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

Last updated: June 27, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Not Recruiting

Phase

3

Condition

Red Blood Cell Disorders

Sickle Cell Disease

Treatment

Exa-cel

Clinical Study ID

NCT05951205
VX21-CTX001-171
2021-006375-41
2023-503247-34-00
  • Ages 12-35
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Participants with documented βS/βC (HbSC) genotype

  • Participants must be eligible for autologous stem cell transplant as perinvestigator's judgment

Exclusion

Key Exclusion Criteria:

  • A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor isavailable per investigator's judgement

  • Participants with prior hematopoietic stem cell transplant (HSCT)

  • Treatment with regular RBC transfusions that, in the opinion of the investigator,cannot be interrupted after engraftment.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 12
Treatment Group(s): 1
Primary Treatment: Exa-cel
Phase: 3
Study Start date:
July 31, 2027
Estimated Completion Date:
December 31, 2033