Anemia, Diamond-Blackfan: all 56 studies

Page 1 of 1. Open to participants first (10), then studies with results posted, then the rest; newest registration first within each.

• Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan AnemiaRecruitingNCT07476183Phase 1
• Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)RecruitingNCT07186179
• French National Registry of Bone Marrow FailuresRecruitingNCT04781790
• Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT04528355
• AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell DepletionRecruitingNCT04099966Phase 2
• T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other AnemiasRecruitingNCT03653338Phase 1/2
• Familial Investigations of Childhood Cancer PredispositionRecruitingNCT03050268
• Investigation of the Genetics of Hematologic DiseasesRecruitingNCT02720679
• Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 2
• Cancer in Inherited Bone Marrow Failure SyndromesRecruitingNCT00027274
• Treatment of Refractory Diamond-Blackfan Anemia With EltrombopagCompletedNCT04269889Phase 1/2Results posted
• The Use of Trifluoperazine in Transfusion Dependent DBATerminatedNCT03966053Phase 1/2Results posted
• Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyTerminatedNCT03733249Phase 1/2Results posted
• Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/2Results posted
• Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerTerminatedNCT03333486Phase 2Results posted
• Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsTerminatedNCT02512679Phase 2Results posted
• Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesTerminatedNCT02231710Phase 1Results posted
• Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell TransplantTerminatedNCT02065869Phase 1/2Results posted
• Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesCompletedNCT01529827Phase 2Results posted
• Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan AnemiaTerminatedNCT01464164Phase 1/2Results posted
• Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan AnemiaCompletedNCT01362595Phase 1/2Results posted
• Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationTerminatedNCT01319851Not applicableResults posted
• Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCsCompletedNCT00957931Phase 2Results posted
• Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersCompletedNCT00919503Phase 2Results posted
• Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant DisordersCompletedNCT00744692Phase 1Results posted
• Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron OverloadCompletedNCT00600938Phase 2Results posted
• Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersCompletedNCT00301834Phase 2Results posted
• Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory CytopeniasCompletedNCT00244010Not applicableResults posted
• Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron OverloadCompletedNCT00235391Phase 3Results posted
• Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan AnemiaCompletedNCT00229619Phase 2Results posted
• Stem Cell Transplant for Bone Marrow Failure SyndromesCompletedNCT00176878Phase 2/3Results posted
• Stem Cell Transplant for HemoglobinopathyCompletedNCT00176852Phase 2/3Results posted
• A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow FailureTerminatedNCT00001962Phase 2Results posted
• Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
• Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)CompletedNCT04965597Phase 2
• L-leucine in Diamond Blackfan Anemia PatientsStatus unknownNCT02386267Phase 2
• Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesTerminatedNCT02179359Not applicable
• CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell TransplantActive, not recruitingNCT02061800Phase 1/2
• CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationActive, not recruitingNCT01966367Phase 1/2
• Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesCompletedNCT01917708Phase 1
• Multi-Center Study of Iron Overload: Survey Study (MCSIO)CompletedNCT01913548
• Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood DisordersCompletedNCT01758042Not applicable
• Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 1
• Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesWithdrawnNCT01419704Phase 1/2
• Multi-Center Study of Iron Overload: Pilot StudyCompletedNCT01114776
• A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe ToxicityCompletedNCT01044186Phase 2
• Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron OverloadCompletedNCT00673608Phase 4
• Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of ErythropoiesisCompletedNCT00578435Phase 2
• Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionCompletedNCT00305708Phase 1/2
• Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic CancerTerminatedNCT00290628Not applicable
• A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron OverloadCompletedNCT00171821Phase 3
• Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic AnemiasCompletedNCT00061763Phase 2
• Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan AnemiaCompletedNCT00011505Phase 2
• Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic DisordersCompletedNCT00005893Not applicable
• Stem Cell Transplantation (SCT) for Genetic DiseasesCompletedNCT00004378Not applicable
• Medical Treatment for Diamond Blackfan AnemiaCompletedNCT00001749Phase 2

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