69 completed, 18 terminated, 16 unknown, 15 active not recruiting. 138 Phase 2, 29 Phase 1, 20 Phase 3.
Follow Thalassemia: new trials by email Literature Review of Thalassemia trials Research Report on Thalassemia
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 |
|---|---|---|---|---|---|---|
| Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia HGI-001 Injection - Beta Thalassemia Transfusion Dependent | Phase 2 | Not yet recruiting | Prince of Wales Hospital, Shatin, Hong Kong | 6 | 2026-09-01 | 2026-07-23 |
| 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 |
| Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells - Leukemia, Lymphoma | Phase 2 | Not yet recruiting | Hematology department of the 920th hospital | 60 | 2026-07-03 | 2026-07-14 |
| A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT) SNH-119014 dose 1, SNH-119014 dose 2, Placebo - Thalassemia | Phase 2 | Not yet recruiting | ScinnoHub Pharmaceutical Co., Ltd. | 30 | 2026-06-18 | 2026-05-12 |
| Open-Label Extension Study to Pioneer Study 6058-SCD-101 Pociredir - Sickle Cell Disease | Phase 2 | Terminated | Fulcrum Therapeutics | 1 | 2026-03-30 | 2026-10-28 |
| Effect of Omega 3 Fatty Acids on Heart and Liver in Beta Thalassemia Patients Omega 3 Fish Oil supplements, Deferasirox - Thalassemia Majors (Beta-Thalassemia Major) | Phase 2 | Completed | Ain Shams University | 100 | 2026-01-01 | 2026-10-07 |
| ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia ALS20 - Beta-Thalassemia | Phase 1 / Phase 2 | Active not recruiting | Children's Hospital of Philadelphia | 12 | 2025-04-14 | 2026-10-29 |
| A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body REGN7999, Placebo - Non-Transfusion Dependent Beta-Thalassemia (NTDT) | Phase 2 | Active not recruiting | Regeneron Pharmaceuticals | 19 | 2024-09-30 | 2026-07-01 |
| An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia Vebeglogene autotemcel - Beta-Thalassemia | Phase 1 / Phase 2 | Active not recruiting | Lantu Biopharma | 6 | 2024-05-11 | 2026-06-15 |
| Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia Thalidomide - Fetal Hemoglobin, Thalassemia Major | Phase 2 | Recruiting | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | 54 | 2024-04-22 | 2025-05-30 |
| Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia L-glutamine - Pulmonary Artery Pressure, Thalassemia | Phase 2 / Phase 3 | Completed | Mazandaran University of Medical Sciences | 8 | 2023-08-15 | 2025-10-07 |
| MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies Fludarabine, Busulfan, Melphalan - Hematologic Malignancy, Acute Leukemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 70 | 2023-05-11 | 2026-04-06 |
| A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) Fludarabine, Cyclophosphamide, Tacrolimus - Sickle Cell Disease, Thalassemia, Beta | Phase 2 | Recruiting | Memorial Sloan Kettering Cancer Center | 24 | 2023-02-09 | 2026-06-03 |
| A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia Luspatercept, Placebo - Anemia | Phase 2 | Active not recruiting | Bristol-Myers Squibb | 189 | 2022-12-09 | 2026-09-10 |
| Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia BRL-101 - Beta-Thalassemia | Phase 1 / Phase 2 | Active not recruiting | Bioray Laboratories | 39 | 2022-11-02 | 2026-07-21 |
| A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. Luspatercept, Placebo - Beta-thalassemia | Phase 2 | Active not recruiting | Bristol-Myers Squibb | 94 | 2022-10-17 | 2025-08-12 |
| 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 |
| Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients CN128 Tablets - Thalassemia, Iron Overload | Phase 2 | Completed | Hangzhou Zede Pharma-Tech Co., Ltd. | 16 | 2022-06-13 | 2025-03-14 |
| Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia TBI, Hydroxyurea, briquilimab - Sickle Cell Anemia, Beta Thalassemia | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 40 | 2022-05-12 | 2026-03-12 |
| EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) EDIT-301 - Transfusion Dependent Beta Thalassemia, Hemoglobinopathies | Phase 1 / Phase 2 | Active not recruiting | Editas Medicine, Inc. | 9 | 2022-04-29 | 2025-04-02 |
Showing 20 of 138. Sign in to see all of them.
beta-Thalassemia (87) · Anemia, Sickle Cell (48) · Iron Overload (18) · Anemia, Diamond-Blackfan (16) · Hemoglobinopathies (15) · Anemia (10) · Genetic Diseases, Inborn (9) · Anemia, Aplastic (8) · Hematologic Diseases (8) · Myelodysplastic Syndromes (8) · Wiskott-Aldrich Syndrome (6) · Granulomatous Disease, Chronic (5) · Leukemia (5) · Lymphoma, Non-Hodgkin (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (138) · Hemic and Lymphatic Diseases (138) · Anemia, Hemolytic, Congenital (137) · Anemia, Hemolytic (136) · Hematologic Diseases (130) · Genetic Diseases, Inborn (129) · Anemia (128) · Hemoglobinopathies (123) · Nutritional and Metabolic Diseases (33) · Metabolic Diseases (30) · Bone Marrow Diseases (23) · Anemia, Hypoplastic, Congenital (17) · Immune System Diseases (17) · Iron Metabolism Disorders (17)
Deferasirox (17) · Hydroxyurea (14) · Alemtuzumab (13) · Cyclophosphamide (11) · fludarabine (11) · Busulfan (9) · Deferoxamine (9) · Thalidomide (8) · fludarabine phosphate (8) · Deferiprone (7) · Sirolimus (7) · Whole-Body Irradiation (6) · Melphalan (5) · Tacrolimus (5) · luspatercept (5)
Novartis Pharmaceuticals (10) · National Heart, Lung, and Blood Institute (NHLBI) (7) · Ain Shams University (5) · Carelon Research (4) · Boston University (3) · Celgene (3) · Masonic Cancer Center, University of Minnesota (3) · National Institute of Blood and Marrow Transplant (NIBMT), Pakistan (3) · Shire (3) · Baylor College of Medicine (2) · Bellicum Pharmaceuticals (2) · Bristol-Myers Squibb (2) · Children's Hospital Karachi (2) · Children's Hospital Los Angeles (2) · Children's Hospital of Philadelphia (2)
United States (71) · Italy (27) · Thailand (18) · Lebanon (16) · United Kingdom (15) · Greece (14) · Canada (12) · China (12) · Turkey (Türkiye) (12) · Egypt (10) · Pakistan (8) · France (4) · Iran (4) · Malaysia (4) · Saudi Arabia (4)
John F Tisdale, M.D. (3) · Catherine J. Wu, MD (2) · Claudia Morris, MD (2) · Janet Kwiatkowski, MD (2) · John Porter, MD (2) · Nancy Olivieri, MD (2) · Paul Szabolcs, MD (2) · Yin X Lin, director (2) · Yutaka Niihara, MD (2) · Adlette Inati, MD (1)
PMID 37975597, PMID 36240296, PMID 42133908, PMID 29931453, PMID 34175041, PMID 40010689, PMID 14550808, PMID 18550258, PMID 20007560, PMID 20421452, PMID 20955402, PMID 21173101, PMID 21393329, PMID 21700776, PMID 21742281, PMID 22251482, PMID 22571408, PMID 2265118, PMID 22875626, PMID 23151373, PMID 23553596, PMID 23585527, PMID 23927461, PMID 24370862, PMID 24385534, PMID 24452753, PMID 24797180, PMID 24976388, PMID 25058217, PMID 25212456, PMID 25345697, PMID 25459642, PMID 25492271, PMID 25600572, PMID 26348869, PMID 27326168, PMID 28249145, PMID 29119631, PMID 29669226, PMID 30381400, PMID 30453981, PMID 30617198, PMID 32395210, PMID 32634238, PMID 32750127, PMID 33283989, PMID 33560399, PMID 33570654, PMID 33931857, PMID 34917079
U01HL065238 (4), 2018 (3), 2014 (2), 2016 (2), 2019 (2), 1OT2HL154815 (1), 2004 (1), 2015 (1), 2017 (1), 2023 (1), P30CA015704 (1), R01HL042814 (1), R33HL147845 (1), U01HL061877 (1), U01HL065232 (1)