2 active not recruiting, 2 completed, 1 enrolling by invitation. 5 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Active not recruiting 2, Completed 2, Enrolling by invitation 1 |
| Phase 2 | 2 | Completed 2 |
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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 |
|---|---|---|---|---|---|---|
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 16 | 2022-05-03 | 2026-07-01 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) CTX001 - Sickle Cell Disease, Hydroxyurea Failure | Phase 3 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 13 | 2022-05-02 | 2026-07-01 |
| A Long-term Follow-up Study in Participants Who Received CTX001 CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 160 | 2021-01-20 | 2026-08-11 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease Exa-cel - Sickle Cell Disease, Hematological Diseases | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 63 | 2018-11-27 | 2026-09-16 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia CTX001 - Beta-Thalassemia, Thalassemia | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 59 | 2018-09-14 | 2025-12-17 |
Hematologic Diseases (5) · Hemoglobinopathies (5) · Anemia, Sickle Cell (3) · Genetic Diseases, Inborn (3) · Thalassemia (3) · beta-Thalassemia (3)
Germany (5) · Italy (5) · United Kingdom (5) · United States (5) · Canada (4) · Belgium (2) · France (1)
PMID 33283989, PMID 34175041, PMID 40857358, PMID 40862696, PMID 42252696, PMID 42274009, PMID 32873924, PMID 36123234, PMID 36860093, PMID 36975201, PMID 38657265, PMID 38661449, PMID 38866240