Clinical Trial

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This is a single-dose, open-label study in pediatric participants with TDT. 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 -> 2027-11-14 2026-07-02
minor Completion pushed: 2026-05-31 -> 2027-11-14 2026-07-02
Trial Details
NCT Number NCT05356195
Lead Sponsor Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics
Conditions Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Enrollment 16 participants
Start Date 2022-05-03
Primary Completion 2027-11-14 (estimated)
Study Completion 2027-11-14 (estimated)
Updated on ClinicalTrials.gov 2026-07-01