67 completed, 19 recruiting, 16 terminated, 15 active not recruiting. 136 Phase 1, 59 Phase 2.
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 |
|---|---|---|---|---|---|---|
| Minocycline In Neurocognitive Outcomes - Sickle Cell Disease Minocycline, Placebo - Sickle Cell Disease, Cognitive Impairment | Phase 1 | Not yet recruiting | University of Cincinnati | 30 | 2026-12-01 | 2026-11-03 |
| Feasibility and Acceptability of Amani Amani - Sickle Cell Disease | Phase 1 / Phase 2 | Not yet recruiting | Massachusetts General Hospital | 40 | 2026-11-01 | 2026-08-07 |
| Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452 - Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | Phase 1 | Recruiting | Daniel Bauer | 5 | 2026-08-01 | 2026-10-21 |
| Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors Total Body Irradiation, Alemtuzumab, Abatacept - Sickle Cell Disease, Beta-thalassemia | Phase 1 / Phase 2 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 90 | 2026-07-08 | 2026-10-13 |
| Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease Ruxolitinib - Sickle Cell Disease, Hematopoetic Stem Cell Transplant | Phase 1 / Phase 2 | Recruiting | Arkansas Children's Hospital Research Institute | 24 | 2026-06-08 | 2026-06-04 |
| STEM CELL MOBILIZATION WITH HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE Plerixafor Dose Escalation - Sickle Cell Disease | Phase 1 / Phase 2 | Not yet recruiting | Hospital Israelita Albert Einstein | 12 | 2026-06-01 | 2026-01-14 |
| Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study hydroxycarbamide - Sickle Cell Disease, Sickle Cell Anemia in Children | Phase 1 / Phase 2 | Recruiting | Children's Hospital Medical Center, Cincinnati | 250 | 2026-05-13 | 2026-07-16 |
| Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA. DREAM01 drug product, anti-inflammatory therapy - Sickle Cell Disease | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 15 | 2026-02-25 | 2026-03-04 |
| A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) DISC-3405 - Sickle Cell Disease | Phase 1 | Recruiting | Disc Medicine, Inc | 24 | 2026-01-06 | 2026-10-05 |
| Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease Pociredir - Sickle Cell Disease | Phase 1 | Terminated | Fulcrum Therapeutics | 13 | 2025-12-13 | 2026-06-18 |
| Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452 - Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | Phase 1 | Recruiting | Daniel Bauer | 10 | 2025-12-01 | 2026-02-03 |
| Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease Extended-Release Methylphenidate - Sickle Cell Disease, Executive Dysfunction | Phase 1 | Recruiting | St. Jude Children's Research Hospital | 72 | 2025-11-25 | 2026-09-09 |
| Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease CRISPR_SCD001 - Sickle Cell Disease | Phase 1 / Phase 2 | Recruiting | Mark Walters, MD | 9 | 2025-08-22 | 2026-10-20 |
| A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants Etavopivat, Moxifloxacin, Placebo - Healthy Volunteers, Sickle Cell Disease | Phase 1 | Completed | Novo Nordisk A/S | 33 | 2025-06-09 | 2026-01-02 |
| Cannabidiol in Sickle Cell Disease Cannabidiol, Placebo - Sickle Cell Disease | Phase 1 / Phase 2 | Recruiting | Icahn School of Medicine at Mount Sinai | 52 | 2025-04-03 | 2025-06-25 |
| Gene Editing For Sickle Cell Disease Plerixafor, Busulfan, Gene-modified CD34+ cells - Sickle Cell Disease | Phase 1 | Recruiting | St. Jude Children's Research Hospital | 25 | 2025-03-21 | 2026-10-15 |
| Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease Fostamatinib - Sickle Cell Disorders, Sickle Cell Disease | Phase 1 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 35 | 2024-12-18 | 2026-11-02 |
| SCD Stem Cell Mobilization and Apheresis Using Motixafortide Motixafortide - Sickle Cell Disease | Phase 1 | Recruiting | St. Jude Children's Research Hospital | 20 | 2024-11-14 | 2026-09-08 |
| A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants Etavopivat - Healthy Volunteers, Sickle Cell Disease | Phase 1 | Completed | Novo Nordisk A/S | 24 | 2024-08-26 | 2025-12-29 |
| A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease ITU512, Placebo - Sickle Cell Disease | Phase 1 / Phase 2 | Recruiting | Novartis Pharmaceuticals | 161 | 2024-08-15 | 2026-10-27 |
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Anemia, Sickle Cell (136) · beta-Thalassemia (10) · Thalassemia (8) · Hemoglobinopathies (5) · Anemia (4) · Pain (4) · Vaso-Occlusive Crises (4) · Acute Chest Syndrome (3) · Anemia, Aplastic (3) · Anemia, Diamond-Blackfan (3) · Anemia, Hemolytic (3) · Granulomatous Disease, Chronic (3) · Hemoglobin SC Disease (3) · Hypertension, Pulmonary (3) · Lymphohistiocytosis, Hemophagocytic (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (136) · Hemic and Lymphatic Diseases (136) · Anemia, Hemolytic, Congenital (134) · Genetic Diseases, Inborn (134) · Hematologic Diseases (134) · Anemia, Hemolytic (133) · Anemia (132) · Hemoglobinopathies (131) · Pathological Conditions, Signs and Symptoms (13) · Thalassemia (10) · Pathologic Processes (8) · Immune System Diseases (7) · Cardiovascular Diseases (6) · Lung Diseases (6) · Neurologic Manifestations (6)
Hydroxyurea (9) · Alemtuzumab (8) · Sirolimus (7) · Hematopoietic Stem Cell Transplantation (5) · fludarabine (5) · plerixafor (5) · voxelotor (5) · Abatacept (4) · Cyclophosphamide (4) · Mycophenolic Acid (4) · Stem Cell Transplantation (4) · Busulfan (3) · Citrulline (3) · Simvastatin (3) · Tacrolimus (3)
National Heart, Lung, and Blood Institute (NHLBI) (10) · City of Hope Medical Center (4) · Emory University (4) · Pfizer (4) · St. Jude Children's Research Hospital (4) · Children's Hospital Los Angeles (3) · Children's Hospital Medical Center, Cincinnati (3) · Fulcrum Therapeutics (3) · Global Blood Therapeutics (3) · Novartis Pharmaceuticals (3) · University of Mississippi Medical Center (3) · Assistance Publique - Hôpitaux de Paris (2) · AstraZeneca (2) · Daniel Bauer (2) · Federal University of Bahia (2)
United States (110) · France (6) · United Kingdom (6) · Brazil (3) · Canada (3) · Jamaica (3) · Kenya (3) · Lebanon (3) · Netherlands (3) · Angola (2) · China (2) · Italy (2) · Nigeria (2) · South Africa (2) · Spain (2)
John F Tisdale, M.D. (5) · Carla Washington, PhD (2) · Courtney F Joseph, M.D. (2) · Swee Lay Thein, M.D. (2) · Abdullah Kutlar, MD (1) · Adeyemi Adenola, MD (1) · Ahmar U Zaidi, MD (1) · Akshay Sharma, MBBS, MSc (1) · Alexander K Glaros, MD (1) · Alexis Leonard, MD (1) · Allistair Abraham, MD (1)
PMID 42133908, PMID 36240296, PMID 36860093, PMID 36975201, PMID 38866240, PMID 40891510, PMID 18550886, PMID 19840322, PMID 20007560, PMID 20117846, PMID 20644039, PMID 20732454, PMID 20797926, PMID 20936416, PMID 21477202, PMID 22887019, PMID 23377438, PMID 23416852, PMID 23973010, PMID 24988449, PMID 25058217, PMID 25938131, PMID 26021713, PMID 26275071, PMID 26348869, PMID 28096387, PMID 28152086, PMID 28249145, PMID 28880867, PMID 29318647, PMID 29419425, PMID 29669226, PMID 29788942, PMID 30501550, PMID 30655275, PMID 31140114, PMID 32384147, PMID 32678452, PMID 32977351, PMID 33283990, PMID 33629819, PMID 33811823, PMID 33956057, PMID 34115136, PMID 34160085, PMID 34166998, PMID 34175041, PMID 34898139, PMID 34922648, PMID 34981144
1RC2HL101367-01 (1), 1ZIAHL006149-01 (1), 2014 (1), 2017 (1), 2019 (1), 2025 (1), K23HL133446 (1), K23HL146904 (1), K24AT009893 (1), P20GM109021 (1), P30CA033572 (1), R01HL154629 (1), R01NS045948 (1), R33HL147845 (1), U01HL163983 (1)