This multi-center study provides long-term safety and efficacy follow-up for subjects with transfusion-dependent β-thalassemia (TDT) who were treated with an ex vivo gene therapy drug product in bluebird bio-sponsored parent studies. After the roughly 2-year parent studies, eligible subjects are followed for an additional 13 years, for a total of 15 years post-infusion. No investigational drug product is administered in this study; participation requires prior treatment in a parent study and written informed consent.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s) * Treated with drug product for therapy of transfusion-dependent β-thalassemia in a bluebird bio-sponsored clinical study Exclusion Criteria: * There are no exclusion criteria for this study