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Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
Age
0 - 50 years
Sex
ALL
Healthy Volunteers
No
Oakland, California, United States
Chicago, Illinois, United States
Bethesda, Maryland, United States
New York, New York, United States
Philadelphia, Pennsylvania, United States
Charleston, South Carolina, United States
Sydney, Australia
Marseille, France
Paris, France
Hanover, Germany
Start Date
January 1, 2014
Primary Completion Date
November 1, 2035
Completion Date
November 1, 2035
Last Updated
April 9, 2025
66
ACTUAL participants
Safety and efficacy assessments
OTHER
Lead Sponsor
Genetix Biotherapeutics Inc.
NCT07055503
NCT06146478
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
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