Clinical trial
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
NCT05356195 is registered in ClinicalTrials.gov with Vertex Pharmaceuticals Incorporated as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Phase
- Phase 3
- Overall status
- Active, not recruiting
- Start date
- May 3, 2022
- Completion date
- November 14, 2027
- Enrollment
- 16 (actual)
- Sites
- 6
Conditions
As the registry lists them; a condition with its own page is linked.