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

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).

Timeline

Start

2022-05-02

Primary completion

2027-06-06

Completion

2027-06-06

Publications

• Frangoul H, de la Fuente J, Chopra Y, Meisel R, Amrolia PJ, Algeri M, Sharma A, Cappellini MD, Corbacioglu S, Kattamis A, Lobitz S, de Montalembert M, Rondelli D, Sheth S, Steinberg MH, Walters MC, Boerner K, Liu T, Zairis S, Hobbs W, Grupp SA, Locatelli F; CLIMB THAL-141 and CLIMB SCD-151 Study Groups. Exa-cel in Children with Transfusion-Dependent beta-Thalassemia or Sickle Cell Disease. N Engl J Med. 2026 Jun 11. doi: 10.1056/NEJMoa2603387. Online ahead of print.

Drugs

Indications