CTX001-111 - PH1/2 in BThalassemia
Research type
Research Study
Full title
A Phase 1/2 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion-Dependent β Thalassemia
IRAS ID
238068
Contact name
Jesus de la Fuente Pereda
Contact email
Sponsor organisation
CRISPR Therapeutics AG
Eudract number
2017-003351-38
Duration of Study in the UK
3 years, 9 months, 0 days
Research summary
This study is designed to evaluate the safety and efficacy of a new investigational therapy named CTX001 in adults up to 35 years old who have an inherited blood disorder called beta-thalassemia. Beta-thalassemia causes severe anaemia, and patients must regularly receive blood transfusions. CTX001 is made using the study participants’s own blood forming stem cells. The stem cells will be collected from the participant, and sent to a manufacturing facility where they will be gene-edited using CRISPR-Cas9 technology. These gene-edited stem cells will then be administered back to the participant as part of a stem cell transplant. The hope is that this investigational treatment will eliminate or reduce the need for frequent blood transfusions. Up to 45 patients will participate in this study around the world. Participants will be in the study for about 2.5 years.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/0700
Date of REC Opinion
2 Jul 2018
REC opinion
Further Information Favourable Opinion