Drug Pipeline

CTX001 Clinical Trials

Data updated daily from ClinicalTrials.gov — last refresh 2026-06-12
4
Active Studies
1
Recruiting
4
Phase 3
1
Top Sponsors
Active Studies by Phase
Phase 3
4
Active Trials
NCT IDTitleConditionStatus
Phase 3
NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With… Beta-Thalassemia Recruiting
NCT05356195 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With… Beta-Thalassemia Active, Not Recruiting
NCT05329649 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle… Sickle Cell Disease Active, Not Recruiting
NCT04208529 A Long-term Follow-up Study in Participants Who Received CTX001 Beta-Thalassemia Enrolling by Invitation
Sponsors Running CTX001 Trials
Vertex Pharmaceuticals Incorporated (4)
Disease Areas
Sickle Cell Disease (3) Thalassemia (3)
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