67 completed. 67 Phase 1, 21 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 67 | Completed 67 |
| Phase 2 | 21 | Completed 21 |
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 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 |
| 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 |
| Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD) Motixafortide, Natalizumab, Leukapheresis - Sickle Cell Disease | Phase 1 | Completed | Washington University School of Medicine | 10 | 2023-07-07 | 2025-08-11 |
| Hydroxyurea and EPO in Sickle Cell Disease Hydroxyurea, Epoetin Alfa-BioSimilar - Anemia, Sickle Cell, Sickle Cell Disease | Phase 1 / Phase 2 | Completed | Julia Xu | 17 | 2023-05-25 | 2026-01-07 |
| Voxelotor CYP and Transporter Cocktail Interaction Study Voxelotor, Bupropion, Repaglinide - Sickle Cell Disease | Phase 1 | Completed | Pfizer | 44 | 2023-04-17 | 2024-11-22 |
| A GBT021601 ADME Microtracer Study in Healthy Volunteers GBT021601 - Sickle Cell Disease | Phase 1 | Completed | Pfizer | 9 | 2022-12-22 | 2025-01-08 |
| PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD Positron Emission Tomography, Cu-64]-LLP2A - Sickle Cell Disease | Phase 1 | Completed | Enrico Novelli | 15 | 2022-11-11 | 2026-08-18 |
| A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD). Crovalimab, Placebo - Sickle Cell Disease | Phase 1 | Completed | Hoffmann-La Roche | 30 | 2022-03-26 | 2025-08-26 |
| Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir Pociredir oral capsule(s) - Sickle Cell Disease, Sickle Cell Anemia | Phase 1 | Completed | Fulcrum Therapeutics | 45 | 2021-12-13 | 2026-02-12 |
| Biotin-RBC Transfusion in SCD Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells - Sickle Cell Disease | Phase 1 | Completed | Marianne Yee | 22 | 2021-10-29 | 2026-05-22 |
| A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD) GBT021601 - Sickle Cell Disease | Phase 1 | Completed | Pfizer | 6 | 2021-05-21 | 2024-06-10 |
| Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease CSL889 - Sickle Cell Disease | Phase 1 | Completed | CSL Behring | 28 | 2021-05-20 | 2023-09-06 |
| A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants GBT021601 - Sickle Cell Disease | Phase 1 | Completed | Pfizer | 129 | 2020-12-09 | 2023-07-25 |
| Safety, Tolerability and Pharmacokinetics of FTX-6058 FTX-6058/placebo oral capsule(s), FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup - Healthy Adult Subjects, Sickle Cell Disease | Phase 1 | Completed | Fulcrum Therapeutics | 109 | 2020-10-26 | 2022-12-12 |
| Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) DREPAGLOBE drug product - Sickle Cell Disease | Phase 1 / Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 6 | 2019-11-12 | 2026-03-05 |
| A Study of SHP655 (rADAMTS13) in Sickle Cell Disease TAK-755, Placebo - Sickle Cell Disease | Phase 1 | Completed | Shire | 19 | 2019-10-21 | 2024-04-23 |
| Sickle Cell Pain: Intervention With Capsaicin Exposure Eight Percent Topical Capsaicin - Neuropathic Pain, Sickle Cell Disease | Phase 1 | Completed | Children's Hospital of Michigan | 10 | 2019-07-03 | 2020-07-29 |
| A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease Plerixafor, Busulfan, BIVV003 - Sickle Cell Disease | Phase 1 / Phase 2 | Completed | Sangamo Therapeutics | 7 | 2019-03-06 | 2025-09-12 |
| Pharmacokinetics of Oral Hydroxyurea Solution Oral Hydroxyurea (100 mg/mL) Solution - Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Nova Laboratories Limited | 33 | 2019-01-03 | 2024-10-28 |
| A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients FT-4202/Placebo - Healthy Volunteers, Sickle Cell Disease | Phase 1 | Completed | Forma Therapeutics, Inc. | 130 | 2018-12-11 | 2024-04-10 |
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Anemia, Sickle Cell (67) · Thalassemia (6) · Anemia (3) · Granulomatous Disease, Chronic (3) · Hemoglobin SC Disease (3) · Mucopolysaccharidosis I (3) · Pain (3) · Wiskott-Aldrich Syndrome (3) · beta-Thalassemia (3) · Acute Chest Syndrome (2) · Anemia, Aplastic (2) · Anemia, Diamond-Blackfan (2) · Chediak-Higashi Syndrome (2) · Hemoglobinopathies (2) · Hypertension, Pulmonary (2)
Anemia, Hemolytic, Congenital (67) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (67) · Hematologic Diseases (67) · Hemic and Lymphatic Diseases (67) · Anemia, Hemolytic (66) · Genetic Diseases, Inborn (66) · Hemoglobinopathies (65) · Anemia (64) · Pathological Conditions, Signs and Symptoms (9) · Pathologic Processes (6) · Immune System Diseases (5) · Cardiovascular Diseases (4) · Chronic Disease (4) · Disease Attributes (4) · Metabolic Diseases (4)
Hydroxyurea (5) · fludarabine (4) · voxelotor (4) · Hematopoietic Stem Cell Transplantation (3) · plerixafor (3) · Abatacept (2) · Alemtuzumab (2) · Cyclosporine (2) · Erythropoietin (2) · Excipients (2) · Melphalan (2) · Mycophenolic Acid (2) · PEGylated carboxyhemoglobin bovine (2) · Saline Solution (2) · Simvastatin (2)
National Heart, Lung, and Blood Institute (NHLBI) (4) · Pfizer (4) · Global Blood Therapeutics (3) · AstraZeneca (2) · Emory University (2) · Fulcrum Therapeutics (2) · Genetix Biotherapeutics Inc. (2) · Novo Nordisk A/S (2) · UCSF Benioff Children's Hospital Oakland (2) · Albert Einstein College of Medicine (1) · ApoPharma (1) · Assistance Publique - Hôpitaux de Paris (1) · Baxalta now part of Shire (1) · Bloodworks (1) · CSL Behring (1)
United States (55) · United Kingdom (6) · France (4) · Jamaica (3) · Lebanon (3) · Netherlands (3) · Italy (2) · Kenya (2) · Nigeria (2) · South Africa (2) · Spain (2) · Australia (1) · Belgium (1) · Brazil (1) · Canada (1)
John F Tisdale, M.D. (3) · Carla Washington, PhD (2) · Adeyemi Adenola, MD (1) · Ahmar U Zaidi, MD (1) · Alexander K Glaros, MD (1) · Angel Hernandez, MD (1) · Angela E Rankine- Mullings, MD (1) · Anjulika Chawla, MD, FAAP (1) · Barbara A Konkle, M.D. (1) · Biljana Horn, MD (1) · Callaghan Michael, MD (1)
PMID 40891510, PMID 21477202, PMID 22887019, PMID 23377438, PMID 23416852, PMID 24988449, PMID 25938131, PMID 26348869, PMID 28152086, PMID 28249145, PMID 28880867, PMID 29419425, PMID 29669226, PMID 30655275, PMID 31140114, PMID 32678452, PMID 32977351, PMID 33629819, PMID 33811823, PMID 33956057, PMID 34115136, PMID 34160085, PMID 34166998, PMID 34175041, PMID 34898139, PMID 34922648, PMID 34981144, PMID 35019238, PMID 35075288, PMID 36161320, PMID 38640200, PMID 40169559, PMID 40569673, PMID 42008008, PMID 42018645, PMID 42133908, PMID 42183775
1RC2HL101367-01 (1), 1ZIAHL006149-01 (1), 2014 (1), 2017 (1), 2019 (1), K23HL146904 (1), R01HL154629 (1), R01NS045948 (1)