NCT02633943

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 66 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: All
• Age: 0 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
OTHER	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.

Timeline

• Start date: 2014-01-01
• Primary completion: 2035-11-01
• Completion: 2035-11-01
• First posted: 2015-12-17
• Last updated: 2025-04-09

Locations

15 sites across 8 countries: United States, Australia, France, Germany, Greece, Italy, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study