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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.
Details
| Lead sponsor | Genetix Biotherapeutics Inc. |
|---|---|
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 66 |
| Start date | 2014-01 |
| Completion | 2035-11 |
Conditions
- Transfusion-dependent Beta-Thalassemia
Interventions
- Safety and efficacy assessments
Primary outcomes
- The number of subjects with malignancies — Up to 15 years post-drug product infusion
- The number of subjects with immune-related AEs — Up to 15 years post-drug product infusion
- The number of subjects with new or worsening hematologic disorders — Up to 15 years post-drug product infusion
- The number of subjects with new or worsening neurologic disorders — Up to 15 years post-drug product infusion
Countries
United States, Australia, France, Germany, Greece, Italy, Thailand, United Kingdom