Trial Profile

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 08 Apr 2025

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At a glance

03 Apr 2025 Planned End Date changed from 1 Feb 2025 to 9 Jun 2027.
03 Apr 2025 Planned primary completion date changed from 1 Feb 2025 to 9 Jun 2027.
11 Feb 2025 According to Vertex Pharmaceuticals media release,the company expects to complete dosing of children 5 to 11 years of age group in 2025.

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