9 active not recruiting. 9 Phase 1, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 9 | Active not recruiting 9 |
| Phase 2 | 7 | Active not recruiting 7 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Haploidentical PBMC Transplant for Severe Congenital Anemias PBSC Transplant, Alemtuzumab, Sirolimus - Sickle Cell Anemia | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 23 | 2009-12-14 | 2026-04-23 |
| 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 |
beta-Thalassemia (7) · Anemia, Sickle Cell (5) · Hemoglobinopathies (2) · Anemia, Hemolytic, Congenital (1) · Graft vs Host Disease (1)
Anemia (9) · Anemia, Hemolytic (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Hematologic Diseases (9) · Hemic and Lymphatic Diseases (9) · Anemia, Hemolytic, Congenital (8) · Hemoglobinopathies (7) · Immune System Diseases (1)
Alemtuzumab (3) · Sirolimus (3) · Cyclophosphamide (2) · Antilymphocyte Serum (1) · Filgrastim (1) · Granulocyte Colony-Stimulating Factor (1) · Hydroxyurea (1) · Mycophenolic Acid (1) · Pentostatin (1) · Pharmaceutical Preparations (1) · Radiation (1) · Tacrolimus (1) · Transplantation (1) · plerixafor (1) · thymoglobulin (1)
National Heart, Lung, and Blood Institute (NHLBI) (3) · Bioray Laboratories (1) · Children's Hospital of Philadelphia (1) · City of Hope Medical Center (1) · David Williams (1) · Editas Medicine, Inc. (1) · Lantu Biopharma (1)
United States (7) · China (2) · Canada (1)
John F Tisdale, M.D. (2) · Courtney D Fitzhugh, M.D. (1) · Erica Esrick, MD (1) · Janet Kwiatkowski, MD (1) · Joseph Rosenthal, MD (1) · Rongmei Liang, PhD (1)
PMID 36240296, PMID 42133908, PMID 20007560, PMID 25058217, PMID 33283990, PMID 34917079, PMID 40010689, PMID 41871072, PMID 41931048, PMID 42233421