NCT05951205View on ClinicalTrials.gov

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

PHASE3Not yet recruitingINTERVENTIONAL

Enrollment

12

Participants

Timeline

Start Date

July 31, 2027

Primary Completion Date

December 31, 2033

Study Completion Date

December 31, 2033

Conditions

Sickle Cell Disease

Interventions

BIOLOGICAL

Exa-cel

Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.

All Listed Sponsors

lead

Vertex Pharmaceuticals Incorporated

INDUSTRY