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

Clinical trial · Vertex Pharmaceuticals Incorporated

- Archive id: nct:NCT05951205
- Published: 2023-07-18
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT05951205
- Source URL: https://clinicaltrials.gov/study/NCT05951205
- Provenance: clinicaltrials

## Abstract

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).

_Abstract reproduced under 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._

## 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- status: WITHDRAWN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

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

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