2 completed. 2 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
| Phase 3 | 2 | Completed 2 |
Follow Exa-cel: new trials by email Literature Review of Exa-cel trials Research Report on Exa-cel
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 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 (2) · Hemoglobinopathies (2) · Anemia, Sickle Cell (1) · Genetic Diseases, Inborn (1) · Thalassemia (1) · beta-Thalassemia (1)
Vertex Pharmaceuticals Incorporated (2)
Canada (2) · Germany (2) · Italy (2) · United Kingdom (2) · United States (2) · Belgium (1) · France (1)
PMID 33283989, PMID 34175041, PMID 32873924, PMID 36123234, PMID 36860093, PMID 36975201, PMID 38657265, PMID 38661449, PMID 38866240, PMID 40857358, PMID 40862696, PMID 42252696