Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
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
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
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
- ACTIVE_NOT_RECRUITING
- phase
- PHASE3
- study type
- INTERVENTIONAL
- enrollment
- 13
- sponsor
- Vertex Pharmaceuticals Incorporated
- conditions
- Sickle Cell Disease; Hydroxyurea Failure; Hydroxyurea Intolerance; Hemoglobinopathies; Hematological Diseases
- interventions
- BIOLOGICAL: CTX001
- start date
- 2022-05-02
- start date type
- ACTUAL
- primary completion date
- 2027-06-06
- 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:NCT05329649
- Published
- Apr 15, 2022
- Updated
- Jul 1, 2026
- Venue
- ClinicalTrials.gov
- ClinicalTrials.gov
- NCT05329649
- Categories
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
- License
- ClinicalTrials.gov terms (attribution, processing date, modifications noted)
- Provenance
- clinicaltrials