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

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

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.

Eligibility

Primary outcome measure(s)

• 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

Trial sites (15)

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