Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or 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.

NCT Number	NCT05477563
Lead Sponsor	Vertex Pharmaceuticals Incorporated
Conditions	Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Enrollment	26 participants
Start Date	2022-08-02
Primary Completion	2027-06-09 (estimated)
Study Completion	2027-06-09 (estimated)
Updated on ClinicalTrials.gov	2026-03-23