5 recruiting, 3 active not recruiting, 2 enrolling by invitation, 1 completed. 3 Phase 3, 2 Not applicable, 2 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Active not recruiting 1, Completed 1, Recruiting 1 |
| Not applicable | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Recruiting 2 |
| Phase 1 | 1 | Recruiting 1 |
Follow Transfusion-dependent Beta-Thalassemia: new trials by email Literature Review of Transfusion-dependent Beta-Thalassemia trials Research Report on Transfusion-dependent Beta-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 |
|---|---|---|---|---|---|---|
| A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) Mitapivat Matched Placebo, Mitapivat - Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | Phase 3 | Recruiting | Agios Pharmaceuticals, Inc. | 54 | 2026-09-01 | 2026-09-28 |
| Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia FT007 - Transfusion-dependent Beta-Thalassemia | Phase 2 | Recruiting | Fondazione Telethon | 9 | 2026-07-06 | 2026-07-27 |
| Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients Transfusion-dependent Beta-Thalassemia, Blood Transfusion | Enrolling by invitation | University Research Institute for the Study of Genetic & Malignant Disorders in Childhood | 30 | 2025-11-20 | 2025-12-17 | |
| Plasticizer Exposure and Its Consequences on Health Beta-thalassemia, Glioma | Not yet recruiting | Medical University of Vienna | 120 | 2024-10-01 | 2024-07-24 | |
| A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 Safety and efficacy assessments - Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia | Enrolling by invitation | Editas Medicine, Inc. | 54 | 2024-06-17 | 2025-04-02 | |
| Β-Thalassemia Treatment with KL003 Cell Injection KL003 Cell Injection Drug Product - Transfusion-dependent Beta-Thalassemia | Phase 1 / Phase 2 | Recruiting | Kanglin Biotechnology (Hangzhou) Co., Ltd. | 41 | 2024-04-05 | 2025-03-10 |
| Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients KL003 cell injection Drug Product - Transfusion-dependent Beta-Thalassemia | Not applicable | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 3 | 2024-01-04 | 2025-08-05 |
| Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy Gene therapy - Transfusion-dependent Beta-Thalassemia, Gene Therapy | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 100 | 2023-06-05 | 2023-08-14 | |
| Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. KL003 cell injection Drug Product - Transfusion-dependent Beta-Thalassemia | Not applicable | Active not recruiting | Institute of Hematology & Blood Diseases Hospital, China | 3 | 2023-05-23 | 2025-08-05 |
| Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients Thalidomide - Transfusion-dependent Beta-Thalassemia | Phase 3 | Completed | Blood Care Clinic | 200 | 2022-01-25 | 2023-11-24 |
| A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) Placebo Matching Mitapivat, Mitapivat - Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 258 | 2021-11-30 | 2026-09-28 |
| Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Safety and efficacy assessments - Transfusion-dependent Beta-Thalassemia | Active not recruiting | Genetix Biotherapeutics Inc. | 66 | 2014-01-01 | 2025-04-09 |
beta-Thalassemia (4) · Anemia (2) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1) · Anemia, Sickle Cell (1) · Genetic Diseases, Inborn (1) · Glioma (1) · Hematologic Diseases (1) · Hemoglobinopathies (1) · Intracranial Hemorrhages (1)
Hemic and Lymphatic Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Hematologic Diseases (4) · Thalassemia (4) · Anemia (3) · Anemia, Hemolytic (3) · Anemia, Hemolytic, Congenital (3) · Genetic Diseases, Inborn (3) · Hemoglobinopathies (3) · Brain Diseases (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1) · Cerebrovascular Disorders (1) · Hemorrhage (1) · Neoplasms (1)
Safety (2) · mitapivat (2) · Genetic Therapy (1) · Thalidomide (1)
Institute of Hematology & Blood Diseases Hospital, China (3) · Agios Pharmaceuticals, Inc. (2) · Blood Care Clinic (1) · Editas Medicine, Inc. (1) · Fondazione Telethon (1) · Genetix Biotherapeutics Inc. (1) · Kanglin Biotechnology (Hangzhou) Co., Ltd. (1) · Medical University of Vienna (1) · University Research Institute for the Study of Genetic & Malignant Disorders in Childhood (1)
China (4) · United States (4) · Greece (3) · Italy (3) · Canada (2) · France (2) · Germany (2) · Thailand (2) · United Kingdom (2) · Australia (1) · Austria (1) · Brazil (1) · Bulgaria (1) · Denmark (1) · Lebanon (1)
Antonis Kattamis, Professor (1) · Haoquan Wu, PhD (1) · Himal L Thakar, MD (1) · Sami Siraj, PhD (1)
PMID 35075288, PMID 39527960, PMID 40446669, PMID 41525466, PMID 42721980
2025 (2)