clinical trial · NCT05951205
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
Vertex Pharmaceuticals Incorporated·phase3·withdrawn
Sickle Cell DiseaseExa-cel
brief 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).
started
Jul 31, 2027
primary completion
Dec 31, 2033
completion
Dec 31, 2033
last updated
Mar 25, 2026
official title
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol