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

Launched by BLUEBIRD BIO · Dec 15, 2015

Keywords

ClinConnect Summary

This clinical trial is focused on following the long-term effects of a special treatment called ex vivo gene therapy for patients with transfusion-dependent beta-thalassemia (TDT), a condition that requires regular blood transfusions. The study is designed for individuals who have already participated in earlier studies sponsored by bluebird bio, and it aims to learn more about the safety and effectiveness of the gene therapy over a total of 15 years after receiving the treatment. It is important to note that no new treatments will be given during this follow-up study.

To be eligible for this trial, participants must have received the gene therapy in a previous bluebird bio study and must provide written consent, either themselves or through a parent or legal guardian if they are minors. There are no specific exclusion criteria, meaning anyone who meets the inclusion criteria can participate. During the study, participants can expect regular check-ins to monitor their health and outcomes related to the gene therapy they received. This long-term follow-up will help researchers understand how well the treatment works over time and its overall impact on patients' lives.

Gender

ALL

Eligibility criteria

• 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