6 completed, 6 terminated, 3 recruiting, 1 unknown. 10 Phase 2, 6 Phase 1, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Terminated 5, Completed 3, Recruiting 1, Unknown 1 |
| Phase 1 | 6 | Terminated 4, Completed 2 |
| Not applicable | 2 | Completed 1, Terminated 1 |
| Phase 3 | 1 | Completed 1 |
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 |
|---|---|---|---|---|---|---|
| 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 | |
| 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 | |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia leucine - Diamond Blackfan Anemia, Blackfan Diamond Syndrome | Phase 1 / Phase 2 | Completed | Northwell Health | 55 | 2013-06-01 | 2022-12-02 |
| Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders Haploidentical Bone Marrow/Kidney - Chronic Kidney Disease, Acute Myeloid Leukemia (AML) | Not applicable | Completed | Massachusetts General Hospital | 10 | 2012-11-01 | 2026-03-06 |
| Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia Sotatercept, Sotatercept with prednisone boost - Diamond Blackfan Anemia | Phase 1 / Phase 2 | Terminated | Northwell Health | 19 | 2012-01-01 | 2023-03-17 |
| Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload Deferasirox - Thalassemia, Sickle Cell Disease | Phase 3 | Completed | Novartis Pharmaceuticals | 1683 | 2005-10-01 | 2011-06-07 |
| Cancer in Inherited Bone Marrow Failure Syndromes Diamond Blackfan Anemia, Dyskeratosis Congenita | Recruiting | National Cancer Institute (NCI) | 4000 | 2001-11-28 | 2026-10-29 | |
| Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia G-CSF, Leukapheresis - Diamond Blackfan Anemia | Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2001-02-01 | 2008-03-04 |
| A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure Daclizumab - Aplastic Anemia, Pure Red Cell Aplasia | Phase 2 | Terminated | National Heart, Lung, and Blood Institute (NHLBI) | 100 | 1999-11-01 | 2021-07-09 |
Anemia, Diamond-Blackfan (16) · Anemia, Sickle Cell (8) · Anemia, Aplastic (6) · Thalassemia (5) · Fanconi Anemia (4) · Lymphoma, Non-Hodgkin (4) · Red-Cell Aplasia, Pure (4) · Anemia (3) · Leukemia (3) · Leukemia, Myeloid, Acute (3) · Myelodysplastic Syndromes (3) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (3) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (3) · Shwachman-Diamond Syndrome (3) · Adrenoleukodystrophy (2)
Bone Marrow Diseases (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Hematologic Diseases (16) · Hemic and Lymphatic Diseases (16) · Congenital Bone Marrow Failure Syndromes (15) · Anemia, Hypoplastic, Congenital (14) · Bone Marrow Failure Disorders (14) · Anemia (13) · Red-Cell Aplasia, Pure (12) · Anemia, Aplastic (10) · Anemia, Hemolytic, Congenital (8) · Anemia, Hemolytic (7) · Metabolic Diseases (7) · Nutritional and Metabolic Diseases (7)
AP 1903 reagent (2) · Leucine (2) · ACE-011 (1) · Daclizumab (1) · Deferasirox (1) · Granulocyte Colony-Stimulating Factor (1) · Leukapheresis (1) · Prednisone (1) · Trifluoperazine (1)
Northwell Health (3) · Bellicum Pharmaceuticals (2) · National Heart, Lung, and Blood Institute (NHLBI) (2) · Adrianna Vlachos, MD (1) · Centre Hospitalier Universitaire de Liege (1) · Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache (1) · Masonic Cancer Center, University of Minnesota (1) · Massachusetts General Hospital (1) · Mitchell Cairo (1) · National Cancer Institute (NCI) (1) · Novartis Pharmaceuticals (1) · University of Florida (1)
United States (12) · Italy (3) · Belgium (2) · United Kingdom (2) · Canada (1) · Germany (1) · Greece (1) · Netherlands (1) · Russia (1) · Spain (1) · Taiwan (1) · Thailand (1) · Turkey (Türkiye) (1)
Adrianna Vlachos, MD (3) · Alexandra Satty, MD (1) · Ashish Gupta, MBBS, MPH (1) · Biljana Horn, MD (1) · Laurent Servais (1) · Lisa J McReynolds, M.D. (1) · Mitchell S Cairo (1) · Neal Young, MD (1) · Yi-Bin A Chen, M.D. (1)
PMID 25304614, PMID 25405500, PMID 25963299, PMID 28280134, PMID 31151984, PMID 31836590, PMID 32790018, PMID 33025707, PMID 33810313, PMID 34162668, PMID 34852175, PMID 35322185, PMID 35360980, PMID 37354000, PMID 39279436, PMID 40762130