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

Phase 3 Recruiting 26 enrolled Vertex Pharmaceuticals Incorporated

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2022-08-02
Primary completion: 2027-06-09
Completion: 2027-06-09

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	EXAGAMGLOGENE AUTOTEMCEL	CRISPR / gene editing	—	Intravenous

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

Summary

Timeline

Drugs

Indications