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

Official 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).

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE

Interventions

• BIOLOGICAL: Exa-cel — Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan.

Primary Outcomes

• Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 Months (From 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion)

Secondary Outcomes

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (From Signing of Informed Consent up to 24 Months After exa-cel Infusion)
• Proportion of Participants With Neutrophil Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days) (Within 42 Days After exa-cel Infusion)
• Time to Neutrophil Engraftment (Up to 24 months After exa-cel Infusion)
• Time to Platelet Engraftment (Up to 24 months After exa-cel Infusion)
• Incidence of Transplant-Related Mortality (TRM) (Up to 100 Days After exa-cel Infusion)

More Sickle Cell Disease Trials

View all Sickle Cell Disease clinical trials