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
View at source ↗Clinical trial
Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.
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).
Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.
Trial details
- status
- WITHDRAWN
- phase
- PHASE3
- study type
- INTERVENTIONAL
- enrollment
- 0
- sponsor
- Vertex Pharmaceuticals Incorporated
- conditions
- Sickle Cell Disease
- interventions
- BIOLOGICAL: Exa-cel
- start date
- 2027-07-31
- start date type
- ESTIMATED
- primary completion date
- 2033-12-31
- has results
- false
- data source
- ClinicalTrials.gov
- data processed
- 2026-10-29T09:00:05
- modifications
- selected fields only; whitespace normalized; brief summary truncated to 2,000 characters
Record
- Archive ID
- nct:NCT05951205
- Published
- Jul 18, 2023
- Updated
- Mar 25, 2026
- Venue
- ClinicalTrials.gov
- ClinicalTrials.gov
- NCT05951205
- Categories
- Sickle Cell Disease
- License
- ClinicalTrials.gov terms (attribution, processing date, modifications noted)
- Provenance
- clinicaltrials