LTF-303
Research type
Research Study
Full title
Longterm Follow-up of Subjects With Transfusion-Dependent β-Thalassemia Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
IRAS ID
275485
Contact name
BROOKE ORR
Contact email
Sponsor organisation
bluebird bio, Inc.
Eudract number
2013-002245-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
15 years, 0 months, 1 days
Research summary
This research study is evaluating the long-term effects of an investigational gene transfer approach to treat beta thalassemia major, also referred to as transfusion-dependent β–thalassemia, and severe sickle cell disease for patients who have been treated with the drug product in the parent studies.
The gene transfer the patients were exposed to in the parent study involved transferring a functioning copy of the beta globin gene into the patient´s blood stem cells. The modified blood stem cells were returned to the patient´s body, and it is hoped that they will continue to produce new cells that contain the functioning copy of the beta globin gene and ultimately correct or improve the anemia from transfusion-dependent β–thalassemia or the severe sickle cell disease.
The investigational gene transfer agent used in the parent studies is a lentiviral vector (a virus that has been changed in the laboratory so that it does not grow once it is in the patient's body). During this long term follow-up study subjects will be monitored regularly (every 6 months during the first 3 years then yearly during the following 10 years). The monitoring includes blood draws to assess efficacy and safety, MRI of the liver and hear and quality of life questionnaires.REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/0192
Date of REC Opinion
27 Apr 2020
REC opinion
Further Information Favourable Opinion