7 completed, 5 terminated, 2 recruiting, 1 active not recruiting. 17 Phase 1, 12 Phase 2.
Follow Red-Cell Aplasia, Pure: new trials by email Literature Review of Red-Cell Aplasia, Pure trials Research Report on Red-Cell Aplasia, Pure
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 | |
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning - Non Malignant Disorders, Immunodeficiencies | Phase 1 | Completed | Duke University | 22 | 2008-10-01 | 2014-08-13 |
| 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 |
Anemia, Diamond-Blackfan (17) · Anemia, Sickle Cell (9) · Anemia, Aplastic (7) · Hemoglobinopathies (6) · Thalassemia (6) · Bone Marrow Failure Disorders (5) · Fanconi Anemia (5) · Lymphohistiocytosis, Hemophagocytic (5) · Primary Immunodeficiency Diseases (5) · Leukemia, Myeloid, Acute (4) · Mucopolysaccharidosis I (4) · Myelodysplastic Syndromes (4) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (4) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (4) · Shwachman-Diamond Syndrome (4)
Bone Marrow Diseases (17) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Genetic Diseases, Inborn (17) · Hematologic Diseases (17) · Hemic and Lymphatic Diseases (17) · Congenital Bone Marrow Failure Syndromes (16) · Anemia, Hypoplastic, Congenital (15) · Anemia (14) · Bone Marrow Failure Disorders (12) · Anemia, Aplastic (10) · Anemia, Hemolytic, Congenital (10) · Nutritional and Metabolic Diseases (10) · Anemia, Hemolytic (9) · Immune System Diseases (9)
Alemtuzumab (3) · Thiotepa (3) · fludarabine (3) · fludarabine phosphate (3) · AP 1903 reagent (2) · Busulfan (2) · Hydroxyurea (2) · Melphalan (2) · ACE-011 (1) · Abatacept (1) · Antilymphocyte Serum (1) · Cell- and Tissue-Based Therapy (1) · Cord Blood Stem Cell Transplantation (1) · Cyclophosphamide (1) · Leucine (1)
Bellicum Pharmaceuticals (3) · Diane George (2) · Northwell Health (2) · Adrianna Vlachos, MD (1) · Apriligen, Inc. (1) · Duke University (1) · Emory University (1) · National Heart, Lung, and Blood Institute (NHLBI) (1) · New York Medical College (1) · Paul Szabolcs (1) · Talaris Therapeutics Inc. (1) · University of California, San Francisco (1) · University of Florida (1)
United States (15) · Italy (2) · United Kingdom (1)
Adrianna Vlachos, MD (3) · Diane George, MD (2) · Biljana Horn, MD (1) · David J Young, M.D. (1) · John T Horan, MD (1) · Mitchell S Cairo, MD (1) · Morton J. Cowan, MD (1) · Paul Szabolcs, MD (1) · Suhag Parikh, MD (1) · Suzanne T Ildstad, MD (1)