Clinical trial
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
NCT05329649 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 Severe Sickle Cell Disease
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Phase
- Phase 3
- Overall status
- Active, not recruiting
- Start date
- May 2, 2022
- Completion date
- June 6, 2027
- Enrollment
- 13 (actual)
- Sites
- 7
Conditions
As the registry lists them; a condition with its own page is linked.
hematological diseases, hemoglobinopathies, hydroxyurea failure, hydroxyurea intolerance, sickle cell disease