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NCT02633943

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

Study 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.

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Interventions

Safety and efficacy assessmentsOTHER
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.

Study Locations

FacilityCityStateCountry
N/AOaklandCaliforniaUnited States
N/AChicagoIllinoisUnited States
N/ABethesdaMarylandUnited States
N/ANew YorkNew YorkUnited States
N/APhiladelphiaPennsylvaniaUnited States
N/ACharlestonSouth CarolinaUnited States
N/ASydneyAustralia
N/AMarseilleFrance
N/AParisFrance
N/AHanoverGermany
N/AHeidelbergGermany
N/AThessalonikiGreece
N/ARomeItaly
N/ABangkokThailand
N/ALondonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026