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

Brief summary

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.

Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.

Trial details

status

RECRUITING

phase

PHASE3

study type

INTERVENTIONAL

enrollment

26

sponsor

Vertex Pharmaceuticals Incorporated

conditions

Beta-Thalassemia; Thalassemia; Hematologic Diseases; Genetic Diseases, Inborn; Hemoglobinopathies; Sickle Cell Disease; Sickle Cell Anemia

interventions

BIOLOGICAL: CTX001

start date

2022-08-02

start date type

ACTUAL

primary completion date

2027-06-09

has results

false

data source

ClinicalTrials.gov

data processed

2026-10-29T09:00:05

modifications

selected fields only; whitespace normalized; brief summary truncated to 2,000 characters