Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype

Phase 3 Withdrawn Vertex Pharmaceuticals Incorporated

NaSingle-groupOpen-labelTreatment

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).

Timeline

Start: 2027-07-31
Primary completion: 2033-12-31
Completion: 2033-12-31

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	EXAGAMGLOGENE AUTOTEMCEL	CRISPR / gene editing	—	Intravenous

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype

Summary

Timeline

Drugs

Indications