Last updated on February 2019

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent -Thalassemia


Brief description of study

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12 subjects 18 to 35 years of age with transfusion-dependent -thalassemia (TDT), non-0/0. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Detailed Study Description

The study may be expanded to include up to 45 subjects.

Clinical Study Identifier: NCT03655678

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CRISPR Therapeutics Clinical Operations

Research site
Regensburg, Germany
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CRISPR Therapeutics Clinical Operations

Research Site
London, United Kingdom
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Recruitment Status: Open


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