Clinical Research Directory
Browse clinical research sites, groups, and studies.
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
Sponsor: Genetix Biotherapeutics Inc.
Summary
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.
Official title: 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
Key Details
Gender
All
Age Range
0 Years - 50 Years
Study Type
OBSERVATIONAL
Enrollment
66
Start Date
2014-01
Completion Date
2035-11
Last Updated
2025-04-09
Healthy Volunteers
No
Conditions
Interventions
Safety and efficacy assessments
Genetic: No interventional drug product utilized in this follow-up study Participants received a single IV infusion of LentiGlobin BB305 Drug Product in the parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term effects of autologous transplant are conducted in this study.
Locations (15)
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
Heidelberg, Germany
Thessaloniki, Greece
Rome, Italy
Bangkok, Thailand
London, United Kingdom