blood, buccal swab, or fibroblasts for testing as stated by subject\n* Ability of subject or Legally Authorized Representative (LAR) to understand the investigational nature of the protocol and the willingness to sign a written informed consent document.\n\nEXCLUSION CRITERIA:\n\n-None","2 Years","99 Years",{"count":108,"type":21},1000,"OBSERVATIONAL","Background:\n\nBone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases.\n\nObjective:\n\nTo follow people diagnosed with acquired or inherited bone marrow failure disease and study the long-term effects of the disease and its treatments on organ function.\n\nEligibility:\n\nPeople aged 2 years and older who have been diagnosed with acquired or inherited bone marrow failure or Telomere Biology Disorder. First degree family members may also be able to take part in the study.\n\nDesign:\n\nParticipants will be screened with a medical history, physical exam, and blood tests. They may have a bone marrow biopsy and aspiration. For this, a large needle will be inserted in the hip through a small cut. Marrow will be drawn from the bone. A small piece of bone may be removed.\n\nParticipants may also be screened with some of the following:\n\nCheek swab or hair follicle sample\n\nSkin biopsy\n\nUrine or saliva sample\n\nEvaluation by disease specialists (e.g., lung, liver, heart)\n\nImaging scan of the chest\n\nLiver ultrasounds\n\nSix-Minute Walk Test\n\nLung function test\n\nParticipants will be put into groups based on their disease. They will have visits every 1 to 3 years. At visits, they may repeat some screening tests. They may fill out yearly surveys about their medicines, transfusions, pregnancy, bleeding, and so on. They may have other specialized procedures, such as imaging scans and ultrasounds.\n\nParticipation will last for up to 20 years.\n\n...",[28,112,113],"Telomere Biology Disorders","Inherited Bone Marrow Failure Syndromes",[115,116,117],"Aplastic Anemia","Telomere","Natural History","2026-06-23",{"date":120,"type":38},"2026-06-24",{"date":122,"type":38},"2021-10-25",{"date":124,"type":21},"2041-02-04",{"name":44,"class":45},{"id":127,"slug":128,"hasResults":11,"nctId":129,"briefTitle":130,"officialTitle":130,"acronym":4,"eligibilityCriteria":131,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":18,"enrollmentInfo":132,"targetDuration":4,"studyType":22,"phases":134,"briefSummary":135,"conditions":136,"keywords":140,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":154,"locationsCount":46},"100547786","phase-2-mt2023-20-hematopoietic-cell-transplant-with-reduced-intensity-conditioning-and-post-transplant-cyclophosphamide-for-severe-aplastic-anemia-and-other-forms-of-acquired-bone-marrow-failure-100547786","NCT06412497","MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.","Inclusion Criteria:\n\n* Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL AND bone marrow cellularity \\\u003C50% (with \\\u003C 30% residual hematopoietic cells)\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%), without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic SAA with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \\>\u002F= 10%\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%) without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Adequate organ function within 30 days of conditioning regimen\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment\n* Uncontrolled infection\n* Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy\n* Known allergy to any of the study components\n* Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol\n* Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)\n* Advanced myelodysplastic syndrome (MDS; BM blasts \\>5%) or acute myeloid leukemia\n* Psychiatric illness\u002Fsocial situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study",{"count":133,"type":21},60,[25],"A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and\u002For clonal hematopoiesis.",[28,137,138,139],"Acquired Amegakaryocytic Thrombocytopenia","Acquired Pure Red Cell Aplasia","Paroxysmal Nocturnal Hemoglobinuria",[141,142,143,144,145,146],"HCT","RIC","SAA","PTCy","aAT","aPRCA","2026-06-02",{"date":149,"type":38},"2026-06-03",{"date":151,"type":38},"2024-06-05",{"date":153,"type":21},"2036-05-01",{"name":155,"class":70},"Masonic Cancer Center, University of Minnesota",{"id":157,"slug":158,"hasResults":11,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":4,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":163,"targetDuration":4,"studyType":109,"phases":4,"briefSummary":165,"conditions":166,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":183,"startDateStruct":185,"completionDateStruct":187,"leadSponsor":189,"locationsCount":192},"100159702","a-multicenter-access-and-distribution-protocol-for-unlicensed-cryopreserved-cord-blood-units-cbus-100159702","NCT01351545","A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)","A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications","Inclusion Criteria:\n\n* Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment\n* Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment\n* Pediatric and adult patients of any age\n\nExclusion Criteria:\n\n* Patients who are receiving only licensed CBUs\n* Cord blood transplant recipients at international transplant centers\n* Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)\n* Patients whose selected unlicensed CBU(s) will be more than minimally manipulated",{"count":164,"type":21},99999,"This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.",[167,168,169,170,171,172,173,174,175,176,177,178,179,180,181,182,28],"Hematologic Malignancies","Inherited Disorders of Metabolism","Inherited Abnormalities of Platelets","Histiocytic Disorders","Acute Myelogenous Leukemia (AML or ANLL)","Acute Lymphoblastic Leukemia (ALL)","Other Acute Leukemia","Chronic Myelogenous Leukemia (CML)","Myelodysplastic (MDS) \u002F Myeloproliferative (MPN) Diseases","Other Leukemia","Hodgkin Lymphoma","Non-hodgkin Lymphoma","Multiple Myeloma\u002F Plasma Cell Disorder (PCD)","Inherited Abnormalities of Erythrocyte Differentiation or Function","Disorders of the Immune System","Autoimmune Diseases",{"date":184,"type":38},"2026-06-04",{"date":186,"type":4},"2011-10",{"date":188,"type":21},"2041-10",{"name":190,"class":191},"Center for International Blood and Marrow Transplant Research","NETWORK",142,{"id":194,"slug":195,"hasResults":11,"nctId":196,"briefTitle":197,"officialTitle":198,"acronym":4,"eligibilityCriteria":199,"healthyVolunteers":11,"sex":16,"minAge":200,"maxAge":18,"enrollmentInfo":201,"targetDuration":4,"studyType":22,"phases":203,"briefSummary":204,"conditions":205,"keywords":206,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":207,"lastUpdatePostDateStruct":208,"startDateStruct":210,"completionDateStruct":212,"leadSponsor":214,"locationsCount":97},"100272624","phase-2-haploidentical-bone-marrow-transplant-with-post-transplant-cyclophosphamide-for-patients-with-severe-aplastic-anemia-100272624","NCT02828592","Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia","A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor","Inclusion Criteria:\n\n* Availability of 3\u002F6 - 5\u002F6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction\n* Age \\\u003C= 65 years for previously treated and \\\u003C= 75 years for previously treated patients\n* KPS \\>= 70%\n* Aplastic Anemia that meets the following criteria:\n\nPeripheral Blood (must fulfill 2 of 3):\n\n* \\\u003C500 PMN\u002Fmm3\n* \\\u003C20,000 platelets\n* absolute reticulocyte count \\\u003C40,000\u002FmicroL\n\nBone Marrow (must be either):\n\n* markedly hypocellular (\\\u003C25% of normal cellularity)\n* moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above\n\nExclusion Criteria:\n\n* poor cardiac function (LVEF \\\u003C40%)\n* poor pulmonary function (FEV1 \\& FVC \\\u003C50% predicted)\n* poor liver function (bili \\>= 2mg\u002FdL)\n* poor renal function (creatinine \\>= 2.0mg\u002FdL or creatinine clearance \\\u003C40mL\u002Fmin)\n* prior allogeneic transplant","1 Year",{"count":202,"type":21},20,[25],"Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1\u002F3 of patients are cured, 1\u002F3 are dependent on long term immunosuppression, and the other 1\u002F3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.",[28],[143],"2026-04-15",{"date":209,"type":38},"2026-04-20",{"date":211,"type":38},"2016-09-09",{"date":213,"type":21},"2028-08-31",{"name":215,"class":70},"Northside Hospital, Inc.",{"id":217,"slug":218,"hasResults":219,"nctId":220,"briefTitle":221,"officialTitle":221,"acronym":4,"eligibilityCriteria":222,"healthyVolunteers":11,"sex":16,"minAge":223,"maxAge":106,"enrollmentInfo":224,"targetDuration":4,"studyType":22,"phases":226,"briefSummary":227,"conditions":228,"keywords":229,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":235,"lastUpdatePostDateStruct":236,"startDateStruct":238,"completionDateStruct":240,"leadSponsor":242,"locationsCount":46},"100385908","phase-2-early-initiation-of-oral-therapy-with-cyclosporine-and-eltrombopag-for-treatment-naive-severe-aplastic-anemia-saa-100385908",true,"NCT04304820","Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)","* INCLUSION CRITERIA:\n\n  1. Age \\>= 3 years old\n  2. Weight \\>12Kg\n  3. Severe aplastic anemia:\n* Bone marrow cellularity \\\u003C30% (excluding lymphocytes) AND At least two of the following:\n\n  * Absolute neutrophil count \\\u003C500\u002Fmicroliter\n  * Platelet count \\\u003C20,000\u002Fmicroliter\n  * Absolute reticulocyte count \\\u003C60,000\u002Fmicroliter\n\nEXCLUSION CRITERIA:\n\n1. Known diagnosis or high suspicion of Fanconi anemia or other constitutional marrow failure syndrome\n2. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry involving chromosome 7 or complex karyotype. Patient will not be excluded if cytogenetics are not done or are pending\n3. A course of prior immunosuppressive therapy (ATG, cyclosporine, alemtuzumab, and high dose cyclophosphamide), or eltrombopag\n4. SGOT or SGPT \\>2.5 times the upper limit of normal or total bilirubin \\>1.5 x upper limit of normal\n5. Subjects with liver cirrhosis (as determined by the investigator).\n6. Subjects with human immunodeficiency virus (HIV) who are not receiving antiretroviral therapy, have detectable HIV RNA viral load and have CD4 cell count \\\u003C200\u002Fmicroliter, or are on anti-retroviral therapy that interacts with the study drugs. subjects will not be excluded if HIV testing is pending or unavailable.\n7. Glomerular filtration rate (GFR) \\\u003C40 mL\u002Fmin\u002F1.73m\\^2\n8. Hypersensitivity to EPAG or its components\n9. Infection not adequately responding to appropriate therapy\n10. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely\n11. Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible\n12. Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.\n13. Inability to swallow\n14. Unable to participate in audio\u002Fvideo telecommunication\n15. Inability to ship the study drug to participant\n16. History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study such as uncontrolled or significant cardiac disease, including any of the following: Recent myocardial infarction (within last 6 months), uncontrolled congestive heart failure, unstable angina (within last 6 months), clinically significant (symptomatic) cardiac arrhythmias (e.g., sustained ventricular tachycardia, and clinically significant second or third degree AV block without a pacemaker.), long QT syndrome, family history of idiopathic sudden death, congenital long QT syndrome or additional risk factors for cardiac repolarization abnormality, as determined by the investigator.\n17. Impaired cardiac function, such as: Corrected QTc \\>450 msec using Fridericia correction (QTcF) on the screening ECG (using triplicate ECGs), other clinically significant cardiovascular disease (e.g., uncontrolled hypertension, history of labile hypertension), history of known structural abnormalities (e.g. cardiomyopathy).\n18. Concurrent participation in an investigational study within 30 days prior to enrollment or within 5-half-lives of the investigational product, whichever is longer. Note: parallel enrollment in a disease registry is permitted.\n19. Known thrombophilic risk factors. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.\n20. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study treatment. Basic contraception methods include:\n\n    * Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception\n    * Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment\n    * Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject\n    * Barrier methods of contraception: Condom or Occlusive cap. For the UK: with spermicidal foam\u002Fgel\u002Ffilm\u002Fcream\u002F vaginal suppository\n    * Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \\\u003C1%), for example hormone vaginal ring or transdermal hormone contraception.\n\n      * In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.\n21. Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1\n22. Sexually active males unless they use a condom during intercourse while taking the drug during treatment, and for 7 days after stopping treatment (and for an additional 12 weeks \\[for genotoxic compounds\\]) and should not father a child in this period. A condom is required to be used also by vasectomized men as well as during intercourse with a male partner in order to prevent delivery of the drug via semen.\n\nINCLUSION OF PREGNANT WOMEN, FETUSES OR NEONATES:\n\nThe protocol does not intentionally enroll pregnant women due to unknown fetal risk with eltrombopag. However, if a patient gets pregnant during the trial participation period, she may remain on study for non-invasive safety and outcomes follow-up.","3 Years",{"count":225,"type":21},80,[25],"Background:\n\nSevere aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells. Standard treatment for SSA is a combination of 3 drugs (Cyclosporine \\[CsA\\], Eltrombopag \\[EPAG\\], and horse anti-thymocyte globulin \\[h-ATG\\]). Researchers want to see if starting people at a lower dose of CsA with EPAG before giving them h-ATG is helpful.\n\nObjective:\n\nTo learn if early initiation of oral therapy with CsA and EPAG is safe and effective in people who have SAA and have not been treated with a course of immunosuppressive therapy and EPAG.\n\nEligibility:\n\nPeople ages 3 and older with SAA\n\nDesign:\n\nParticipants will be screened with:\n\n* medical history\n* physical exam\n* electrocardiogram\n* blood tests\n* family history\n* bone marrow biopsy\n* current medicines.\n\nParticipants may be screened remotely via telephone conference.\n\nParticipants will take a lower oral dose of CsA and EPAG. They will take CsA twice a day for 6 months. They will take EPAG for 6 months. Those who cannot visit the NIH Clinical Center within 72 hours will start taking the drugs at home. They will have weekly telephone calls with NIH staff until they visit the Clinical Center.\n\nParticipants may get h-ATG at the Clinical Center for 4 days. For this, they will have a central line placed. It is a plastic tube inserted into a neck, chest, or arm vein.\n\nParticipants will repeat most screening tests throughout the study.\n\nParticipants will have follow-up visits at the Clinical Center at 3 months, 6 months, and annually for 5 years after the start of the study....",[28],[230,231,232,233,234],"Immunosuppression","T-cells","Hematopoiesis","Autoimmunity","Thrombocytopenia","2026-02-17",{"date":237,"type":38},"2026-03-09",{"date":239,"type":38},"2020-05-07",{"date":241,"type":21},"2033-12-18",{"name":44,"class":45},{"id":244,"slug":245,"hasResults":11,"nctId":246,"briefTitle":247,"officialTitle":248,"acronym":249,"eligibilityCriteria":250,"healthyVolunteers":11,"sex":16,"minAge":223,"maxAge":18,"enrollmentInfo":251,"targetDuration":4,"studyType":22,"phases":252,"briefSummary":253,"conditions":254,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":255,"lastUpdatePostDateStruct":256,"startDateStruct":257,"completionDateStruct":259,"leadSponsor":261,"locationsCount":263},"100555865","phase-2-clinical-trial-of-upfront-haploidentical-or-unrelated-donor-bmt-to-restore-normal-hematopoiesis-in-aplastic-anemia-100555865","NCT06517641","Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia","A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Haploidentical Related, Partially HLA-Mismatched, or Matched Unrelated Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia","BMT CTN CureAA","Inclusion Criteria:\n\n1. Age 3 years to 75 years\n2. Confirmed diagnosis of acquired SAA defined as:\n\n   a. Bone marrow cellularity \\\u003C 25% or variable marrow cellularity but with \\\u003C 30% residual hematopoietic cells deemed HYPOcellular for age AND b. Two (2) out of 3 of the following (in peripheral blood). i. Neutrophils \\\u003C 0.5 x109\u002FL ii. Platelets \\\u003C 20 x109\u002FL iii. Reticulocyte count \\\u003C 20 x109\u002FL (\\\u003C 60 x 109\u002FL using an automated analysis)\n3. No suitable fully matched related donor as per Investigator's discretion (6\u002F6 match for HLA A and B at intermediate or high-resolution and DRB1 at high-resolution using deoxyribonucleic acid \\[DNA\\]-based typing) available.\n4. Available donor as defined in the protocol.\n5. Participant and\u002For legal guardian must sign informed consent.\n6. Adequate organ function defined by institutional transplant standards or defined as below:\n\n   1. Cardiac: Left ventricular ejection fraction (LVEF) at rest \\> 40% with no clinical signs of cardiac failure. For participants aged \\\u003C 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or multigated acquisition (MUGA) may be substituted for LVEF.\n   2. Hepatic: Total bilirubin \\\u003C 2.0 mg\u002FdL unless Gilbert's disease is present\n   3. Renal: For participants \\> 13.0 years of age at the time of enrollment: estimated creatinine clearance (CrCl) \\> 60 mL\u002Fminute (per institutional standard). For participants \\\u003C 13.0 years of age at enrollment: glomerular filtration rate (GFR) estimated by the updated Schwartz formula ≥ 90 mL\u002Fmin\u002F1.73 m2. If the estimated GFR is \\\u003C 90 mL\u002Fmin\u002F1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be \\> 50 mL\u002Fmin\u002F1.73 m2.\n   4. Pulmonary:\n\n   i. For participants \\> 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO, corrected\u002Fadjusted for hemoglobin \\[Hb\\]) \\> 50%, or Spirometry with forced expiratory volume 1 (FEV1) \\> 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) \\> 50% predicted.\n\n   ii. For participants \\\u003C 13.0 years of age unable to perform pulmonary function tests (PFTs) due to age or developmental ability: (1) no evidence of dyspnea at rest and (2) no need for supplemental oxygen and (3) O2 saturation \\> 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care \\[e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%\\]).\n7. Karnofsky or Lansky performance status ≥ 60%.\n8. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence.\n\nExclusion Criteria:\n\n1. Inherited bone marrow failure syndromes such as Fanconi anemia and short telomere syndromes must be ruled out according to center standards. It is recommended that functional testing for Fanconi Anemia (di-epoxybutane \\[DEB\\] chromosomal breakage analysis) and telomere length assessment be performed. If available, genetic panels for inherited bone marrow failure syndromes can be considered as an alternative to functional testing.\n2. Clonal cytogenetic abnormalities consistent with pre-MDS or MDS on marrow examination (e.g., monosomy 7 and other MDS-defining changes per recent pathology guidelines).\n3. Formal diagnosis of MDS by World Health Organization (WHO) 2022 or International Consensus Classification (ICC).\n4. Recipient positive for HLA antibodies against a mismatched HLA in the selected donor determined by the presence of donor specific HLA antibodies (DSA) to any mismatched HLA allele\u002Fantigen at any of the following loci (HLA-A, -B, -C, -DRB1, DRB3, DRB4, DRB5, -DQA1, -DQB1, -DPA1, -DPB1) with median fluorescence intensity (MFI) \\>3000 by microarray-based single antigen bead testing. In patients receiving red blood cell or platelet transfusions, DSA evaluation must be performed or repeated post-transfusion and immediately prior to initiation of recipient preparative regimen to ensure there is confirmation of no DSA to the selected donor when conditioning starts.\n5. Prior desensitization attempt for HLA antibodies to chosen donor. Any intervention with the sole intent to reduce the level of HLA DSA, (e.g., plasmapheresis, intravenous immunoglobulin \\[IVIG\\], MMF, etc.) would constitute a desensitization attempt.\n6. Prior treatment for SAA (e.g., immunosuppressive therapy using ATG, calcineurin inhibitors \\[CNIs\\], thrombopoietin receptor agonists or androgens). Short courses of steroids or IVIG that were not explicitly administered for SAA therapy will be allowed.\n7. Prior allogeneic stem cell transplant.\n8. Prior solid organ transplant.\n9. Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® (Sanofi) that would prohibit use for the participant as this study requires use of the Thymoglobulin® (Sanofi) preparation of ATG.\n10. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.\n11. Female participants who are pregnant, as detected using a pregnancy test as per institutional practice, or breast-feeding.\n12. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent \\> 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years previously will not be allowed unless approved by the Protocol Chairs and\u002For Protocol Officer.\n\nOf note, participants with seropositivity for the human immunodeficiency virus (HIV) may be considered if viral load is undetectable. Similarly, carriers of hepatitis B (HepB) or hepatitis C (HepC) may not have a detectable viral load of HepB virus or HepC virus.\n\nParticipants with HIV that is well-controlled on combination antiretroviral therapy and no AIDS related complications within the past 12 months are eligible.\n\nInfections other than HIV:\n\n* Prior infections must be controlled\n* HepB participants are eligible if on effective suppressive therapy and otherwise meet inclusion\u002Fexclusion criteria\n* HepC participants are eligible if otherwise meet inclusion\u002Fexclusion criteria",{"count":133,"type":21},[25],"BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.",[28],"2026-02-12",{"date":235,"type":38},{"date":258,"type":38},"2025-05-12",{"date":260,"type":21},"2029-02-01",{"name":262,"class":70},"Medical College of Wisconsin",25,{"id":265,"slug":266,"hasResults":11,"nctId":267,"briefTitle":268,"officialTitle":269,"acronym":4,"eligibilityCriteria":270,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":271,"enrollmentInfo":272,"targetDuration":4,"studyType":22,"phases":274,"briefSummary":275,"conditions":276,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":283,"lastUpdatePostDateStruct":284,"startDateStruct":286,"completionDateStruct":288,"leadSponsor":290,"locationsCount":46},"100330256","phase-2-alphabeta-tcd-hct-in-patients-with-inherited-bmf-disorders-100330256","NCT03579875","Alpha\u002FBeta TCD HCT in Patients With Inherited BMF Disorders","MT2017-17:T Cell Receptor Alpha\u002FBeta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders","Patient Selection:\n\nInclusion Criteria:\n\nFor FA patients:\n\n* Diagnosis of Fanconi anemia\n\n  * Age \\\u003C65 years of age\n* Has one of the following risk factors:\n\n  * Severe aplastic anemia (SAA)\n  * Myelodysplastic features\n  * High risk genotype\n  * Immunodeficiency associated with history of recurrent infections\n* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \\\u003C16 years of age\n\n  * Adequate pulmonary, cardiac and liver function\n  * Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care\n\nFor TBD patients:\n\n• Diagnosis of TBD\n\n* Age \\\u003C70 years of age\n* Has one of the following risk factors:\n* Severe aplastic anemia (SAA)\n* Myelodysplastic features\n* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score\n\n  ≥ 50% for patients \\\u003C16 years of age\n* Adequate pulmonary, cardiac and liver function\n* Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration\n* Active, uncontrolled infection within 1 week prior to starting study therapy\n* Malignant solid tumor cancer within previous 2 years\n\nDonor Selection (Inclusion Criteria): meets one of the following match criteria:\n\n* an HLA-A, B, DRB1 matched sibling donor (matched sibling)\n* an HLA-A, B, DRB1 matched related donor (other than sibling)\n* a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen\n* 7-8\u002F8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.\n* Body weight of at least 40 kilograms and at least 12 years of age\n* Willing and able to undergo mobilized peripheral blood apheresis\n* In general good health as determined by the medical provider\n* Adequate organ function defined as:\n\n  * Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)\n  * Hepatic: ALT \\\u003C 2 x upper limit of normal\n  * Renal: serum creatinine \\\u003C 1.8 mg\u002Fdl\n* Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1\u002F2 Antibody, HTLVA 1\u002F2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B\n* Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start\n* Voluntary written consent (parent\u002Fguardian and minor assent, if \\\u003C 18 years) prior to the performance of any research related procedure","65 Years",{"count":273,"type":21},48,[25],"This is a phase II trial of T cell receptor alpha\u002Fbeta depletion (α\u002Fβ TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.",[277,28,278,279,280,281,282,112],"Fanconi Anemia","Myelodysplastic Syndromes","T Cell Receptor Alpha\u002FBeta Depletion","Telomere Biology Disorder","Bone Marrow Failure","Dyskeratosis Congenita","2026-01-29",{"date":285,"type":38},"2026-01-30",{"date":287,"type":38},"2018-11-13",{"date":289,"type":21},"2029-01-05",{"name":155,"class":70},{"id":292,"slug":293,"hasResults":11,"nctId":294,"briefTitle":295,"officialTitle":296,"acronym":4,"eligibilityCriteria":297,"healthyVolunteers":11,"sex":16,"minAge":298,"maxAge":4,"enrollmentInfo":299,"targetDuration":4,"studyType":22,"phases":301,"briefSummary":302,"conditions":303,"keywords":306,"overallStatus":309,"whyStopped":4,"lastUpdateSubmitDate":310,"lastUpdatePostDateStruct":311,"startDateStruct":313,"completionDateStruct":315,"leadSponsor":317,"locationsCount":4},"100615818","phase-1-safety-and-efficacy-of-the-jak1-inhibitor-combined-with-intensive-immunosuppressive-therapy-in-severe-aplastic-anemia-100615818","NCT07297550","Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia","Phase Ib\u002FII Study to Evaluate Safety and Preliminary Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia","Phase Ib:\n\nInclusion Criteria:\n\n* According to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and the Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology Guideline (Br J Haematol. 2024; 204: 784-804), the patient is diagnosed with primary acquired AA and meets the diagnostic criteria for SAA.\n* Subjects received intensive immunosuppressive therapy combined with a standard-dose TPO receptor agonist for at least 4 months but failed to respond or experienced relapse.\n* Unsuitable for or unwilling to undergo allogeneic hematopoietic stem cell transplantation and lacking other superior treatment options.\n* Age ≥ 18 years\n* ECOG performance status ≤2\n* Willing and able to comply with the requirements for this study and written informed consent.\n\nExclusion Criteria:\n\n* Presence of any other primary or secondary bone marrow failure syndrome, including but not limited to Fanconi anemia, myelodysplastic syndrome (MDS), clonal cytopenia of undetermined significance, or large granular lymphocytic leukemia.\n* Bone marrow reticulin fibrosis ≥ grade 2\n* Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50% or with evidence of active hemolysis.\n* Presence of clonal cytogenetic abnormalities characteristic of MDS, excluding +8, del(20q), and -Y.\n* Active infection within 2 weeks before first dosing of the investigational product, including bacterial, viral, or fungal infections (common cold and onychomycosis excluded). Any anti-infective treatment must be completed at least 2 weeks before dosing. History of HIV infection or HIV antibody positive at screening. Positive treponemal antibody for syphilis at screening. Active pulmonary tuberculosis, defined as evidence of active TB on chest imaging or other relevant assessments within 3 months before first dosing or during screening. Active hepatitis at screening, defined as: HBsAg positivity; or HBcAb positivity with HBV DNA ≥ 30 IU\u002FmL; or HCV antibody positivity with detectable HCV RNA.\n* Active bleeding involving the gastrointestinal tract, respiratory tract, central nervous system, or other sites.\n* History of clinically significant diseases that, in the investigator's judgment, may pose a risk to the participant's safety if enrolled in the study, or may interfere with the assessment of efficacy or safety during the study, including but not limited to: 1. Cardiovascular diseases: history within the past year of acute myocardial infarction or unstable angina, severe arrhythmias (e.g., frequent multifocal ventricular premature beats, ventricular tachycardia, ventricular fibrillation), congestive heart failure, arterial or venous thrombosis, or New York Heart Association (NYHA) class III-IV heart failure. 2. History of psychiatric disorders or presence of severe cerebrovascular disease or cognitive sequelae.\n* Treatment with mycophenolate mofetil, tacrolimus, sirolimus, cyclophosphamide, anti-CD52 monoclonal antibody, or other similar therapies within 4 weeks or 5 half-lives of the first dose of the investigational product, whichever is shorter.\n* Planned participation in another clinical trial, or prior exposure to investigational products in another clinical trial within 4 weeks or within 5 half-lives of the first dose of the investigational product, whichever is shorter.\n* Received a live attenuated vaccine within 4 weeks before the first dose of the investigational product, planned administration of a live attenuated vaccine during the study, or received a COVID-19 vaccine within 7 days before dosing.\n* Prior treatment with a JAK inhibitor.\n* Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study; female participants of childbearing potential and male participants whose partners are of childbearing potential who do not agree to use highly effective contraception throughout the study period (from signing the ICF until 6 months after the last dose of the investigational product), or those planning to donate oocytes or sperm during the study.\n* Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study.\n\nPhase II:\n\nInclusion Criteria:\n\n* According to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and the Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology Guideline (Br J Haematol. 2024; 204: 784-804), the patient is diagnosed with primary acquired AA and meets the diagnostic criteria for SAA.\n* Meets the diagnostic criteria for severe aplastic anemia (Br J Haematol. 2024; 204: 784-804), which require: 1. Bone marrow cellularity less than 25% of normal; if ≥ 25% but \\\u003C 50%, residual hematopoietic cells must be \\\u003C 30%. 2. Peripheral blood counts must meet at least two of the following three criteria: Absolute neutrophil count (ANC) \\\u003C 0.5 × 10\\^9\u002FL, Absolute reticulocyte count \\\u003C 60 × 10\\^9\u002FL, Platelet count (PLT) \\\u003C 20 × 10\\^9\u002FL\n* Unsuitable for or unwilling to undergo allogeneic hematopoietic stem cell transplantation.\n* Age ≥ 18 years\n* ECOG performance status ≤2\n* Willing and able to comply with the requirements for this study and written informed consent.\n\nExclusion Criteria:\n\n* Presence of any other primary or secondary bone marrow failure syndrome, including but not limited to Fanconi anemia, myelodysplastic syndrome (MDS), clonal cytopenia of undetermined significance, or large granular lymphocytic leukemia.\n* Bone marrow reticulin fibrosis ≥ grade 2\n* Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50% or with evidence of active hemolysis.\n* Presence of clonal cytogenetic abnormalities characteristic of MDS, excluding +8, del(20q), and -Y.\n* Participation in another clinical trial and use of investigational products or medical devices within 30 days prior to the first dose of the investigational product or within 5 half-lives of the investigational product, whichever is longer.\n* Prior use of any of the following before the first dose of the investigational product: rabbit anti-thymocyte globulin (rATG) or porcine anti-lymphocyte globulin (pALG); alemtuzumab; or medium- to high-dose cyclophosphamide (≥30-45 mg\u002Fkg\u002Fday).\n* Prior use of standard-dose cyclosporine or standard-dose TPO-RA for more than 1 month before the first dose of the investigational product.\n* Bleeding and\u002For infection that remain uncontrolled despite receiving standard therapy prior to the first dose of the investigational product.\n* Active CMV or EBV infection.\n* History of, or concurrent presence of: Positive human immunodeficiency virus (HIV) antibody test; Positive hepatitis B surface antigen (HBsAg), or positive hepatitis B core antibody (HBcAb) with HBV DNA above the assay upper limit of quantification.\n* Uncontrolled conditions prior to the first dose of the investigational product, including: 1. Hypertension: blood pressure remains uncontrolled despite lifestyle modification and treatment with two or more appropriately dosed, tolerable antihypertensive drugs (including diuretics) for more than 1 month, or requiring four or more antihypertensive drugs for effective control. 2. Severe arrhythmias, such as complete left bundle branch block, atrial fibrillation, or torsades de pointes. 3. Unstable angina. 4. Pulmonary hypertension, defined as estimated pulmonary artery systolic pressure (PASP) ≥ 60 mmHg by echocardiography. 5. Active bleeding involving the gastrointestinal tract, respiratory tract, central nervous system, or other sites.\n* Any arterial or venous thrombosis within 1 year prior to the first dose (excluding fully resolved deep or superficial venous thromboembolism), such as myocardial infarction or pulmonary embolism.\n* History of other malignancies within 5 years prior to the first dose (excluding cured early-stage tumors such as basal cell or squamous cell skin cancer, early-stage papillary thyroid cancer, superficial bladder cancer, cervical or breast carcinoma in situ, or localized prostate cancer with Gleason score \\\u003C6).\n* Pregnant or breastfeeding.\n* Presence of contraindications to rATG, pALG, or cyclosporine.\n* Prior treatment with a JAK inhibitor.\n* Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study.","18 Years",{"count":300,"type":21},42,[24,25],"This study is designed as a Phase Ib\u002FII trial. The phase Ib cohort will enroll patients with severe aplastic anemia (SAA) who have failed to respond to intensified immunosuppressive therapy. In contrast, the phase II cohort will include newly diagnosed and treatment-naïve patients with SAA. A Safety Review Committee (SRC), chaired by the principal investigators, will be established to oversee patient safety throughout the study. Suppose the Phase Ib results demonstrate acceptable safety and tolerability. In that case, the data will be submitted to the Ethics Committee for review, and, upon approval, the study will advance to Phase II.\n\nPhase Ib uses a 3+3 dose-escalation design with two cohorts: 150 mg golidocitinib orally every other day (low dose) or once daily (high dose). Phase II is a single-arm trial with Simon's two-stage optimal design.",[28,304,305],"Refractory Aplastic Anemia","Newly Diagnosed Aplastic Anemia",[307,308],"JAK1 inhibitor","Intensive immunosuppressive therapy","NOT_YET_RECRUITING","2025-12-09",{"date":312,"type":38},"2025-12-22",{"date":314,"type":21},"2025-12-15",{"date":316,"type":21},"2027-07-01",{"name":318,"class":70},"Institute of Hematology & Blood Diseases Hospital, China",{"id":320,"slug":321,"hasResults":11,"nctId":322,"briefTitle":323,"officialTitle":324,"acronym":4,"eligibilityCriteria":325,"healthyVolunteers":11,"sex":16,"minAge":326,"maxAge":327,"enrollmentInfo":328,"targetDuration":4,"studyType":22,"phases":329,"briefSummary":330,"conditions":331,"keywords":338,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":342,"lastUpdatePostDateStruct":343,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":46},"100370178","phase-2-allosct-for-malignant-and-non-malignant-hematologic-diseases-utilizing-alphabeta-t-cell-and-cd19-b-cell-depletion-100370178","NCT04099966","AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion","Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion - NYMC 588","Inclusion Criteria:\n\n1. ALL:ALL high risk including one or more of the following: (t(9;22) or 11q23 chromosomal abnormality, primary induction failure (\\\u003C15% blasts at time of registration), mixed phenotype acute leukemia (MPAL), persistent MRD (\\\u003C0.01% by flow or persistent abnormal karyotype detected by cytogenetics) or hypodiploidy (44 chromosomes)) in first remission ' ALL in second remission and beyond;\n2. AML: History of AML induction\u002Freinduction Failure (\\\u003C15% blasts at time of registration); AML in CR1 with poor cytogenetics (i.e. 12p, 5a, -7, FLT3 mutation\u002Fduplication, t(9;11) and others); AML with persistent minimal residual disease (MRD) in CR1(\\\u003C0.01% on flow or persistent abnormal karyotype detected by cytogenetics); AML CR2 or beyond; AML in refractory relapse but ≤15% bone marrow leukemia blasts; Therapy-related AML\n3. High Risk Myelodysplastic syndrome (MDS) 4 Lymphoma: Hodgkin (HL) or Non-Hodgkin (NHL): HL or NHL in induction failure; HL or NHL in PR1 or PR2 ; HL or NHL in CR2 or subsequent remission\n\n5\\. Bone marrow failure syndromes: Kostmann syndrome refractory or intolerant to granulocyte colony-33stimulating factor; Diamond-Blackfan anemia refractory or intolerant to corticosteroids and\u002For cyclosporine'; amegakaryocytic thrombocytopenia 6. Sickle Cell Disease (Homozygous Hemoglobin S Disease, or Hemoglobin S β 0\u002F+ thalassemia, or Hemoglobin SC Disease) 7. age 0-30 years 8. adequate organ function\n\nExclusion Criteria:\n\n1. Females who are pregnant or breast-feeding are not eligible.\n2. Patients with documented uncontrolled infection at the time of study entry are not eligible.\n3. Karnofsky\u002FLansky (age appropriate) Performance Score \\\u003C60\n4. Demonstrated lack of compliance with medical care\n5. Patients who have received allogeneic HSCT within 6 months, unless being done as a boost.\n6. Patients with active \\\u003CGrade 2 GVHD.","1 Day","30 Years",{"count":202,"type":21},[25],"Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α\u002Fβ CD3+\u002FCD19+ cell depletion. All other treatment is standard of care.",[332,28,178,177,333,334,335,336,337],"Acute Leukemia","Kostmann","Diamond Blackfan Anemia","Amegakaryocytic Thrombocytopenia","Sickle Cell Disease","Beta-Thalassemia",[339,340,341],"allogeneic stem cell transplantation","t-cell depletion","alpha beta cell depletion","2025-08-07",{"date":344,"type":38},"2025-08-08",{"date":346,"type":38},"2021-04-01",{"date":348,"type":21},"2027-12-31",{"name":350,"class":70},"Mitchell Cairo",{"id":352,"slug":353,"hasResults":11,"nctId":354,"briefTitle":355,"officialTitle":356,"acronym":4,"eligibilityCriteria":357,"healthyVolunteers":11,"sex":16,"minAge":298,"maxAge":18,"enrollmentInfo":358,"targetDuration":4,"studyType":22,"phases":360,"briefSummary":361,"conditions":362,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":366,"lastUpdatePostDateStruct":367,"startDateStruct":369,"completionDateStruct":371,"leadSponsor":373,"locationsCount":46},"100563940","phase-1-cd7-car-t-cells-rd13-02-in-the-treatment-of-relapsedrefractory-severe-aplastic-anemia-100563940","NCT06622694","CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed\u002FRefractory Severe Aplastic Anemia","A Clinical Study of Safety, Efficacy, and Pharmacokinetics of Universal CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed\u002FRefractory Severe Aplastic Anemia","Inclusion Criteria:\n\n* Willing and able to provide written informed consent.\n* Age ≥18 years and ≤75 years.\n* Diagnosed with severe aplastic anemia: Diagnostic criteria refer to the UK Hematology Guidelines (Br J Haematol, 2024, 204(3): 784-804): 1) Bone marrow cellularity \\\u003C25% (or 25-50% but \\\u003C30% residual hematopoietic cell area); 2) At least two of the following conditions must be met: ANC \\\u003C0.5×10\\^9\u002FL, PLT \\\u003C20×10\\^9\u002FL, absolute reticulocyte count \\\u003C60×10\\^9\u002FL; 3) Exclusion of other congenital or acquired hematopoietic failure disorders.\n* Had at least one course of standard-dose therapy with anti-thymocyte globulin (ATG) or anti-lymphocyte globulin (ALG) combined with TPO-RA that was ineffective or relapsed after remission within the past 6 months.\n* Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.\n* Willing and able to comply with the study procedures.\n\nExclusion Criteria:\n\n* Blood cell reduction and hypoplastic bone marrow diseases due to other causes (e.g., hemolytic PNH, hypoplastic MDS\u002FAML, antibody-mediated pancytopenia).\n* Received ATG\u002FALG treatment less than 6 months ago; received TPO-RA treatment less than 4 months ago. Note: Patients on stable doses of cyclosporine or hematopoietic agents at screening can be enrolled if laboratory values are stable.\n* Significant neurological disease history, including dementia, stroke without paralysis, aphasia, seizures, or any neurological history that may pose safety risks regarding cell therapy as judged by the investigator.\n* History of allogeneic bone marrow or stem cell transplantation, or solid organ transplantation (e.g., kidney, lung, heart), or plans for such transplantation in the future.\n* History of autologous or allogeneic CAR T therapy.\n* Any primary immunodeficiency disease.\n* Significant cardiovascular dysfunction history or current significant cardiovascular dysfunction, particularly:\n* Signs or symptoms of congestive heart failure classified as NYHA class ≥III within 12 months prior to enrollment.\n\n  1. Echocardiogram showing left ventricular ejection fraction (LVEF) \\\u003C45% (assessed during screening).\n  2. History of any serious arrhythmia, currently on treatment for arrhythmias, significant history of myocardial infarction, clinically significant valvular heart disease (including mild or greater aortic regurgitation or moderate or greater mitral regurgitation), recurrent syncope, or significant hypercoagulable vascular events (e.g., transient ischemic attack or stroke); history of pulmonary embolism.\n* Liver or lung dysfunction, defined as:\n\n  1. Serum alanine aminotransferase (ALT)\u002Faspartate aminotransferase (AST) ≥2.5× the upper limit of normal (ULN);\n  2. Total bilirubin ≥1.5×ULN, except in subjects with Gilbert's syndrome.\n  3. Pulmonary hypertension, including secondary pulmonary hypertension, classified as WHO functional class \\>2.\n* Males with QTcF \\>450 msec; females with QTcF \\>470 msec, based on a single ECG or the average of three repeat ECGs taken more than 3 minutes apart.\n* History of malignancy, excluding adequately treated or surgically removed non-melanoma skin cancer or in situ cancers (e.g., cervical cancer, bladder cancer, breast cancer) without residual disease.\n* Pregnant or breastfeeding females, or females planning to become pregnant during the study or within 12 months after RD13-02 infusion.\n* History or signs of significant chronic active or recurrent infections, or significant chronic active or recurrent infections requiring antibiotics, antivirals, or antifungal treatments based on screening laboratory results.\n* Uncontrolled infections at screening. Patients with uncomplicated urinary tract infections (UTIs) and uncomplicated bacterial pharyngitis responding to active treatment may be included with investigator and medical monitor approval.\n* Positive screening for HBsAg, HCV RNA, or HIV. If a subject is HBsAg negative but HBcAb positive, hepatitis B virus DNA testing should be performed; subjects with positive hepatitis B virus DNA should be excluded from the study.\n* Vaccination with live attenuated vaccines within 4 weeks prior to enrollment, or plans to receive live attenuated vaccines during the study.\n* PT, APTT, or INR \\>1.2×ULN, except for subjects on stable doses of anticoagulants who can discontinue treatment prior to using intravenous access procedures.\n* eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m² calculated using the CKD-EPI formula; if eGFR ≥30 mL\u002Fmin\u002F1.73 m² but \\\u003C45 mL\u002Fmin\u002F1.73 m², adjust fludarabine dosing in the lymphodepleting regimen based on renal function.\n* History of drug or alcohol abuse within the past year.\n* Currently enrolled in other experimental device or drug studies, or less than 30 days since ending other experimental device or drug interventions, or less than 5 half-lives (whichever is longer).\n* History of hypersensitivity or life-threatening reactions to any component or formulation of the study drug or treatment (including lymphodepleting regimen). For detailed information about study drug components, refer to the investigator brochure (IB).\n* Any condition the investigator believes may affect participation, pose safety risks to the subjects, or potentially confound the interpretation of study results.",{"count":359,"type":21},15,[24],"This trial is exploratory research aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD13-02, a universal CD7 CAR T therapy, in subjects with relapsed\u002Frefractory severe aplastic anemia (SAA)",[28,363,364,365],"Relapse","Refractory","CAR T-cell Therapy","2025-07-23",{"date":368,"type":38},"2025-07-24",{"date":370,"type":38},"2025-06-19",{"date":372,"type":21},"2026-12-31",{"name":318,"class":70},{"id":375,"slug":376,"hasResults":11,"nctId":377,"briefTitle":378,"officialTitle":379,"acronym":380,"eligibilityCriteria":381,"healthyVolunteers":11,"sex":16,"minAge":382,"maxAge":4,"enrollmentInfo":383,"targetDuration":4,"studyType":22,"phases":385,"briefSummary":386,"conditions":387,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":390,"lastUpdatePostDateStruct":391,"startDateStruct":393,"completionDateStruct":395,"leadSponsor":396,"locationsCount":46},"100569553","phase-2-reduced-dose-of-cyclophosphamide-combined-with-standard-immunosuppressive-therapy-to-treat-severe-aplastic-anemia-100569553","NCT06695741","Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia","Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia","hypo-CASH","Inclusion Criteria:\n\n* Subject has a diagnosis of naïve severe or very severe aplastic anemia\n* Male or female age ≥ 12 years\n* Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.\n* ECOG performance status ≤2\n* Willing and able to comply with the requirements for this study and written informed consent.\n\nExclusion Criteria:\n\n* Previously received immunosuppressive therapy \\> 4 weeks\n* Previously treated with TPO-RA \\> 4 weeks\n* Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.\n* Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.\n* Tested positive for HIV or syphilis\n* Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.\n* History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years\n* Combined with other serious disorders\n* Pregnant or breast-feeding patients\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above.","12 Years",{"count":384,"type":21},75,[25],"This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of anti-lymphocyte globulin plus herombopag in combination with the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.",[28,388,389],"Cyclophosphamide Reduced-dose","Immunosuppressive Therapy","2024-11-16",{"date":392,"type":38},"2024-11-19",{"date":394,"type":38},"2024-11-01",{"date":67,"type":21},{"name":318,"class":70},{"id":398,"slug":399,"hasResults":11,"nctId":400,"briefTitle":401,"officialTitle":402,"acronym":4,"eligibilityCriteria":403,"healthyVolunteers":219,"sex":16,"minAge":223,"maxAge":271,"enrollmentInfo":404,"targetDuration":4,"studyType":22,"phases":406,"briefSummary":407,"conditions":408,"keywords":409,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":413,"lastUpdatePostDateStruct":414,"startDateStruct":416,"completionDateStruct":418,"leadSponsor":420,"locationsCount":46},"100545143","phase-2-clinical-study-on-modified-allogeneic-hematopoietic-stem-cell-transplantation-regimen-for-severe-aplastic-anemia-100545143","NCT06378060","Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia","A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia","Inclusion Criteria:\n\n1. Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;\n2. Age 3-65 years old;\n3. Weight 10Kg-100Kg;\n4. Eastern Cooperative Oncology Group (ECOG) score ≤3;\n5. No major organ injury (ECG ejection fraction \\>45%; bilirubin \\\u003C 2 times the upper limit of normal value; AST and ALT \\\u003C 3 times the upper limit of normal value; serum creatinine \\\u003C 2 times the upper limit of normal value);\n6. No severe infection;\n7. Subjects voluntarily participated in this clinical trial and signed the informed consent.\n\nExclusion Criteria:\n\n1. patients with nonhematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;\n2. patients with an expected survival of less than 1 month;\n3. patients with previous autologous or allogeneic hematopoietic stem cell transplantation;\n4. pregnant patients;\n5. patients with severe mental or neurological disorders that would affect the ability to provide informed consent and\u002For to report or observe adverse events;\n6. other conditions that the investigator determines to be inappropriate for enrollment.",{"count":405,"type":21},30,[25],"The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.",[28],[28,410,411,412],"Modified transplantation system","Ruxolitinib","Melphalan","2024-04-19",{"date":415,"type":38},"2024-04-22",{"date":417,"type":38},"2024-03-01",{"date":419,"type":21},"2027-04-01",{"name":421,"class":70},"Hematology department of the 920th hospital",{"id":423,"slug":424,"hasResults":11,"nctId":425,"briefTitle":426,"officialTitle":427,"acronym":4,"eligibilityCriteria":428,"healthyVolunteers":11,"sex":16,"minAge":298,"maxAge":429,"enrollmentInfo":430,"targetDuration":4,"studyType":22,"phases":431,"briefSummary":432,"conditions":433,"keywords":435,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":439,"lastUpdatePostDateStruct":440,"startDateStruct":442,"completionDateStruct":444,"leadSponsor":446,"locationsCount":46},"100535648","phase-2-rituximab-for-serious-aplastic-anemia-with-platelet-transfusion-refractoriness-100535648","NCT06254560","Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness","The Study of Rituximab in the Treatment of Severe Aplastic Anemia With Platelet Transfusion Refractoriness","Inclusion Criteria:\n\n* Initial diagnosed SAA with PTR\n* Age\\>18 years old, regardless of gender\n* Initial diagnosed SAA with PTR\n* Age\\>18 years old, regardless of gender\n\nExclusion Criteria:\n\n* Allergy to rituximab\n* Severe active infection\n* Hypogammaglobulinemia\n* Pregnant and lactating women\n* Heart failure (NYHA classification IV)\n* Individuals with epilepsy, dementia, and other mental disorders that require medication treatment who cannot understand or follow the research protocol\n* Chronic infections or other chronic diseases that may be risk to the experiment\n* The researchers believe that it is not suitable for participants","70 Years",{"count":202,"type":21},[25],"Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.",[28,434],"Platelet Transfusion Refractoriness",[436,437,438],"Severe aplastic anemia","Platelet transfusion refractoriness","Rituximab","2024-02-03",{"date":441,"type":38},"2024-02-12",{"date":443,"type":38},"2023-02-23",{"date":445,"type":21},"2027-12-30",{"name":318,"class":70},{"id":448,"slug":449,"hasResults":11,"nctId":450,"briefTitle":451,"officialTitle":452,"acronym":453,"eligibilityCriteria":454,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":298,"enrollmentInfo":455,"targetDuration":4,"studyType":22,"phases":456,"briefSummary":457,"conditions":458,"keywords":4,"overallStatus":309,"whyStopped":4,"lastUpdateSubmitDate":461,"lastUpdatePostDateStruct":462,"startDateStruct":464,"completionDateStruct":466,"leadSponsor":468,"locationsCount":4},"100471512","phase-2-up-front-matched-unrelated-donor-transplantation-in-pediatric-patients-with-idiopathic-aplastic-anemia-100471512","NCT05419843","Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia","Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia: a Phase II Feasibility Study","UPFRONT-MUD","Inclusion Criteria:\n\n* age\\\u003C18years old\n* Pediatric patients aged less than 18 years with idiopathic aplastic anemia and an indication for treatment (severe aplastic anemia or moderate aplastic anemia requiring transfusions)\n* With a good probability to have a HLA-10\u002F10 matched unrelated donor available (the patient needs to have at least 3 MUD identified within the book BMDW (Bone Marrow Donors Worldwide) or using the easy match software to be included)\n* With usual criteria for allo-SCT:\n* Lansky \\>70% for those below 16 years and Karnofsky \\> 70% for those above 16 years\n* No severe and uncontrolled infection\n* Adequate organ function: ASAT and ALAT ≤ 5N\\*, total bilirubin ≤ 2N, creatinine clearance \\> 70% of higher normal values for age.\n* With health insurance coverage\n* Contraception methods\\*\\* for young girl and men of childbearing age must be prescribed during all the duration of the research.\n* Parents having read and understand the information note and signed a written informed consent (the patient's agreement depending on his age will be sought)\n\n  \\*because typical presentation of aplastic anemia post-hepatitis\n\n  \\*\\* NB : The authorized contraceptive methods are:\n* For women of childbearing age and in absence of permanent sterilization: oral, intravaginal or transdermal combined hormonal contraception, oral, injectable or transdermal progestogen-only hormonal contraception, intrauterine hormonal-releasing system (IUS).\n* For man in absence of permanent sterilization: condoms\n\nExclusion Criteria:\n\nPatients :\n\n* With a matched related donor available\n* With uncontrolled infection\n* With seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and associated hepatic cytolysis\n* Renal failure with creatinine clearance below 70% of higher normal values for age\n* Pregnant (βHCG positive) or breast-feeding\n* With Heart failure according to NYHA (II or more)\n* Preexisting acute hemorrhagic cystitis\n* Urinary tract obstruction\n* Yellow fever vaccine within 2 months before transplantation\n* Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up (depending of his age and understanding).\n* With Contraindication to treatments used during the research",{"count":263,"type":21},[25],"Pediatric patients with idiopathic aplastic anemia (AA) respond better than adults to immunosuppressive therapy (IST) but the long-term risks of relapse, ciclosporine dependence, and clonal evolution are high. UK investigators reported a 5-year estimated failure-free survival (FFS) after IST of 13.3%. In contrast, in 44 successive children who received a matched unrelated donor (MUD), hematopoietic stem cell transplantation (HSCT), there was an excellent estimated 5-year FFS of 95%. Forty of these children had previously failed IST. Because of those excellent results, up-front fully matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) became an attractive first-line option. In 2005 to 2014, a UK cohort of 29 children with idiopathic AA thus received MUD HSCTs as first-line therapy (they did not receive IST prior to HSCT). Results were excellent, with low Graft versus Host Disease rates and only 1 death (idiopathic pneumonia). This cohort was then compared with historical matched controls, transplanted or not. Outcomes for the up-front unrelated cohort HSCT were similar to Matched Related Donor HSCT and superior to IST and unrelated HSCT post-IST failure. Since then, many investigators are offering up-front MUD HSCT in pediatric patients worldwide. However, those results should be treated with extreme caution: 1) the design is retrospective; 2) the excellent up-front MUD HSCT may arise from the use of alemtuzumab in the conditioning regimen (alemtuzumab is not easily available worldwide) and 3) there was no formal quality-of-life assessment. Moreover, this strategy is highly dependent on donor identification (Caucasian patients have the highest likelihood of having a MUD) and donor not eventually receive HSCT because of the risk of infections\u002Fcomplications caused by unexpected donor delays or cancellation. Prospective trials are thus urgently needed to address the feasibility of such procedure, in term of timing (delay to offer MUD HSCT) and conditioning regimen (nothing is known of the use of other regimens, non alemtuzumab-based, in this setting).\n\nThe main objective of this Two-Stage Phase 2 multicenter study is to realize up-front HSCT within 2 months once a MUD has been identified.",[459,28,460],"Idiopathic Aplastic Anemia","Moderate Aplastic Anemia Requiring Transfusions","2022-06-10",{"date":463,"type":38},"2022-06-15",{"date":465,"type":21},"2022-06",{"date":467,"type":21},"2027-06",{"name":469,"class":70},"Assistance Publique - Hôpitaux de Paris"]