Clinical Trial

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
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).
Protocol Amendment History 2 changes
notable Primary completion pushed: 2026-05-31 -> 2027-06-06 2026-07-02
minor Completion pushed: 2026-05-31 -> 2027-06-06 2026-07-02
Trial Details
NCT Number NCT05329649
Lead Sponsor Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics
Conditions Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
Enrollment 13 participants
Start Date 2022-05-02
Primary Completion 2027-06-06 (estimated)
Study Completion 2027-06-06 (estimated)
Updated on ClinicalTrials.gov 2026-07-01