13 completed, 3 recruiting, 3 terminated, 1 active not recruiting. 11 Phase 2, 6 Phase 1, 3 Not applicable, 2 Phase 3, 1 Phase 4.
Follow Diamond-Blackfan Anemia: new trials by email Literature Review of Diamond-Blackfan Anemia trials Research Report on Diamond-Blackfan Anemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) Treosulfan, Fludarabine Phosphate, Tacrolimus - Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia | Phase 2 | Completed | Fred Hutchinson Cancer Center | 40 | 2022-04-19 | 2026-03-09 |
| T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea - Sickle Cell Anemia, Beta-thalassemia Major | Phase 1 / Phase 2 | Recruiting | Paul Szabolcs | 5 | 2018-08-02 | 2026-08-13 |
| Familial Investigations of Childhood Cancer Predisposition Acute Leukemia, Adenomatous Polyposis | Recruiting | St. Jude Children's Research Hospital | 1500 | 2017-04-06 | 2026-06-17 | |
| Investigation of the Genetics of Hematologic Diseases Bone Marrow Failure Syndromes, Erythrocyte Disorder | Recruiting | St. Jude Children's Research Hospital | 1716 | 2016-06-17 | 2026-06-09 | |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation CD34 Stem Cell Selection Therapy - Bone Marrow Failure Syndrome, Severe Aplastic Anemia | Phase 1 / Phase 2 | Active not recruiting | Diane George | 37 | 2013-03-01 | 2025-08-14 |
| Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies fludarabine phosphate, melphalan, total-body irradiation - Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission | Phase 2 | Completed | Roswell Park Cancer Institute | 94 | 2012-02-28 | 2019-09-24 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies Enriched Hematopoetic Stem Cell Infusion - Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies | Phase 1 / Phase 2 | Withdrawn | Talaris Therapeutics Inc. | 2011-05-01 | 2023-03-03 | |
| Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation Alefacept - Thalassemia, Sickle Cell Disease | Not applicable | Terminated | Emory University | 3 | 2010-09-01 | 2017-07-27 |
| Multi-Center Study of Iron Overload: Survey Study (MCSIO) Sickle Cell Disease, Thalassemia | Completed | UCSF Benioff Children's Hospital Oakland | 423 | 2010-03-31 | 2020-09-23 | |
| Multi-Center Study of Iron Overload: Pilot Study Sickle Cell Disease, Thalassemia | Completed | UCSF Benioff Children's Hospital Oakland | 20 | 2009-11-01 | 2020-09-24 | |
| Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs Bone marrow transplantation, Mesenchymal Stromal Cells - Sickle Cell Disease, Thalassemia | Phase 2 | Completed | Stanford University | 6 | 2009-03-01 | 2018-08-06 |
| Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload deferasirox - Hemoglobinopathies, Myelodysplastic Syndromes | Phase 4 | Completed | Novartis | 118 | 2007-11-01 | 2017-02-23 |
| Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3 - Stem Cell Transplantation, Bone Marrow Transplantation | Phase 2 | Terminated | Children's Hospital Los Angeles | 20 | 2007-02-01 | 2017-02-27 |
| Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias Allogeneic stem cell transplant - Anemia, Aplastic, Amegakaryocytic Thrombocytopenia | Not applicable | Completed | St. Jude Children's Research Hospital | 4 | 2005-10-01 | 2017-05-30 |
| Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders alemtuzumab, busulfan, cyclosporine - Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia | Phase 2 | Completed | University of California, San Francisco | 35 | 2005-01-01 | 2017-09-28 |
| Stem Cell Transplant for Hemoglobinopathy Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide - Sickle Cell Disease, Thalassemia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 22 | 2002-06-01 | 2020-02-27 |
| 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 Remission anti-thymocyte globulin, busulfan, fludarabine phosphate - Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia | Phase 1 / Phase 2 | Completed | University of California, San Francisco | 40 | 2000-08-01 | 2012-11-12 |
| Stem Cell Transplant for Bone Marrow Failure Syndromes Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation - Diamond-Blackfan Anemia, Kostmann's Neutropenia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 10 | 2000-06-01 | 2017-12-28 |
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Anemia, Diamond-Blackfan (21) · Anemia, Sickle Cell (10) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (9) · Shwachman-Diamond Syndrome (7) · Thalassemia (7) · Anemia, Aplastic (6) · Fanconi Anemia (6) · Bone Marrow Failure Disorders (5) · Myelodysplastic Syndromes (5) · Congenital amegakaryocytic thrombocytopenia (4) · Dyskeratosis Congenita (4) · Leukemia, Myeloid, Chronic-Phase (4) · Wiskott-Aldrich Syndrome (4) · Granulomatous Disease, Chronic (3) · Hodgkin Disease (3)
Anemia, Hypoplastic, Congenital (21) · Bone Marrow Diseases (21) · Congenital Bone Marrow Failure Syndromes (21) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Hematologic Diseases (21) · Hemic and Lymphatic Diseases (21) · Red-Cell Aplasia, Pure (21) · Anemia (20) · Genetic Diseases, Inborn (20) · Bone Marrow Failure Disorders (16) · Anemia, Aplastic (15) · Anemia, Hemolytic (13) · Nutritional and Metabolic Diseases (13) · Metabolic Diseases (12) · Anemia, Hemolytic, Congenital (11)
fludarabine phosphate (7) · Antilymphocyte Serum (5) · Busulfan (5) · Peripheral Blood Stem Cell Transplantation (4) · Alemtuzumab (3) · Bone Marrow Transplantation (3) · Cord Blood Stem Cell Transplantation (3) · Cyclophosphamide (3) · Methotrexate (3) · Cyclosporine (2) · Melphalan (2) · Methylprednisolone (2) · Mycophenolic Acid (2) · Radiotherapy (2) · Stem Cell Transplantation (2)
Masonic Cancer Center, University of Minnesota (3) · St. Jude Children's Research Hospital (3) · Emory University (2) · UCSF Benioff Children's Hospital Oakland (2) · University of California, San Francisco (2) · Children's Hospital Los Angeles (1) · Diane George (1) · Fred Hutchinson Cancer Center (1) · New York Medical College (1) · Novartis (1) · Paul Szabolcs (1) · Roswell Park Cancer Institute (1) · Stanford University (1) · Talaris Therapeutics Inc. (1)
United States (19) · Germany (2) · United Kingdom (2) · Australia (1)
Elliott Vichinsky, MD (2) · Morton J. Cowan, MD (2) · Angela Smith, MD (1) · Diane George, MD (1) · George Chen (1) · John B Porter, MD (1) · John E. Wagner, MD (1) · John Horan, MD (1) · John Porter, MD (1) · John T Horan, MD (1) · Kim E. Nichols, MD (1) · Kimberly Kasow, DO (1) · Lauri Burroughs, MD (1) · Marcin Wlodarski, MD, PhD (1) · Margaret MacMillan, MD (1)
PMID 24370862, PMID 25459642, PMID 26095669, PMID 27537786
R01DK057778 (2), U10HL069294 (1)