18 completed, 7 terminated, 3 recruiting, 2 unknown. 32 Phase 2, 12 Phase 1, 2 Phase 3.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide - Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML) | Phase 1 / Phase 2 | Unknown | Diane George | 14 | 2013-06-03 | 2024-06-04 |
| 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 |
| 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 |
| 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 |
| 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 | |
| Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine - Non-Neoplastic Hematologic and Lymphocytic Disorder | Phase 2 | Completed | Fred Hutchinson Cancer Center | 98 | 2009-07-31 | 2021-08-13 |
| 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 |
| Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox - Transfusional Iron Overload, Transfusional Hemosiderosis | Phase 2 | Completed | Novartis Pharmaceuticals | 197 | 2007-11-01 | 2014-08-27 |
Showing 20 of 32. Sign in to see all of them.
Anemia, Sickle Cell (14) · Thalassemia (12) · Anemia, Aplastic (11) · Bone Marrow Failure Disorders (9) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (8) · Shwachman-Diamond Syndrome (8) · Myelodysplastic Syndromes (7) · Congenital amegakaryocytic thrombocytopenia (6) · Primary Immunodeficiency Diseases (6) · Red-Cell Aplasia, Pure (6) · Wiskott-Aldrich Syndrome (6) · Hemoglobinopathies (5) · Leukemia, Myeloid, Acute (5) · Lymphohistiocytosis, Hemophagocytic (5)
Bone Marrow Diseases (32) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (32) · Hemic and Lymphatic Diseases (32) · Congenital Bone Marrow Failure Syndromes (31) · Hematologic Diseases (31) · Anemia, Hypoplastic, Congenital (30) · Genetic Diseases, Inborn (30) · Anemia (29) · Red-Cell Aplasia, Pure (26) · Bone Marrow Failure Disorders (23) · Anemia, Aplastic (21) · Anemia, Hemolytic (19) · Anemia, Hemolytic, Congenital (18) · Nutritional and Metabolic Diseases (18) · Metabolic Diseases (16)
fludarabine phosphate (11) · Antilymphocyte Serum (6) · Busulfan (6) · Peripheral Blood Stem Cell Transplantation (6) · Alemtuzumab (5) · Cyclophosphamide (5) · Methotrexate (4) · Tacrolimus (4) · Whole-Body Irradiation (4) · fludarabine (4) · Bone Marrow Transplantation (3) · Cord Blood Stem Cell Transplantation (3) · Cyclosporine (3) · Deferasirox (3) · Melphalan (3)
National Heart, Lung, and Blood Institute (NHLBI) (5) · Novartis Pharmaceuticals (3) · Bellicum Pharmaceuticals (2) · Diane George (2) · Fred Hutchinson Cancer Center (2) · Masonic Cancer Center, University of Minnesota (2) · Northwell Health (2) · Paul Szabolcs (2) · Roswell Park Cancer Institute (2) · University of California, San Francisco (2) · Adrianna Vlachos, MD (1) · Children's Hospital Los Angeles (1) · Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache (1) · Memorial Sloan Kettering Cancer Center (1) · Mitchell Cairo (1)
United States (27) · Italy (4) · United Kingdom (2) · Canada (1) · China (1) · Cyprus (1) · Egypt (1) · Greece (1) · Lebanon (1) · Russia (1) · Taiwan (1) · Thailand (1) · Turkey (Türkiye) (1) · United Arab Emirates (1)
Adrianna Vlachos, MD (3) · Diane George, MD (2) · Morton J. Cowan, MD (2) · Paul Szabolcs, MD (2) · Angela Smith, MD (1) · Biljana Horn, MD (1) · David J Young, M.D. (1) · Farid Boulad, MD (1) · George Chen (1) · Lauri Burroughs, MD (1) · Lauri M. Burroughs (1) · Margaret MacMillan, MD (1) · Mitchell S Cairo (1)
PMID 24370862, PMID 24385534, PMID 25345697, PMID 25459642, PMID 32634238, PMID 32750127, PMID 33025707
2004 (1), P30CA015704 (1), U10HL069294 (1)