NCT03745287

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 63 (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 subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Conditions

• Sickle Cell Disease
• Hematological Diseases
• Hemoglobinopathies

Interventions

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

Timeline

• Start date: 2018-11-27
• Primary completion: 2025-07-07
• Completion: 2025-07-07
• First posted: 2018-11-19
• Last updated: 2025-08-11

Locations

17 sites across 7 countries: United States, Belgium, Canada, France, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study