[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"bone-marrow-failure\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:bone-marrow-failure":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,47,78,121,158,187,216],{"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":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100641447","phase-2-naive-t-cell-deplete-grafts-for-gvhd-prevention-in-non-malignant-diseases-100641447",false,"NCT07660783","Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases","A Phase II Prospective Study Evaluating Selective Depletion of CD45RA+ (Naïve) T Cells (TND) From Peripheral Blood Stem Cell (PBSC) Grafts for Prevention of Graft Versus Host Disease (GVHD) in Non-Malignant Diseases (NMDs)","Inclusion Criteria:\n\n* Considered appropriate candidate for allogeneic HCT following low dose (4Gy) TBI containing-conditioning and have one of the following diagnoses: A) BMF B)Hemoglobinopathies C)PID D) Autoimmune cytopenias E) Immune dysregulation F) HLH G) Other NMD treatable by HCT and NMD that is not clearly defined (a patient with a NMD for whom genetic testing has been done and a genetic mutation responsible for their NMD phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol PI\n* Patients aged 6 months- 5 years old (inclusive) at the time of informed consent\n* Patient with suitable HCT donor (see inclusion criteria below)\n* Recipient informed consent\u002Fassent (13 years and older), and\u002For legal guardian permission must be obtained\n\nExclusion Criteria:\n\n* Patient with aplastic anemia\n* Patients with severe combined immunodeficiency (SCID)\n* Fanconi anemia\n* Dyskeratosis congenita\n* Patient weight \\> 100 kg\n* Patients who are positive for HIV-1, HIV-2\n* Patients with current neoplastic disorders\n* Patients with uncontrolled infections for whom HCT is considered contraindicated by the consulting infectious disease physician.\n* Patients with organ dysfunction including A) Renal insufficiency B) Impaired cardiac function C)Impaired pulmonary function D) Liver dysfunction\n* Patients who are pregnant or breast-feeding\n* Patients on other experimental protocols for prevention of GVHD\n* Patients of childbearing age who are presumed to be fertile and are unwilling to use an effective birth control method or refrain from sexual intercourse during and for 12 months post-HCT\n* Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI\n* Patients with a known hypersensitivity to tacrolimus or MMF","ALL","6 Months","50 Years",{"count":20,"type":21},40,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.",[27,28,29,30,31,32,33],"Bone Marrow Failure","Hemoglobinopathies","Primary Immune Deficiency (PID)","Autoimmune Cytopenia","Immune Dysregulation","Hemophagocytic Lymphohistiocytosis (HLH)","Non Malignant Disorders","NOT_YET_RECRUITING","2026-06-16",{"date":37,"type":38},"2026-06-22","ACTUAL",{"date":40,"type":21},"2026-09",{"date":42,"type":21},"2035-09",{"name":44,"class":45},"Fred Hutchinson Cancer Center","OTHER",2,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":53,"eligibilityCriteria":54,"healthyVolunteers":55,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":56,"targetDuration":58,"studyType":59,"phases":4,"briefSummary":60,"conditions":61,"keywords":63,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":77},"100610405","the-hope-biobank-resource-bmt-ctn-2402-hope-100610405","NCT07227155","The HOPE Biobank Resource (BMT CTN 2402 HOPE)","Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)","HOPE Resource","HCT\u002FGT Inclusion:\n\n1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol:\n\n   1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians.\n   2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.\n   3. Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included.\n2. Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed.\n3. Patients or their legal guardian must consent to participate in the CIBMTR \"Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries\" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR.\n4. All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required.\n5. All participants or parent\u002Flegal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing bmtctn2402@emmes.com.\n\nHCT\u002FGT Exclusion\n\n1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT.\n2. Active malignancy.\n3. Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years.\n4. Weight ≤ 10.0 kg at time of study enrollment.\n5. Prior autologous or allogeneic transplant.\n\nRelated Donor Inclusion:\n\n1\\. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study.\n\nRelated Donor Exclusion:\n\n1\\. Donor weight ≤ 10.0 kg at time of study enrollment",true,{"count":57,"type":21},375,"5 Years","OBSERVATIONAL","A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.",[62,28,27],"Aplastic Anemias",[64,65,66,67],"Biorepository","Biospecimens","Allogenic Transplant","Gene Therapy","RECRUITING","2026-06-12",{"date":35,"type":38},{"date":72,"type":38},"2026-05-15",{"date":74,"type":21},"2031-03-30",{"name":76,"class":45},"Medical College of Wisconsin",6,{"id":79,"slug":80,"hasResults":11,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":4,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":16,"minAge":85,"maxAge":86,"enrollmentInfo":87,"targetDuration":4,"studyType":22,"phases":89,"briefSummary":91,"conditions":92,"keywords":105,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":120},"100580604","phase-1-phase-12-cd45ra-depleted-stem-cell-addback-to-prevent-viral-or-fungal-infections-post-tcrabcd19-depleted-hsct-100580604","NCT06839456","Phase 1\u002F2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab\u002FCD19 Depleted HSCT","Phase 1\u002F2 Study: CD45RA Depleted Peripheral Stem Cell Addback to Prevent Viral and Fungal Infections Following Alternative Donor TCRab\u002FCD19 Depleted Hematopoietic Stem Cell Transplant","Inclusion Criteria:\n\n1. Disease for which allogeneic HSCT may be curative.\n2. Remission status of hematologic malignancies and additional disease-specific eligibility determinations will be according to standards of practice within the CHOP Cellular Immunotherapy and Transplant Program (CTTS).\n3. Patients must be 25 years of age and less\n4. Evaluation for organ and infectious status as per our CTTS standard operating procedure.\n5. Signed consent by parent\u002Fguardian or able to give consent if 18 years of age and older.\n6. Participants of childbearing potential must have a negative pregnancy test as per institutional SOP.\n\nExclusion Criteria:\n\n1. Patients who have performance score less than 60.\n2. No suitable donor available for mobilized peripheral stem cells.\n3. Patients with Hodgkin lymphoma or non-Burkitt, non-lymphoblastic lymphoma.\n4. Planned receipt of alemtuzumab during conditioning.\n5. Patients with an available 10\u002F10 HLA matched sibling donor.\n6. Patients who do not meet institutional disease, organ or infectious criteria.\n\nDonor selection and eligibility:\n\n1. Unrelated donor meets National Marrow Donor Program criteria for donation.\n2. Related donor (at least haploidentical) willing and able to donate mobilized peripheral stem cells.\n3. HLA testing\u002Fmatching\n\n   * HLA testing to be done by molecular methods for A, B, C, DRB1, DQB1\n   * Related donor: Must be ≥ 5\u002F10 match\n   * Unrelated donor: 10\u002F10 or 9\u002F10 match\n   * KIR typing for haploidentical donor for hematologic malignancies\n   * Donor specific HLA antibodies (DSA) should be assessed for all subjects receiving an HLA mismatched graft (≤ 9\u002F10).\n4. Donor must be willing to undergo granulocyte colony stimulating factor (GCSF) mobilization and peripheral blood stem cell collection\n5. Donors must be willing to sign consent to participate in this study.","1 Month","25 Years",{"count":88,"type":21},100,[90,24],"PHASE1","The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab\u002FCD19 depleted PSCT.",[93,94,95,96,97,98,99,100,101,102,28,27,103,104],"Leukemia","High Risk Acute Lymphoblastic Leukemia","High Risk Acute Myeloid Leukemia","Relapse Leukemia","MDS (Myelodysplastic Syndrome)","Relapsed Non-Hodgkin Lymphoma","Acquired Aplastic Anemia","Inherited BMF Syndrome","Immunodeficiency","Primary Immune Regulatory Disorder","Inborn Errors of Metabolism","HLH",[106,107,108,109,110],"alpha beta T cell depletion","CD45RA","CD45RO","GVHD prevention","Memory T cells","2026-04-13",{"date":113,"type":38},"2026-04-15",{"date":115,"type":38},"2025-03-21",{"date":117,"type":21},"2032-03",{"name":119,"class":45},"Children's Hospital of Philadelphia",1,{"id":122,"slug":123,"hasResults":11,"nctId":124,"briefTitle":125,"officialTitle":126,"acronym":4,"eligibilityCriteria":127,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":128,"enrollmentInfo":129,"targetDuration":4,"studyType":22,"phases":131,"briefSummary":132,"conditions":133,"keywords":139,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":150,"lastUpdatePostDateStruct":151,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":157,"locationsCount":120},"100411935","phase-2-cord-blood-transplant-in-children-and-young-adults-with-blood-cancers-and-non-malignant-disorders-100411935","NCT04644016","Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders","Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders","Participant Inclusion Criteria:\n\nAge and Donor Status:\n\nPatients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period.\n\nDiagnoses :\n\nI. Acute myelogenous leukemia (AML) :\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).\n  * Therapy-related AML (t-AML).\n  * White cell count at presentation \\> 100,000.\n  * Presence of extramedullary leukemia at diagnosis.\n  * Any unfavorable subtype by FAB or WHO classification.\n  * High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.\n  * Requirement for 2 or more inductions to achieve CR1.\n  * Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.\n  * Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.\n  * Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in ≤ 10% of cells are eligible.\n\nII. Acute lymphoblastic leukemia (ALL):\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * White cell count at presentation \\> 30,000 for B-cell lineage and \\> 100,000 for T-cell lineage.\n  * Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.\n  * Failure to achieve complete remission (CR) after four weeks of induction therapy.\n  * Persistence or recurrence of MRD on therapy.\n  * Any patient unable to tolerate consolidation and\u002For maintenance chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and\u002For CAR-T cell therapy.\n\nIII. Other acute leukemias:\n\nLeukemias of ambiguous lineage or of other types (e.g. blastic plasmacytoid dendritic cell neoplasm) with less than 5% blasts by BM morphology. Patients with persistent\u002Frelapsed disease with cytogenetic, flow cytometric or molecular aberrations in ≤ 5% of cells are eligible.\n\nIV. Myelodysplastic Syndrome (MDS) \u002F Myeloproliferative Disorders (MPD) other than myelofibrosis:\n\n* International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.\n* Any IPSS risk category if life-threatening cytopenia(s) exists.\n* Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.\n* MDS\u002F myeloproliferative disorder overlap syndromes without myelofibrosis.\n* MDS\u002F MPD patients must have less than 10% bone marrow myeloblasts and ANC ≥ 0.2 (growth factor supported if necessary) at transplant work-up.\n\nV. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission:\n\n* Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.\n* Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease\u002F CR\u002F PR with no single lesion equal to or more than 5 cm.\n* Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion ≥ 5 cm.\n\nVI. Inherited Metabolic Disorders \\[also see EBMT Handbook for discussion on patient eligibility for allogeneic transplant; in general, patients are considered early in the disease course, before they develop neurologic symptoms (46)\\]:\n\n* Hurler Syndrome\n* Hunter (MPS 2 - early disease)\n* Sly syndrome (MPSVIII)\n* α-Mannosidosis\n* X- ALD\n* Osteopetrosis\n* Metachromatic Leukodystrophy\n* Globoid (GLD)\n\nVII. Non-Malignant disorders (other) \\[also see EBMT Handbook for criteria for transplant (46)\\]\n\n* Hemoglobinopathies\n* Bone Marrow Failure syndromes\n* Immunodeficiencies, including HLH\n\nOrgan Function and Performance Status Criteria:\n\n* Karnofsky or Lansky score ≥ 70% (see Appendix)\n* Bilirubin ≤ 1.5 mg\u002FdL (unless benign congenital hyperbilirubinemia).\n* ALT ≤ 3 x upper limit of normal.\n* Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .\n* Left ventricular ejection fraction ≥ 50%.\n* Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.\n* Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \\> 50 mL\u002Fmin\u002F1.73m2 (calculated or estimated) or GFR (mL\u002Fmin\u002F1.72m2) \\>30% of predicted normal for age.\n\nNormal GFR in Children and Young Adults (Age) : Mean GFR +- SD (mL\u002Fmin\u002F1.73 m2)\n\n1 week: 40.6 + \u002F - 14.8 2-8 weeks: 65.8 + \u002F - 24.8 \\>8 weeks: 95.7 + \u002F - 21.7 2-12 years: 133.0 + \u002F - 27.0 13-21 years (males): 140.0 + \u002F - 30.0 13-21 years (females: 126.0 + \u002F - 22.0\n\nGFR, glomerular filtration rate; SD, standard deviation greater than 2 years old: Normal GFR is 100 mL\u002F min. Infants: GFR must be corrected for body surf ace area.\n\nFor metabolic diseases: disease status to be evaluated according to EBMT Handbook \\[45\\].\n\nGraft Criteria\n\nCB units will be selected according to the current MSKCC unit selection algorithm. High resolution 8 allele HLA typing and recipient HLA antibody profile will be performed. Cord unit selection will occur based on HLA-match, total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body weight. The cord bank of origin will also be considered. Donor specific HLA antibodies, if present, will also be taken into consideration and may influence the selection of the graft. CB graft will consist of one or two CB units (CBU) based on MSKCC selection algorithm.\n\n* Each CB unit must be at least 3\u002F8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.\n* For malignant diseases follow MSKCC CBU selection algorithm\n* For non-malignant diseases, CBU will be required to have \\> 5 x 107 TNC\u002Fkg; high HLA allele level match is preferable\n\nParticipant Exclusion Criteria:\n\n* Inadequate performance status\u002F organ function.\n* Advanced metabolic disease (EBMT handbook).\n* Active CNS leukemic involvement.\n* Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.\n* Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.\n* Autologous stem cell transplant within the preceding 6 months.\n* Any prior allogeneic stem cell transplant.\n* Active and uncontrolled infection (bacterial\u002Ffungal\u002Fviral) at time of transplantation.\n* HIV infection.\n* Seropositivity for HTLV-1.\n* Pregnancy or breast feeding.\n* Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.","21 Years",{"count":130,"type":21},31,[24],"This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.",[134,16,135,136,137,138,28,27,104],"AML","MDS","MPD Withou Myelofibrosis","NHL or HL","Inherited Metabolic Disorders",[140,134,141,135,142,143,144,145,146,147,148,149],"Acute myelogenous leukemia","Myelodysplasia","Myeloproliferative Disorder","Therapy-Related AML and MDS","Therapy-Related Acute Myeloid Leukemia","T-AML","High-risk cytogenetics","Cord Blood Transplantation","20-480","Memorial Sloan Kettering Cancer Center","2026-03-12",{"date":152,"type":38},"2026-03-13",{"date":154,"type":38},"2020-11-20",{"date":156,"type":21},"2026-12-20",{"name":149,"class":45},{"id":159,"slug":160,"hasResults":11,"nctId":161,"briefTitle":162,"officialTitle":163,"acronym":4,"eligibilityCriteria":164,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":165,"enrollmentInfo":166,"targetDuration":4,"studyType":22,"phases":168,"briefSummary":169,"conditions":170,"keywords":4,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":178,"lastUpdatePostDateStruct":179,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":120},"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":167,"type":21},48,[24],"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.",[171,172,173,174,175,27,176,177],"Fanconi Anemia","Severe Aplastic Anemia","Myelodysplastic Syndromes","T Cell Receptor Alpha\u002FBeta Depletion","Telomere Biology Disorder","Dyskeratosis Congenita","Telomere Biology Disorders","2026-01-29",{"date":180,"type":38},"2026-01-30",{"date":182,"type":38},"2018-11-13",{"date":184,"type":21},"2029-01-05",{"name":186,"class":45},"Masonic Cancer Center, University of Minnesota",{"id":188,"slug":189,"hasResults":11,"nctId":190,"briefTitle":191,"officialTitle":191,"acronym":4,"eligibilityCriteria":192,"healthyVolunteers":11,"sex":16,"minAge":193,"maxAge":18,"enrollmentInfo":194,"targetDuration":4,"studyType":22,"phases":196,"briefSummary":197,"conditions":198,"keywords":205,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":207,"lastUpdatePostDateStruct":208,"startDateStruct":210,"completionDateStruct":212,"leadSponsor":214,"locationsCount":120},"100380322","phase-2-regenerative-medicine-to-restore-hematopoiesis-and-immune-function-in-immunodeficiencies-and-inherited-bone-marrow-failures-100380322","NCT04232085","Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures","Inclusion Criteria\n\nCohort A:\n\nPrimary Immune Deficiencies with indication for HCT:\n\n* Chronic granulomatous disease (CGD)\n* Wiskott-Aldrich syndrome (WAS)\n* Hyper-IgM syndrome\n* Common variable immunodeficiency (CVID)\n* Leukocyte adhesion deficiency-1 (LAD-1)\n* Severe Combined Immunodeficiency (SCID)\n* CTLA-4 deficiency\n* CARD9 deficiency\n* DOCK8 deficiency\n\nImmune Dysregulatory Syndromes:\n\n* Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome\n* Hemophagocytic lymphohistiocytosis (HLH) or related disorder with indication for transplant\n* CAEBV: Patients with chronic EBV infection (CAEBV) with indication for BMT:\n\nInherited Bone marrow failure disorders\n\n* Congenital amegakaryocytic thrombocytopenia (CAMT)\n* Diamond Blackfan anemia (DBA)\n* Shwachman Diamond Syndrome (SDS)\n* Thrombocytopenia Absent Radii (TAR)\n* Glanzmans thrombasthenia (GT)\n* Kostmann syndrome\n* Other indications and\u002For other PID, IDS, and IBMFS diagnoses as deemed appropriate by the PI.\n\nCohort B: Short telomere syndrome\n\nCohort C: Confirmed diagnosis of Fanconi anemia or non-Fanconi DNA-dsb repair disorders\n\n* Fanconi anemia\n* Non-Fanconi DNA-dsb repair disorders\n* Cerunnos-XRCC4-like factor deficiency (XLF or NHEJ1)\n* DNA ligase IV deficiency (LIG4)\n* Nijmegen breakage syndrome (NBS)\n* Increased DNA breakage after exposure of patient cells to DNA cross-linking agents such as diepoxybutane or mitomycin C and germline mutation(s) in an identified Fanconi pathway gene.\n\nAvailable donor as follows:\n\n* Fully HLA matched sibling or other first-degree family member.\n* Fully HLA matched unrelated 10\u002F10 donor using high-resolution DNA-based typing at the following genetic loci: HLA-A, -B, -C, DRB1, and DQB1.\n* Mismatched unrelated donor at 8 or 9\u002F10 alleles, using high-resolution typing as above.\n* HLA-haploidentical family members of any degree who match at least one allele of each of the following genetic loci: HLA-A, -B, -C, DRB1, and DQB1. A minimum match of 5\u002F10 is therefore required, and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.\n* The patient and\u002For legal guardian must sign informed consent for BMT.\n* Patients with adequate organ function as measured by\n* Cardiac: Left ventricular ejection fraction (LVEF) at rest must be ≥ 35%. For patients aged \\\u003C13 years, shortening fraction (SF) \\> 25% by echocardiogram or LVEF by MUGA may be used.\n* Hepatic: Bilirubin ≤ 3.0 mg\u002FdL; and ALT, AST, and Alkaline Phosphatase \\\u003C 5 x ULN.\n* Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) \\> 40 mL\u002Fmin\u002F1.73m2.\n* Pulmonary: PFT with FEV1 and FVC \\>\u002F= 50% of normal and DLCO corrected for Hgb \\>\u002F= 40% of normal. Patients unable to undergo PFTs should have stable resp status with SaO2 \\>90% on a max of 2L\u002Fmin supplemental O2.\n* Karnofsky or Lansky performance status ≥70%\n* 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\n* Patients will not be excluded on the basis of sex, racial or ethnic background.\n* Positive leukocytotoxic crossmatch.\n* Prior allogeneic stem cell transplant.\n* 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. The investigators recognize that patients with CAEBV may have ongoing EBV viremia at the time of initiating pre-transplant therapy, but other patients should have no uncontrolled bacterial, viral, or fungal infections.\n* Diagnosis of idiopathic aplastic anemia\n* Seropositivity for the human immunodeficiency virus (HIV)\n* Active Hepatitis B or C determined by serology and\u002For NAT\n* Female patients who are diagnosed as pregnant by beta bHCG testing (per institutional practice) or who are breast-feeding.\n* Active malignancy or within the timeframe for significant concern for relapse of prior malignancy\n* For Cohort B and C: liver biopsy (if performed, not required) with moderate-severe fibrosis\u002Fcirrhosis\n\nDonor Eligibility\n\n* Donor must be medically, socially, and psychologically fit to donate\n* Bone marrow is the preferred graft source, however, PBSCs may be requested. In particular, PBSCs may be preferred for patients with active viral reactivations and\u002For for patients who would benefit from a higher count in the graft. Cord blood is not permitted.\n* First-degree relatives should be tested for degree of HLA match, CMV serology, ABO type, and complete blood count (CBC). An unrelated donor search should be initiated at the time the patient is referred for BMT.\n* Age ≥5 years\n* Donors must meet the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT).\n* Lack of recipient anti-donor HLA antibody in recipient\n* Note: In some instances, low level, non-cytotoxic HLA specific antibodies may be permissible if found to be at a level well below that detectable by flow cytometry. This will be decided on a case-by-case basis by the PI and one of the immunogenetics directors.\n* In inherited disorders, family members must be tested for carrier and disease status of the underlying disorders. In the event that family members are unaffected carriers, eligibility as donors will be decided upon by the PI on a case-by-case basis\n* In the event that two or more eligible donors are identified, the donor will be selected per institutional standards. Suggested criteria include the following:\n* Related is preferred over unrelated.\n* The potential donor that is youngest in age is preferred.\n* For CMV seronegative patients, a CMV seronegative donor is preferred. For CMV seropositive patients, a CMV seropositive donor is preferred.\n* Red blood cell compatibility, in order of preference:\n* RBC cross match compatible Minor ABO incompatibility, Major ABO incompatibility\n* If the patient is male, male donors are preferred.","4 Months",{"count":195,"type":21},27,[24],"Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.",[199,200,27,201,171,202,203,176,175,204],"Primary Immune Deficiency Disorder","Immune Deficiency Disease","Short Telomere Length","Non Fanconi DNA-DSB Repair Disorder","Hoyeraal-Hreidarsson Syndrome","Short Telomere Syndrome",[206],"Bone Marrow Transplantation","2025-11-21",{"date":209,"type":38},"2025-11-28",{"date":211,"type":38},"2020-02-12",{"date":213,"type":21},"2028-12-31",{"name":215,"class":45},"Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins",{"id":217,"slug":218,"hasResults":11,"nctId":219,"briefTitle":220,"officialTitle":220,"acronym":221,"eligibilityCriteria":222,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":223,"enrollmentInfo":224,"targetDuration":226,"studyType":59,"phases":4,"briefSummary":227,"conditions":228,"keywords":239,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":248,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":120},"100535343","european-rare-blood-disorders-platform-enrol-100535343","NCT06250595","European Rare Blood Disorders Platform (ENROL)","ENROL","Inclusion Criteria:\n\n* Patients must meet all of the following criteria to be included in the ENROL Registry\n* Age from 0-100, both female and male\n* Diagnosed as RHDs according to ORPHANET classification\n* Able and willing to provide written informed consent (patient or legal representative for minors) if needed according to national legislation.\n\nExclusion Criteria:\n\n* Patients diagnosed as traits or trait conditions for other recessive RHDs","100 Years",{"count":225,"type":21},37090,"15 Years","ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform.\n\nENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data.\n\nAccordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when \u002F where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies.\n\nThe standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources.\n\nMoreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal.\n\nENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670",[229,27,230,231,232,233,234,93,235,236,237,238],"Anemia","Bleeding Disorder","Iron Metabolism Disorders","Myeloma","Lymphoid Neoplasm","Myeloma, Malignant","Anemia, Sickle Cell","Thalassemia","Blood Cancer","Red Cell Membrane and Enzyme Abnormalities",[229,27,240,241,242,243,244,93,245,236,246],"Bleeding disorder","Iron metabolism disorder","Myeloid","Lymphoid","Blood cancer","Red Cell membrane and Enzyme Abnormalities","Sickle Cell Disease","2024-02-06",{"date":249,"type":38},"2024-02-09",{"date":251,"type":38},"2022-07-01",{"date":253,"type":21},"2037-07",{"name":255,"class":45},"Hospital Universitari Vall d'Hebron Research Institute"]