31 completed, 11 terminated, 10 recruiting, 2 unknown. 32 Phase 2, 17 Phase 1, 7 Not applicable, 4 Phase 3, 1 Phase 4.
Follow Anemia, Diamond-Blackfan: new trials by email Literature Review of Anemia, Diamond-Blackfan trials Research Report on Anemia, Diamond-Blackfan
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 |
|---|---|---|---|---|---|---|
| Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) Mobilization Regimen - Diamond Blackfan Anemia | Recruiting | Northwell Health | 10 | 2026-06-01 | 2026-04-27 | |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia APR-2020 - RPS19 Deficient Diamond-Blackfan Anemia | Phase 1 | Recruiting | Apriligen, Inc. | 4 | 2026-04-16 | 2026-07-21 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| 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 |
| AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion alpha beta depletion - Acute Leukemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Mitchell Cairo | 20 | 2021-04-01 | 2026-10-23 |
| Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag Eltrombopag - Anemia, Diamond-Blackfan | Phase 1 / Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2020-12-01 | 2025-04-27 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| The Use of Trifluoperazine in Transfusion Dependent DBA Trifluoperazine - Diamond Blackfan Anemia, Pure Red Cell Aplasia | Phase 1 / Phase 2 | Terminated | Adrianna Vlachos, MD | 2 | 2019-09-13 | 2022-12-14 |
| 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 |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis - Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission | Phase 2 | Terminated | Roswell Park Cancer Institute | 31 | 2017-12-07 | 2025-07-04 |
| Familial Investigations of Childhood Cancer Predisposition Acute Leukemia, Adenomatous Polyposis | Recruiting | St. Jude Children's Research Hospital | 1500 | 2017-04-06 | 2026-06-17 | |
| French National Registry of Bone Marrow Failures Bone Marrow Failure - Bone Marrow Failure Syndrome | Recruiting | Assistance Publique - Hôpitaux de Paris | 5000 | 2017-02-06 | 2021-03-04 | |
| Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study Rimiducid, rivogenlecleucel - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2017-01-01 | 2023-09-26 |
| 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 | |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen - Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia | Not applicable | Terminated | Masonic Cancer Center, University of Minnesota | 38 | 2014-09-02 | 2025-03-03 |
| L-leucine in Diamond Blackfan Anemia Patients L-leucine - Diamond Blackfan Anemia | Phase 2 | Unknown | Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache | 30 | 2014-09-01 | 2015-03-11 |
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2014-04-01 | 2023-09-29 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
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Anemia, Sickle Cell (26) · Thalassemia (20) · Anemia, Aplastic (19) · Fanconi Anemia (14) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (14) · Shwachman-Diamond Syndrome (14) · Myelodysplastic Syndromes (13) · Bone Marrow Failure Disorders (11) · Wiskott-Aldrich Syndrome (10) · beta-Thalassemia (10) · Granulomatous Disease, Chronic (9) · Lymphohistiocytosis, Hemophagocytic (9) · Dyskeratosis Congenita (8) · Hemoglobinopathies (8)
Bone Marrow Diseases (56) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (56) · Hemic and Lymphatic Diseases (56) · Anemia, Hypoplastic, Congenital (54) · Hematologic Diseases (53) · Congenital Bone Marrow Failure Syndromes (52) · Genetic Diseases, Inborn (52) · Anemia (51) · Red-Cell Aplasia, Pure (48) · Bone Marrow Failure Disorders (45) · Anemia, Aplastic (37) · Anemia, Hemolytic (34) · Anemia, Hemolytic, Congenital (33) · Nutritional and Metabolic Diseases (33) · Metabolic Diseases (30)
fludarabine phosphate (11) · Antilymphocyte Serum (8) · Busulfan (8) · Cyclophosphamide (7) · Alemtuzumab (6) · Deferasirox (6) · Peripheral Blood Stem Cell Transplantation (6) · Melphalan (5) · fludarabine (5) · Bone Marrow Transplantation (4) · Cord Blood Stem Cell Transplantation (4) · Cyclosporine (4) · Methotrexate (4) · Tacrolimus (4) · Thiotepa (4)
National Heart, Lung, and Blood Institute (NHLBI) (5) · Novartis Pharmaceuticals (5) · Masonic Cancer Center, University of Minnesota (4) · Bellicum Pharmaceuticals (3) · Northwell Health (3) · Paul Szabolcs (3) · St. Jude Children's Research Hospital (3) · Diane George (2) · Emory University (2) · Fred Hutchinson Cancer Center (2) · Roswell Park Cancer Institute (2) · UCSF Benioff Children's Hospital Oakland (2) · University of California, San Francisco (2) · Adrianna Vlachos, MD (1) · Apriligen, Inc. (1)
United States (47) · Italy (6) · United Kingdom (6) · Germany (4) · Belgium (3) · Greece (3) · Taiwan (3) · Thailand (3) · Turkey (Türkiye) (3) · Australia (2) · Canada (2) · China (2) · Egypt (2) · France (2) · Lebanon (2)
Adrianna Vlachos, MD (3) · Paul Szabolcs, MD (3) · Diane George, MD (2) · Elliott Vichinsky, MD (2) · Morton J. Cowan, MD (2) · Alexandra Satty, MD (1) · Angela Smith, MD (1) · Ashish Gupta, MBBS, MPH (1) · Biljana Horn, MD (1) · David J Young, M.D. (1) · Farid Boulad, MD (1) · George Chen (1)
PMID 19951979, PMID 19996412, PMID 20566896, PMID 21071497, PMID 23585526, PMID 24296492, PMID 24370862, PMID 24385534, PMID 25304614, PMID 25345697, PMID 25405500, PMID 25459642, PMID 25691036, PMID 25963299, PMID 26095669, PMID 27537786, PMID 28280134, PMID 31151984, PMID 31836590, PMID 32634238, PMID 32750127, PMID 32790018, PMID 33025707, PMID 33810313, PMID 34162668, PMID 34852175, PMID 35322185, PMID 35360980, PMID 37354000, PMID 39279436, PMID 40762130
2004 (2), R01DK057778 (2), P30CA015704 (1), U10HL069294 (1)