NCT03655678

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Conditions

• Beta-Thalassemia
• Thalassemia
• Genetic Diseases, Inborn
• Hematologic Diseases
• Hemoglobinopathies

Interventions

Type	Name	Description
BIOLOGICAL	CTX001	Administered by IV infusion following myeloablative conditioning with busulfan

Timeline

• Start date: 2018-09-14
• Primary completion: 2025-11-13
• Completion: 2025-11-13
• First posted: 2018-08-31
• Last updated: 2025-12-17

Locations

14 sites across 5 countries: United States, Canada, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study