15 active not recruiting. 15 Phase 1, 10 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 15 | Active not recruiting 15 |
| Phase 2 | 10 | Active not recruiting 10 |
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 |
|---|---|---|---|---|---|---|
| Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) OTQ923 - Sickle Cell Disease | Phase 1 | Active not recruiting | Novartis Pharmaceuticals | 4 | 2024-04-16 | 2026-07-28 |
| 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 |
| 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 |
| TMLI and Alemtuzumab for Treatment of Sickle Cell Disease Alemtuzumab, Hematopoietic Cell Transplantation, Intensity-Modulated Radiation Therapy - Sickle Cell Disease | Phase 1 | Active not recruiting | City of Hope Medical Center | 20 | 2022-07-13 | 2026-07-30 |
| 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 |
| A Blood Stem Cell Transplant for Sickle Cell Disease Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin - Sickle Cell Disease, Sickle Cell Disorder | Phase 1 | Active not recruiting | City of Hope Medical Center | 3 | 2019-01-04 | 2026-03-05 |
| Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease Plerixafor - Sickle Cell Disease | Phase 1 | Active not recruiting | City of Hope Medical Center | 5 | 2018-09-19 | 2026-09-02 |
| Gene Transfer for Sickle Cell Disease single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a - Sickle Cell Disease | Phase 1 | Active not recruiting | David Williams | 10 | 2018-02-13 | 2026-01-09 |
| 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 |
| 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 |
| 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 (15) · Hemoglobinopathies (3) · Anemia, Hemolytic (2) · Anemia, Hemolytic, Congenital (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) · Graft vs Host Disease (1) · Hematologic Diseases (1) · Histiocytosis (1) · Histiocytosis, Langerhans-Cell (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Hemic and Lymphatic Diseases (15) · Anemia (14) · Genetic Diseases, Inborn (14) · Hematologic Diseases (14) · Anemia, Hemolytic (13) · Anemia, Hemolytic, Congenital (13) · Hemoglobinopathies (12) · Immune System Diseases (2) · Thalassemia (2) · Urogenital Diseases (2) · Anemia, Hypoplastic, Congenital (1) · Blood-Borne Infections (1) · Bone Marrow Diseases (1) · Communicable Diseases (1)
Sirolimus (5) · Alemtuzumab (4) · Cyclophosphamide (3) · Filgrastim (2) · Granulocyte Colony-Stimulating Factor (2) · Hydroxyurea (2) · Pentostatin (2) · Abatacept (1) · Antilymphocyte Serum (1) · Aspirin (1) · Hematopoietic Stem Cell Transplantation (1) · Mycophenolic Acid (1) · Pharmaceutical Preparations (1) · Radiotherapy, Intensity-Modulated (1) · Stem Cell Transplantation (1)
National Heart, Lung, and Blood Institute (NHLBI) (4) · City of Hope Medical Center (3) · Beam Therapeutics Inc. (1) · Children's Hospital Medical Center, Cincinnati (1) · David Williams (1) · Diane George (1) · Editas Medicine, Inc. (1) · Instituto Nacional de Investigacao em Saude, Angola (1) · Novartis Pharmaceuticals (1) · University of Illinois at Chicago (1)
United States (13) · Angola (2) · Canada (1) · Democratic Republic of the Congo (1) · Kenya (1) · Uganda (1)
Courtney F Joseph, M.D. (2) · Anna Pawlowska (1) · Damiano Rondelli, MD (1) · Diane George, MD (1) · Erica Esrick, MD (1) · John F Tisdale, M.D. (1) · Joseph Rosenthal, MD (1) · Leo Wang, MD (1) · Russell Ware, MD, PhD (1) · Swee Lay Thein, M.D. (1)
PMID 36240296, PMID 42133908, PMID 20007560, PMID 25058217, PMID 26275071, PMID 29318647, PMID 30501550, PMID 33283990, PMID 36860093, PMID 36975201, PMID 38450513, PMID 38701812, PMID 38866240, PMID 40010689, PMID 41026975, PMID 41809202, PMID 41871072, PMID 41931046, PMID 41931047, PMID 41954645, PMID 42220645, PMID 42233421
P30CA033572 (1)