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

Study 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).

Conditions

Beta-Thalassemia Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

Interventions / Drugs

CTX001

View on ClinicalTrials.gov ↗

Sponsor

VRTX

Vertex Pharmaceuticals

Gene Therapy

C55/100

Study Details

Enrollment16 participants

Primary Completion—

Lead SponsorVertex Pharmaceuticals Incorporated

Data Retrieved2026-04-11 02:55:58.035274+00:00

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