[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"bone-marrow-failure-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:bone-marrow-failure-syndrome":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,43,67,101,124,143,169],{"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":25,"conditions":26,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100389867","phase-2-tcr-alpha-beta-t-cell-depleted-haploidentical-hct-in-the-treatment-of-non-malignant-hematological-disorders-in-children-100389867",false,"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.","ALL","0 Years","21 Years",{"count":20,"type":21},17,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","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.",[27,28,29],"Hemoglobinopathy (Disorder)","Severe Aplastic Anemia","Bone Marrow Failure Syndrome","RECRUITING","2026-06-29",{"date":33,"type":34},"2026-07-01","ACTUAL",{"date":36,"type":34},"2020-07-22",{"date":38,"type":21},"2027-06-30",{"name":40,"class":41},"Johns Hopkins All Children's Hospital","OTHER",1,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":53,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":42},"100638480","phase-1-stem-cell-mobilization-and-apheresis-for-life-threatening-blood-disorders-100638480","NCT07585136","Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders","Inclusion Criteria:\n\n* Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy\n* Age ≥ 18 years - 25 years\n* The following hematological parameters need to be met (regardless of transfusion or growth factor support)\n\n  * Hb \\> 8 g\u002FdL\n  * ANC \\> 500\u002Fmm3\n  * Platelet \\> 30,000\u002Fmm3\n* Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis\n* Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed\n* Karnofsky score \\>80\n* Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1\u002FII\n* Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration\n\nExclusion Criteria:\n\n* Participant with sickle cell disease\n* Participant who has had a prior autologous or allogeneic HSCT\n* Active viral, bacterial, fungal, or parasitic infection\n* Total bilirubin \\>2.5x ULN or transaminases \\>5x ULN\n* Moderate or severe renal failure defined as serum\u002Fplasma creatinine \\>1.5 mg\u002FdL and an estimated glomerular filtration rate (eGFR) \\\u003C 60 mL\u002Fmin\u002F1.73 m2 based on the CKD-Epi equation or the St. Jude equation\n* Diagnosis of MDS or other hematologic malignancy\n* History of malignancy\n* Known allergy to or contraindication for Filgrastim or plerixafor administration, or medications routinely administered during apheresis\n* Splenomegaly (size greater than upper limit of normal on examination)\n* Any disease or concomitant process that is not compatible with the study as per investigator opinion\n* Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent\n* Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF\n* Pregnancy\n* Inability or unwillingness of research participant to give written informed consent.","18 Years","25 Years",{"count":52,"type":21},12,[54],"PHASE1","The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy.\n\nPrimary objective:\n\n\\- To characterize the safety of Filgrastim plus plerixafor in participants with bone marrow failure syndromes as determined by the incidence of adverse events (AEs).\n\nSecondary Objectives:\n\n* To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts.\n* To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts.\n* To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells\u002Fkg).",[29],"NOT_YET_RECRUITING","2026-05-18",{"date":60,"type":34},"2026-05-22",{"date":62,"type":21},"2026-06",{"date":64,"type":21},"2029-07-01",{"name":66,"class":41},"St. Jude Children's Research Hospital",{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":71,"acronym":4,"eligibilityCriteria":72,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":4,"enrollmentInfo":73,"targetDuration":4,"studyType":22,"phases":75,"briefSummary":77,"conditions":78,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":100},"100282586","pre-myeloid-cancer-and-bone-marrow-failure-clinic-study-100282586","NCT02958462","Pre-myeloid Cancer and Bone Marrow Failure Clinic Study","Inclusion Criteria:\n\n* Patients with idiopathic cytopenias of unclear significance (ICUS)\n* Patients with clonal hematopoiesis of indeterminate significance (clonal hematopoiesis of indeterminate potential \\[CHIP\\]), including the recently described CHIP syndrome called VEXAS (vacuoles, E1 ubiquitin ligase, X chromosomal, autoimmune and somatic)\n* Patients with clonal cytopenias of undetermined significance (CCUS)\n* Marrow failure syndromes with myeloid malignancy predisposition - telomere dysfunction, chromosomal breakage disorders\n* Germ line inherited syndromes with risk for malignant transformation - GATA2, CEBPA, ETV-6, RUNX1, JAK2, PF6, etc.\n* Low risk MDS (idiopathic dysplasia of unclear significance)\n* Family member of a patient with one of the above conditions\n* Patient at high risk or suspected of developing one of the above conditions\n\nExclusion Criteria:\n\n* Patients under 18 years of age",{"count":74,"type":21},2000,[76],"NA","This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes. NGS is a procedure that looks at relevant cancer associated genes and what they do. Finding genetic markers for pre-malignant conditions may help identify patients who are at risk of pre-myeloid cancers and bone marrow failure syndromes and lead to earlier intervention.",[79,80,81,82,29,83,84,85,86,87,88,89,90],"Myeloid Malignancy","Inherited Bone Marrow Failure Syndrome","Clonal Expansion","Cytopenia","Clonal Cytopenia of Undetermined Significance","Clonal Hematopoiesis of Indeterminate Potential","Hematologic Neoplasms","Hematopoietic and Lymphatic System Neoplasm","Hereditary Neoplastic Syndrome","Idiopathic Cytopenia of Undetermined Significance","Idiopathic Dysplasia of Uncertain Significance","Low Risk Myelodysplastic Syndrome","2026-02-20",{"date":93,"type":34},"2026-02-23",{"date":95,"type":34},"2017-01-16",{"date":97,"type":21},"2035-09-15",{"name":99,"class":41},"Mayo Clinic",3,{"id":102,"slug":103,"hasResults":11,"nctId":104,"briefTitle":105,"officialTitle":106,"acronym":4,"eligibilityCriteria":107,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":18,"enrollmentInfo":108,"targetDuration":4,"studyType":22,"phases":110,"briefSummary":111,"conditions":112,"keywords":114,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":116,"lastUpdatePostDateStruct":117,"startDateStruct":119,"completionDateStruct":121,"leadSponsor":123,"locationsCount":42},"100405389","phase-2-haploidentical-hct-for-severe-aplastic-anemia-100405389","NCT04558736","Haploidentical HCT for Severe Aplastic Anemia","Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia","Inclusion Criteria for Transplant Recipient\n\n1. Age less than or equal to 21 years at time of enrollment.\n2. Confirmed diagnosis of SAA or a single lineage cytopenia\n\n   (a) SAA or single lineage cytopenia will be defined as follows:\n   * i. Bone marrow cellularity \\\u003C 25% or hypocellular marrow for age, AND\n   * ii. One or more of the following (in peripheral blood): (i) Neutrophils \\\u003C 0.5 x10\\^9\u002FL (ii) Platelets \\\u003C 20 x10\\^9\u002FL, or platelet transfusion dependence (iii) Hemoglobin \\\u003C8g\u002FdL, or red blood cell transfusion dependence\n3. Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10\u002F10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.\n4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenias after first-line IST but then a later return to fulfillment of SAA disease criteria when IST is decreased or ceased). IST could have included ATG based regimens, calcineurin inhibitors and\u002For other higher dose therapy directed at the treatment of primary SAA. Patients with very severe aplastic anemia who are likely not to benefit from IST do not need to have failed a trial of IST and can proceed directly to HCT if they meet the rest of the criteria.\n5. Has a suitable single haplotype matched (≥ 3 of 6) family member donor.\n6. Patient and\u002For legal guardian must sign informed consent for HCT.\n7. Adequate organ function defined as:\n\n   1. Left ventricular ejection fraction \\> 40% or shortening fraction ≥ 25%.\n   2. Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml\u002F min\u002F1.73m2.\n   3. Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry\n   4. ≥ 92% on room air if patient is unable to perform pulmonary function testing.\n   5. Karnofsky or Lansky (age-dependent) performance score ≥ 50.\n   6. Bilirubin ≤ 3 times the upper limit of normal for age.\n   7. Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.\n8. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered\n\nExclusion Criteria for Transplant Recipient:\n\n1. Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.\n2. Known clinical or genetic diagnosis of dyskeratosis congenita\n3. Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).\n4. Diagnosis of myelodysplastic syndrome (MDS).\n5. Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement- dependent cytotoxicity or flow cytometric testing or the presence of anti- donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) \\> 1000 by solid phase immunoassay).\n6. Prior allogeneic hematopoietic cell transplant.\n7. Prior solid organ transplant.\n8. Known life-threatening reaction (i.e., anaphylaxis) to ATG that would prohibit use for the patient.\n9. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as progression or no clinical improvement on appropriate medical treatment.\n10. Female patients who are pregnant (per institutional practice) or breast- feeding.\n11. 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 PI.\n12. Alemtuzumab or ATG within 2 weeks of enrollment.\n\nInclusion Criteria for Haploidentical Donor\n\n1. At least single haplotype matched (≥ 3 of 6) family member.\n2. At least 18 years of age.\n3. HIV negative.\n4. Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).\n5. Not breast feeding.\n6. Related donors must be ruled out for telomere disease by appropriate clinical and diagnostic measures (for example, clinical evaluation, telomere length testing, genetic testing, and\u002For bone marrow examination).\n7. The HAPLO donor and\u002For legal guardian must be able to sign informed consent documents.\n8. The potential HAPLO donor must be willing and able to donate PBSCs.",{"count":109,"type":21},21,[24],"This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT).\n\nPrimary Objectives\n\n* To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.\n* To estimate the overall survival and event free survival at 1-year post transplantation.\n\nSecondary Objectives\n\n* To calculate the incidence of acute and chronic GVHD after HCT.\n* To calculate the rate of secondary graft rejection at 1-year post transplantation\n* To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).\n* To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.\n\nExploratory Objectives\n\n* To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.\n* To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.\n* To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.\n* To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.\n* Quantify donor derived Treg cells at different time points in patients received HCT.\n* Determine Treg activation status at different stages after HCT.\n* Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?\n* How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?\n* Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?",[113,29],"Aplastic Anemia",[115],"Haploidentical Transplant","2025-07-03",{"date":118,"type":34},"2025-07-04",{"date":120,"type":34},"2021-01-21",{"date":122,"type":21},"2030-07-01",{"name":66,"class":41},{"id":125,"slug":126,"hasResults":11,"nctId":127,"briefTitle":128,"officialTitle":129,"acronym":4,"eligibilityCriteria":130,"healthyVolunteers":11,"sex":16,"minAge":131,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":132,"phases":4,"briefSummary":133,"conditions":134,"keywords":4,"overallStatus":137,"whyStopped":4,"lastUpdateSubmitDate":138,"lastUpdatePostDateStruct":139,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":141,"locationsCount":42},"100296942","expanded-access-protocol-using-alphabeta-t-and-cd19-depleted-pbsc-100296942","NCT03145545","Expanded Access Protocol Using Alpha\u002FBeta T and CD19+ Depleted PBSC","Expanded Access Protocol Using TCR Alpha\u002FBeta T Cell\u002FCD19+ Depleted Donor Peripheral Stem Cells","PATIENT AND DONOR ELIGIBILITY\n\nPatients who lack an HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using CliniMACs device for β T\u002FCD19+ depletion. Patients with matched related donors including matched sibling donors may also be eligible if special clinical indications are met.\n\nPatients with the following transplantable diseases:\n\nNon-malignant diseases:\n\n* Metabolic storage diseases correctable by HSCT\n* Bone marrow failure syndromes\n* Immunodeficiencies\u002Fimmune dysregulation syndromes\n* Sickle cell disease or thalassemia\n* Other diseases treated with HSCT\n\nMalignant diseases:\n\n* Acute leukemias\n* Chronic leukemias\n* Lymphomas\n* Myelodyplastic syndrome\n\nPatient Eligibility criteria:\n\nIt is important to note that the conditioning prescribed to the patient will be determined based on the disease and organ status and will include agents that are standard. Appropriate combinations of chemotherapy, immunotherapy and\u002For radiation will be determined on an individual basis.\n\nPatient eligibility will be assessed as per our institutional standard operating procedures:\n\n* Signed informed consent\n* Lansky or Karnofsky performance ≥ 60\n* Hematologic and Organ Function as per current institutional SOP\n* Infectious Evaluation as per current institutional SOP\n* Participants of childbearing potential must have a negative pregnancy test as per institutional SOP\n* Subjects with graft failure who require a second HSCT will not need to meet eligibility criteria again prior to the second transplant. Graft failure is a medical emergency that requires HSCT\n\nDonor Eligibility Patients must have an identified living donor\n\n* Donor selection will comply with 21 CFR 1271\\*\n* Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10\u002F10 match, a 9\u002F10 match, or an 8\u002F10 match if one of the mismatches is at DQB1.\n* Related donor mismatched at one to five HLA alleles (haploidentical)\n* Matched related donor may be considered suitable donors for this protocol if a peripheral stem cell donation is deemed by the clinical team to be a safer donation option (if donor is not a suitable candidate for a bone marrow harvest) or if there is concern that bone marrow harvest would not yield adequate cell doses Additionally, a matched related donor would be considered suitable for this protocol if there are safety concerns regarding a patient receiving a standard T cell replete bone marrow transplant due to individualized GVHD risk or risk related to standard GVHD prevention medications.\n* Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, HepB, HepC PCR negative.\n* We assess donor eligibility as per our Allogenic Donor Evaluation for Eligibility standard operating procedure. These procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is FACT accredited.\n* Related donors will be consented and enrolled under IRB approved research protocol for cell collection per IRB 04-004078 CHP 784 Clinical and Research Collection and Future Research Use of Bone Marrow, Stem Cells or T Cells.\n* Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis.\n* The donors selected for this IDE will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors, NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases\n\nExclusion criteria:\n\n* Uncontrolled bacterial, viral, or fungal infections\n* Fully HLA matched sibling donor (fully matched related donors including siblings may be included in special circumstances)\n* Donor unable to donate peripheral stem cells\n* Pregnant Females","1 Month","EXPANDED_ACCESS","The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.",[135,29,136],"Leukemia","Immunodeficiencies","AVAILABLE","2025-05-18",{"date":140,"type":34},"2025-05-22",{"name":142,"class":41},"Children's Hospital of Philadelphia",{"id":144,"slug":145,"hasResults":11,"nctId":146,"briefTitle":147,"officialTitle":148,"acronym":4,"eligibilityCriteria":149,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":150,"targetDuration":4,"studyType":22,"phases":152,"briefSummary":154,"conditions":155,"keywords":156,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":160,"lastUpdatePostDateStruct":161,"startDateStruct":163,"completionDateStruct":165,"leadSponsor":167,"locationsCount":42},"100576612","early-phase-1-cd7-car-t-cell-sequential-allo-hsct-for-non-malignant-blood-and-immune-system-diseases-100576612","NCT06787560","CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System Diseases","Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases","Inclusion Criteria:\n\n* 1\\. Non-malignant blood and immune system diseases include: hereditary bone marrow failure, congenital immune deficiency, hemoglobinopathy and other non-malignant blood and immune system diseases,\n\n  1. Confirmed hereditary bone marrow failure syndrome. Including: Fanconi anemia, congenital pure red cell aplastic anemia, congenital dyskeratosis, Scheux-Day syndrome, congenital neutropenia, various bone marrow failure related congenital thrombocytopenia and other unclassified congenital bone marrow exhaustion diseases;\n  2. It meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease;\n  3. Diagnosed with hemoglobinopathy and dependent on blood transfusions; serum ferritin levels are \\\u003C 3000 μg\u002FL, with cardiac and hepatic iron content indicating moderate or lower iron overload; documentation of iron chelation therapy (including prescriptions or invoices) for at least three months prior to screening is available; no hydroxyurea, ruxolitinib, decitabine, or cytarabine has been administered in the three months preceding enrollment. The spleen size must not extend beyond the umbilical horizontal line or the midline of the abdomen. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated, and suitable donors for related allo-HSCT are available.\n* 2\\. Serum total bilirubin ≤1.5 times the upper limit of normal value, serum Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal value range;\n* 3\\. Echocardiography showed Left ventricular ejection fraction (LVEF) \\>50%;\n* 4\\. Pulse oxygen saturation ≥92% (non-oxygen state);\n* 5\\. The estimated survival is more than 3 months;\n* 6\\. ECOG score 0-1;\n* 7\\. Abdominal B-ultrasonography and other examinations were performed to evaluate spleen size. Splenectomy should be evaluated before transplantation for patients with giant spleen;\n* 8\\. Women and men who are fertile must consent to the use of appropriate contraception before entering the study, during study participation, and for 6 months after transfusion (the safety of this therapy for the unborn child is not known, with unknown risks);\n* 9\\. Subjects who are willing to participate in the study are able to understand and have the ability to sign informed consent.\n\nExclusion Criteria:\n\n* 1\\. People with a history of epilepsy or other central nervous system disorders;\n* 2\\. Epstein-Barr virus (EBV) DNA positive;\n* 3\\. People with a history of prolonged QT interval or serious heart disease;\n* 4\\. People with active hepatitis B or C virus;\n* 5\\. Tuberculosis, AIDS and other major infectious diseases;\n* 6\\. Sepsis, pulmonary infection, intestinal infection and other major organ infection and poor control, and\u002For hypersensitive C-reactive protein, procalcitonin significantly elevated;\n* 7\\. People who have previously received other clinical studies and gene therapy;\n* 8\\. Any situation that the investigator believes may increase the risk to the subject or interfere with the test results.",{"count":151,"type":21},20,[153],"EARLY_PHASE1","A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases",[29],[157,158,159],"CAR-T","Allo-HSCT","Immuno Deficiency Disorders","2025-01-16",{"date":162,"type":34},"2025-01-22",{"date":164,"type":21},"2025-01-31",{"date":166,"type":21},"2028-01-31",{"name":168,"class":41},"Zhejiang University",{"id":170,"slug":171,"hasResults":11,"nctId":172,"briefTitle":173,"officialTitle":174,"acronym":175,"eligibilityCriteria":176,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":177,"targetDuration":4,"studyType":179,"phases":4,"briefSummary":180,"conditions":181,"keywords":182,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":190,"lastUpdatePostDateStruct":191,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":197,"locationsCount":42},"100422512","french-national-registry-of-bone-marrow-failures-100422512","NCT04781790","French National Registry of Bone Marrow Failures","French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project","RIME","Inclusion Criteria:\n\n* All age\n* All diagnostic of BMF\n* Having given his non-opposition to registry after understand overall aims\n* Having signed a written informed consent (2 parents for patients aged less than 18) for collection of biological samples\n* With health insurance coverage\n\nExclusion Criteria:\n\nWith myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia",{"count":178,"type":21},5000,"OBSERVATIONAL","This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and throughout follow-up during the natural history of the disease, treated or not. In parallel, biological samples (blood and\u002For bone marrow and\u002For skin) are collected during clinical care and are biobanked in Saint-Louis Hospital (Hematology laboratory) in order to be used in translational research related to bone marrow failure diseases.\n\nThis registry has two main objectives:\n\n* Public health care evaluation and improvement: to assess the medical and social needs inherent to the management of these rare diseases; to precisely assess the level of diagnosis and management of bone marrow failure syndromes in France; to evaluate the impact and guidance of the French reference center guidelines for diagnosis and treatment; to evaluate the real-life efficacy and tolerance of any given specific treatments; to analyze treatment's cost-effectiveness according to each situation.\n* Research:\n\n  * Epidemiology: to determine the incidence, prevalence, and distribution of different bone marrow failure syndromes at the national level;\n  * Biology: to better understand the pathophysiology of BMF; to identify and to study complications within each entity, such as mechanisms underlying clonal evolution, new forms of inherited BMF and acute myeloid leukemia (AML)\u002FMDS-predisposition syndromes, and to better and deeper characterize known entities;\n  * Treatment: to identify prognostic factors and predictors of response; to identify side effects and impact of treatment on others organs and natural functions; to assess patients' quality of life as early as possible since diagnosis and throughout follow-up.",[29],[183,184,185,186,187,188,189],"aplastic anemia","Fanconi anemia","blackfan diamond anemia","telomeropathies","inherited aplastic anemia","acquired aplastic anemia","medullary hypoplasia","2021-03-01",{"date":192,"type":34},"2021-03-04",{"date":194,"type":34},"2017-02-06",{"date":196,"type":21},"2027-02-06",{"name":198,"class":41},"Assistance Publique - Hôpitaux de Paris"]