Clinical Trial: Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Observational




Official Title: Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Brief Summary: This is a multi-center, long-term safety and efficacy follow-up study for subjects with hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (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 the study.