[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemoglobinopathies\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemoglobinopathies":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,47,78,103,140,164,205,238,268,304,331,361],{"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":55,"sex":16,"minAge":4,"maxAge":85,"enrollmentInfo":86,"targetDuration":4,"studyType":22,"phases":88,"briefSummary":89,"conditions":90,"keywords":4,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":102},"100583131","phase-2-allo-hsct-for-high-risk-hemoglobinopathies-100583131","NCT06872333","Allo HSCT for High Risk Hemoglobinopathies","Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders","Inclusion Criteria:\n\n* Sickle Cell Disease (SCD)\n* SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents\u002Fpatient must be counseled as to the risks and benefits and provide their voluntary informed consent\n* Transfusion Dependent Alpha- or Beta- Thalassemia\n* Diamond Blackfan Anemia\n* Other Non-Malignant Hematologic Disorders\n* Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation.\n* Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.\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 30 days of the start of treatment\n* HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.\n* Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted\n* Known allergy to any of the study components\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","55 Years",{"count":87,"type":21},62,[24],"A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.",[91,92,28],"Graft Failure","Sickle Cell Disease","2026-06-01",{"date":95,"type":38},"2026-06-04",{"date":97,"type":38},"2024-11-19",{"date":99,"type":21},"2032-06-01",{"name":101,"class":45},"Masonic Cancer Center, University of Minnesota",1,{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":111,"maxAge":112,"enrollmentInfo":113,"targetDuration":4,"studyType":22,"phases":115,"briefSummary":117,"conditions":118,"keywords":124,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":139},"100295674","phase-1-reduced-intensity-conditioning-and-familial-hla-mismatched-bmt-for-non-malignant-disorders-100295674","NCT03128996","Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders","A Phase I\u002FII Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders","FAM BMT","Inclusion Criteria:\n\n* Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy\n* For patients with sickle cell disease, must have one of the following severe manifestations:\n\n  1. Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy\n  2. Recurrent acute chest syndrome with significant respiratory compromise each time\n  3. Sickle nephropathy\n  4. Recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity\n  5. Red cell alloimmunization with the need for chronic transfusions\n  6. Recurrent osteonecrosis or multiple joint involvement from avascular necrosis\n* Patients with sickle cell disease must have hemoglobin S \\\u003C 30% within 30 days prior to beginning alemtuzumab\n* Age \\\u003C\u002F= 20.99 years at the time of enrollment\n* Performance score \\>\u002F= 50\n* Left ventricular ejection fraction \\> 40% or left ventricular shortening fraction \\> 26% by echocardiogram\n* DLCO \\> 40% (corrected for hemoglobin) or pulse oximetry with a baseline O2 saturation of \\>\u002F= 90% on room air if too young to perform PFTs\n* Serum creatinine \\\u003C\u002F= 1.5x upper limit of normal for age and\u002For GFR \\> 70 mL\u002Fmin\u002F1.73m2\n* Direct bilirubin \\\u003C 2x upper limit of normal for age\n* ALT and AST \\\u003C 5x upper limit of normal for age\n* Participants who have or are receiving \\>\u002F= 8 packed red blood cell transfusions for \\>\u002F= 1 year or \\>\u002F= 20 packed red blood cell transfusions (lifetime cumulative) will undergo liver MRI for estimation of hepatic iron content.\n\n  1\\. Liver biopsy is indicated for hepatic iron content \\>\u002F= 7mg Fe\u002Fmg liver dry weight by liver MRI. Histologic examination of the liver must document for the absence of cirrhosis, bridging fibrosis, and active hepatitis\n* Female subjects of childbearing potential, must agree to practice 2 methods of contraception at the same time from the time of signing of informed consent through 12 months post transplant. Male subjects must agree to practice effective barrier contraception or practice true abstinence from the time of signing informed consent through 12 months post transplant.\n* Written informed consent must be obtained from all recipients in accordance with the guidelines of the institution's Human Studies Committee.\n\nExclusion Criteria:\n\n* Patients who have an HLA-identical sibling who is able and willing to donate bone marrow\n* Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis\n* Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment\n* Evidence of HIV infection or known HIV positive serology\n* Patients who have received a previous stem cell transplant\n* Patients who have received an investigational drug or device or off-label use of a drug or device within 3 months of enrollment\n* Females who are pregnant or breast feeding\n* Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma)","1 Day","21 Years",{"count":114,"type":21},29,[116,24],"PHASE1","This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.",[119,120,121,122,28,123],"Severe Sickle Cell Disease","Bone Marrow Failure Syndromes","Metabolic Disorders","Immunologic Disorders","Non-malignant Disorders",[125,126,127,128,129],"Bone marrow transplant","Transplant","Transplantation","Reduced Intensity","Familial HLA mismatched","2026-05-26",{"date":132,"type":38},"2026-05-29",{"date":134,"type":38},"2017-03-20",{"date":136,"type":21},"2033-04",{"name":138,"class":45},"Washington University School of Medicine",4,{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":144,"acronym":4,"eligibilityCriteria":145,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":146,"targetDuration":4,"studyType":59,"phases":4,"briefSummary":148,"conditions":149,"keywords":150,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":155,"lastUpdatePostDateStruct":156,"startDateStruct":158,"completionDateStruct":160,"leadSponsor":162,"locationsCount":102},"100414024","discarded-bone-marrow-for-hematology-research-100414024","NCT04671212","Discarded Bone Marrow for Hematology Research","Inclusion Criteria:\n\n* Patients of any age receiving orthopedic surgery for clinical management that involve bone marrow containing bone discard.\n* Patients receiving orthopedic surgery for clinical management will be considered for this study if they have the following diagnosis and criteria:\n\n  * Homozygous S\u002FS disease or doubly heterozygous for S and β thalassemia who are two years or older are eligible.\n  * HbE-β- thalassemia or homozygous (severe) β-thalassemia. including those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Diagnostic criteria include standard hematological parameters, red cell indices, hemoglobin electrophoresis and quantitative determination of HbF and HbA2.\n\nExclusion Criteria:\n\n* Active, acute manifestations of sickle cell disease including painful crisis, acute chest syndrome, cerebrovascular events or active infection.\n* Pregnant women will not be eligible for study enrollment\n* Inability or unwillingness of the research participant or legal guardian\u002Frepresentative to give written informed consent will preclude enrollment on this research protocol.\n* Platelet count \\\u003C 150,000\u002Fmm\\^3\n* Neutrophil count \\\u003C 2000\u002Fmm\\^3\n* Neutrophil count \\\u003C 1000\u002Fmm\\^3 for patients on hydroxyurea therapy\n* Prothrombin Time \\> 17 seconds\n* Partial thromboplastin Time \\> 43 seconds\n* History of excessive bleeding in the context of previous procedures including surgery and dental extractions",{"count":147,"type":21},50,"The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals. The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant.\n\nSecondary objectives\n\n* To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies (e.g. sickle cell disease (SCD) and β-thalassemia).\n* To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies.\n* To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies.",[28],[151,152,153,28,154],"Bone samples","Bone marrow","Discarded","Non-hemoglobinopathies","2026-04-29",{"date":157,"type":38},"2026-05-05",{"date":159,"type":38},"2022-07-26",{"date":161,"type":21},"2035-01",{"name":163,"class":45},"St. Jude Children's Research Hospital",{"id":165,"slug":166,"hasResults":11,"nctId":167,"briefTitle":168,"officialTitle":169,"acronym":4,"eligibilityCriteria":170,"healthyVolunteers":11,"sex":16,"minAge":171,"maxAge":172,"enrollmentInfo":173,"targetDuration":4,"studyType":22,"phases":175,"briefSummary":176,"conditions":177,"keywords":190,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":102},"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":174,"type":21},100,[116,24],"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.",[178,179,180,181,182,183,184,185,186,187,28,27,188,189],"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",[191,192,193,194,195],"alpha beta T cell depletion","CD45RA","CD45RO","GVHD prevention","Memory T cells","2026-04-13",{"date":198,"type":38},"2026-04-15",{"date":200,"type":38},"2025-03-21",{"date":202,"type":21},"2032-03",{"name":204,"class":45},"Children's Hospital of Philadelphia",{"id":206,"slug":207,"hasResults":11,"nctId":208,"briefTitle":209,"officialTitle":210,"acronym":4,"eligibilityCriteria":211,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":212,"enrollmentInfo":213,"targetDuration":4,"studyType":22,"phases":215,"briefSummary":216,"conditions":217,"keywords":219,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":229,"lastUpdatePostDateStruct":230,"startDateStruct":232,"completionDateStruct":234,"leadSponsor":236,"locationsCount":237},"100126972","phase-1-campathfludarabinemelphalan-transplant-conditioning-for-non-malignant-diseases-100126972","NCT00920972","Campath\u002FFludarabine\u002FMelphalan Transplant Conditioning for Non-Malignant Diseases","A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime","Inclusion Criteria:\n\nStratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8\u002F8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nStratum 3: Patient must have a hemoglobinopathy receiving 7\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nStratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7\u002F8 HLA-matched bone marrow or 5-8\u002F8 HLA-matched UCB. Related or unrelated.\n\nAll strata:\n\n* Recipient age \\\u003C 21 years\n* Lansky\u002FKarnofsky \\>\u002F= 40\n* Adequate pulmonary, renal, liver, and other organ function as defined in protocol\n* Negative pregnancy test\n* Adequate total nucleated cell or CD34+ dose of product as defined in protocol\n* If sickle cell, Hemoglobin S \\\u003C30%\n\nExclusion Criteria:\n\n* HIV positive\n* Invasive infection\n* Pregnancy\u002Flactating","20 Years",{"count":214,"type":21},220,[116,24],"The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.",[121,218,28,123],"Hematologic, Immune, or Bone Marrow Disorders",[152,126,127,220,221,222,223,224,225,226,227,228],"Hematopoietic","Umbilical cord","Related","Unrelated","Reduced","Non-myeloablative","Nonmyeloablative","Non-malignant","Nonmalignant","2026-03-24",{"date":231,"type":38},"2026-03-25",{"date":233,"type":38},"2001-12",{"date":235,"type":21},"2031-12",{"name":138,"class":45},28,{"id":239,"slug":240,"hasResults":11,"nctId":241,"briefTitle":242,"officialTitle":243,"acronym":4,"eligibilityCriteria":244,"healthyVolunteers":11,"sex":16,"minAge":245,"maxAge":246,"enrollmentInfo":247,"targetDuration":4,"studyType":22,"phases":249,"briefSummary":251,"conditions":252,"keywords":4,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":258,"lastUpdatePostDateStruct":259,"startDateStruct":261,"completionDateStruct":263,"leadSponsor":265,"locationsCount":77},"100475948","phase-3-evaluation-of-efficacy-and-safety-of-a-single-dose-of-ctx001-in-participants-with-transfusion-dependent--thalassemia-and-severe-sickle-cell-disease-100475948","NCT05477563","Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease","A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease","Key Inclusion Criteria:\n\n* Participants with TDT and SCD:\n* Eligible for autologous stem cell transplant as per investigator's judgment.\n* Participants with TDT:\n* Diagnosis of TDT as defined by:\n* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia\u002Fhemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning\n* History of at least 100 milliliter (mL)\u002Fkilograms (kg)\u002Fyear or 10 units\u002Fyear of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening\n* Participants with SCD:\n* Diagnosis of severe SCD as defined by:\n* Documented SCD genotypes\n* History of at least two severe VOCs events per year for the previous two years prior to enrollment\n\nKey Exclusion Criteria:\n\n* Participants with TDT and SCD:\n* A willing and healthy 10\u002F10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement\n* Prior hematopoietic stem cell transplant (HSCT)\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator\n* Participants with TDT:\n* Participants with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications\n* Participants with sickle cell β-thalassemia variant\n* Participants with SCD:\n* History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.","12 Years","35 Years",{"count":248,"type":21},26,[250],"PHASE3","This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.",[253,254,255,256,28,92,257],"Beta-Thalassemia","Thalassemia","Hematologic Diseases","Genetic Diseases, Inborn","Sickle Cell Anemia","2026-03-18",{"date":260,"type":38},"2026-03-23",{"date":262,"type":38},"2022-08-02",{"date":264,"type":21},"2027-06-09",{"name":266,"class":267},"Vertex Pharmaceuticals Incorporated","INDUSTRY",{"id":269,"slug":270,"hasResults":11,"nctId":271,"briefTitle":272,"officialTitle":273,"acronym":4,"eligibilityCriteria":274,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":112,"enrollmentInfo":275,"targetDuration":4,"studyType":22,"phases":277,"briefSummary":278,"conditions":279,"keywords":285,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":296,"lastUpdatePostDateStruct":297,"startDateStruct":299,"completionDateStruct":301,"leadSponsor":303,"locationsCount":102},"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.",{"count":276,"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.",[280,16,281,282,283,284,28,27,189],"AML","MDS","MPD Withou Myelofibrosis","NHL or HL","Inherited Metabolic Disorders",[286,280,287,281,288,289,290,291,292,293,294,295],"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":298,"type":38},"2026-03-13",{"date":300,"type":38},"2020-11-20",{"date":302,"type":21},"2026-12-20",{"name":295,"class":45},{"id":305,"slug":306,"hasResults":11,"nctId":307,"briefTitle":308,"officialTitle":308,"acronym":4,"eligibilityCriteria":309,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":85,"enrollmentInfo":310,"targetDuration":4,"studyType":22,"phases":312,"briefSummary":314,"conditions":315,"keywords":318,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":323,"lastUpdatePostDateStruct":324,"startDateStruct":326,"completionDateStruct":328,"leadSponsor":330,"locationsCount":102},"100183644","second-or-greater-allogeneic-hematopoietic-stem-cell-transplant-using-reduced-intensity-conditioning-ric-100183644","NCT01666080","Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)","Inclusion Criteria:\n\n* Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of \"insufficiency of donor chimerism\" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.\n* Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program\n\n  * Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.\n  * Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.\n  * Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease.\n\nAt the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.\n\n* Age, Performance Status, Consent\n\n  * Age: 0 to 55 years\n  * Consent: voluntary written consent (adult or parental\u002Fguardian)\n\nExclusion Criteria:\n\n* Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI\n* Pregnant or breastfeeding\n* Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted\n* HIV positive\n* While it would be advantageous to begin therapy on this second transplant regimen \\> 6 months following a prior myeloablative regimen or \\>2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.",{"count":311,"type":21},30,[313],"NA","This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HCT.",[316,28,317],"Hematologic Disorders","Immunodeficiencies",[319,320,321,322],"second stem cell transplant","donor hematopoietic engraftment","hematopoietic stem cell transplantation","inherited metabolic disorder","2025-07-23",{"date":325,"type":38},"2025-07-24",{"date":327,"type":38},"2012-08",{"date":329,"type":21},"2027-06",{"name":101,"class":45},{"id":332,"slug":333,"hasResults":11,"nctId":334,"briefTitle":335,"officialTitle":336,"acronym":4,"eligibilityCriteria":337,"healthyVolunteers":11,"sex":16,"minAge":245,"maxAge":246,"enrollmentInfo":338,"targetDuration":4,"studyType":22,"phases":340,"briefSummary":342,"conditions":343,"keywords":346,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":352,"lastUpdatePostDateStruct":353,"startDateStruct":355,"completionDateStruct":357,"leadSponsor":359,"locationsCount":102},"100524336","early-phase-1-safety-and-efficacy-of-rm-004-cells-for-hemoglobin-h-constant-spring-disease-100524336","NCT06107400","Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease","Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease","Inclusion Criteria:\n\n1. Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment.\n2. At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender.\n3. History of at least 100 mL\u002Fkilograms (kg)\u002Fyear of packed red blood cells (pRBC) transfusions in the prior 2 years before screening.\n4. Subjects diagnosed with Hemoglobin H-Constant Spring disease (--\u002FααCS) with HBA2 c.427T\\>C mutation.\n\nExclusion Criteria:\n\n1. Subject who has an available HLA-matched\u002Fwell-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT).\n2. Prior HSCT or gene therapy.\n3. History of severe hemorrhagic disease.\n4. Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.",{"count":339,"type":21},5,[341],"EARLY_PHASE1","The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.",[344,28,345],"Alpha Thalassemia Hemoglobin H Constant Spring","Hereditary Diseases",[347,348,349,350,351],"RM-004","Hemoglobin H-Constant Spring disease","Alpha-Thalassemia","Gene therapy","CBE","2024-05-30",{"date":354,"type":38},"2024-06-03",{"date":356,"type":38},"2023-10-08",{"date":358,"type":21},"2026-10-31",{"name":360,"class":45},"The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army",{"id":362,"slug":363,"hasResults":11,"nctId":364,"briefTitle":365,"officialTitle":365,"acronym":366,"eligibilityCriteria":367,"healthyVolunteers":11,"sex":16,"minAge":368,"maxAge":4,"enrollmentInfo":369,"targetDuration":4,"studyType":59,"phases":4,"briefSummary":371,"conditions":372,"keywords":375,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":380,"lastUpdatePostDateStruct":381,"startDateStruct":383,"completionDateStruct":385,"leadSponsor":387,"locationsCount":248},"100500658","study-of-the-role-of-genetic-modifiers-in-hemoglobinopathies-100500658","NCT05799118","Study of the Role of Genetic Modifiers in Hemoglobinopathies","INHERENT","Inclusion Criteria:\n\n* Clinical diagnosis of an inherited hemoglobinopathy, including sickle cell disease (SCD), β-thalassemia, and α-thalassemia; all genotypes will be considered.\n* Age ≥ 2 years old at the time of the collection of the phenotypic data.\n* There will be no limits on study participants in terms of gender, ethnicity, morbidities.\n\nExclusion Criteria:\n\n* Patients treated with stem cell transplantation or genetic therapy.\n* Age \\\u003C 2 years old at the time of the collection of the phenotypic data.\n* Patient or legal representative for minors unwilling or unable to give consent.","2 Years",{"count":370,"type":21},30000,"This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).",[92,373,374,28],"Thalassemia, Beta","Thalassemia Alpha",[376,377,378,379],"GWAS","thalassemia","sickle cell disease","genetic modifiers","2024-03-19",{"date":382,"type":38},"2024-03-20",{"date":384,"type":38},"2022-10-01",{"date":386,"type":21},"2027-09-30",{"name":388,"class":45},"Cyprus Institute of Neurology and Genetics"]