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

Bluebird Bio

Status: Active, not recruiting

Conditions: Transfusion-dependent Beta-Thalassemia

Treatments: Other: Safety and efficacy assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT02633943

LTF-303

Details and patient eligibility

About

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.

Enrollment: 66 patients

Sex: All

Ages: Under 50 years old

Volunteers: No Healthy Volunteers

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

Trial design

66 participants in 1 patient group

Subjects with Transfusion-Dependent β-Thalassemia

Description:

Long-term follow-up for participants treated with ex vivo gene therapy product in applicable bluebird bio-sponsored parent clinical trials and who agreed to participate in this study. Participants will be followed in this study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies)

Treatment:

Other: Safety and efficacy assessments