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CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
NCT05329649Phase 3Active, not recruiting
- Sponsor
- Vertex Pharmaceuticals
- Start
- May 2022
- Primary completion
- Jun 2027
- Countries
- 4
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoint: Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months at 24 months. Started May 2022; status: active, not recruiting.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)
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