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Clinical Trial Summary

The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT05951205
Study type Interventional
Source Vertex Pharmaceuticals Incorporated
Contact Medical Information
Phone 617-341-6777
Email medicalinfo@vrtx.com
Status Not yet recruiting
Phase Phase 3
Start date April 2024
Completion date December 2029

See also
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