19 active not recruiting. 19 Phase 2, 10 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 19 | Active not recruiting 19 |
| Phase 1 | 10 | Active not recruiting 10 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow Sickle Cell Disease: new trials by email Literature Review of Sickle Cell Disease trials Research Report on Sickle Cell Disease
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 |
|---|---|---|---|---|---|---|
| A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD) Tebapivat, Tebapivat Matched Placebo - Sickle Cell Disease | Phase 2 | Active not recruiting | Agios Pharmaceuticals, Inc. | 59 | 2025-05-01 | 2026-10-28 |
| LEARNER- Low dosE AspiRiN prEterm tRial (Angola) Aspirin 100mg - Sickle Cell Disease, Pregnancy Related | Phase 1 / Phase 2 | Active not recruiting | Instituto Nacional de Investigacao em Saude, Angola | 450 | 2024-03-16 | 2026-07-06 |
| Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function Briquilimab, Filgrastim, Abatacept - Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 6 | 2023-12-28 | 2026-04-29 |
| Realizing Effectiveness Across Continents With Hydroxyurea Hydroxyurea - Sickle Cell Disease, Children | Phase 2 | Active not recruiting | Children's Hospital Medical Center, Cincinnati | 811 | 2023-10-27 | 2026-04-21 |
| BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease BEAM-101 - Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | Beam Therapeutics Inc. | 15 | 2022-08-30 | 2025-12-16 |
| A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a - Sickle Cell Disease | Phase 2 | Active not recruiting | David Williams | 25 | 2022-07-12 | 2026-08-25 |
| A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Mitapivat, Mitapivat-matching placebo - Sickle Cell Disease | Phase 2 / Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 286 | 2022-02-11 | 2026-10-26 |
| A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) EDIT-301 - Sickle Cell Disease, Hemoglobinopathies | Phase 1 / Phase 2 | Active not recruiting | Editas Medicine, Inc. | 45 | 2021-05-04 | 2025-01-31 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat Mitapivat - Sickle Cell Disease, Hemolytic Anemia | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2020-12-09 | 2026-11-06 |
| Reduced Intensity Transplantation for Severe Sickle Cell Disease hydroxyurea, azathioprine, alemtuzumab, thioptepa, low dose total body irradiation and sirolimus, hydroxyurea, azathioprine, alemtuzumab, thiotepa, plerixafor, low dose total body irradiation, cyclophosphamide and sirolimus - Sickle Cell Disease | Phase 2 | Active not recruiting | St. Jude Children's Research Hospital | 46 | 2020-04-30 | 2026-09-01 |
| An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia IMR-687 - Sickle Cell Disease | Phase 2 | Active not recruiting | Imara, Inc. | 30 | 2019-05-22 | 2022-03-04 |
| Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation Alemtuzumab, Fludarabine, Melphalan - Sickle Cell Disease | Phase 2 | Active not recruiting | Emory University | 43 | 2019-03-22 | 2026-09-24 |
| Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease haploidentical stem cell transplant, Sirolimus, Alemtuzumab - Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 57 | 2017-04-06 | 2026-04-23 |
| Realizing Effectiveness Across Continents With Hydroxyurea (REACH) Hydroxyurea - Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | Children's Hospital Medical Center, Cincinnati | 635 | 2014-06-01 | 2026-06-30 |
| Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure Alemtuzumab, Sirolimus, Cyclophosphamide - Sickle Cell Disease, Thalassemia | Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 56 | 2014-04-21 | 2026-08-18 |
| 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 |
| Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease CD34 selected T-cell depleted allogeneic SCT - Sickle Cell Disease | Phase 2 | Active not recruiting | New York Medical College | 21 | 2012-01-01 | 2025-08-08 |
| Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD) Allogeneic Non-Myeloablative Stem Cell Transplantation, Alemtuzumab, Sirolimus - Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | University of Illinois at Chicago | 45 | 2011-11-11 | 2026-07-08 |
| Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis - Congenital Hemolytic Anemia, Sickle Cell Disease | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 130 | 2004-07-16 | 2026-09-09 |
Anemia, Sickle Cell (19) · Anemia, Hemolytic (2) · Anemia, Hemolytic, Congenital (2) · Graft vs Host Disease (2) · Hemoglobinopathies (2) · beta-Thalassemia (2) · Acquired Immunodeficiency Syndrome (1) · Anemia (1) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Bone Marrow Failure Disorders (1) · Genetic Diseases, Inborn (1) · Hematologic Diseases (1) · Histiocytosis (1) · Histiocytosis, Langerhans-Cell (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19) · Hemic and Lymphatic Diseases (19) · Anemia (18) · Genetic Diseases, Inborn (18) · Hematologic Diseases (18) · Anemia, Hemolytic (17) · Anemia, Hemolytic, Congenital (17) · Hemoglobinopathies (17) · Immune System Diseases (3) · Thalassemia (2) · Urogenital Diseases (2) · Anemia, Hypoplastic, Congenital (1) · Blood-Borne Infections (1) · Bone Marrow Diseases (1) · Communicable Diseases (1)
Alemtuzumab (6) · Sirolimus (6) · Cyclophosphamide (4) · Hydroxyurea (4) · Filgrastim (3) · Granulocyte Colony-Stimulating Factor (2) · Pentostatin (2) · mitapivat (2) · Abatacept (1) · Aspirin (1) · Azathioprine (1) · Cyclosporine (1) · Cyclosporins (1) · Melphalan (1) · Methotrexate (1)
National Heart, Lung, and Blood Institute (NHLBI) (5) · Agios Pharmaceuticals, Inc. (2) · Children's Hospital Medical Center, Cincinnati (2) · Beam Therapeutics Inc. (1) · David Williams (1) · Diane George (1) · Editas Medicine, Inc. (1) · Emory University (1) · Imara, Inc. (1) · Instituto Nacional de Investigacao em Saude, Angola (1) · New York Medical College (1) · St. Jude Children's Research Hospital (1) · University of Illinois at Chicago (1)
United States (16) · Canada (4) · Angola (3) · Kenya (3) · United Kingdom (3) · Belgium (2) · Democratic Republic of the Congo (2) · France (2) · Netherlands (2) · Uganda (2) · Brazil (1) · Germany (1) · Ireland (1) · Israel (1) · Italy (1)
Courtney F Joseph, M.D. (2) · Russell Ware, MD, PhD (2) · Akshay Sharma, MBBS (1) · Ann Haight, MD (1) · Damiano Rondelli, MD (1) · David Williams (1) · Diane George, MD (1) · John F Tisdale, M.D. (1) · Karen Tang, MD (1) · Matthew M Hsieh, M.D. (1) · Mitchell S Cairo, MD (1) · Swee Lay Thein, M.D. (1)
PMID 36240296, PMID 42133908, PMID 40010689, PMID 41809202, PMID 41954645, PMID 20007560, PMID 25058217, PMID 26275071, PMID 29318647, PMID 30501550, PMID 35110690, PMID 36860093, PMID 36975201, PMID 37254256, PMID 37934880, PMID 38450513, PMID 38691679, PMID 38701812, PMID 38841694, PMID 38866240, PMID 39644907, PMID 41026975, PMID 41871072, PMID 41931046, PMID 41931047, PMID 42220645, PMID 42233421, PMID 42913682
1OT2HL154815 (1), 2018 (1), U01HL133883 (1)