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

Active, not recruitingOBSERVATIONAL
Enrollment

66

Participants

Timeline

Start Date

January 31, 2014

Primary Completion Date

November 30, 2035

Study Completion Date

November 30, 2035

Conditions
Transfusion-dependent Beta-Thalassemia
Interventions
OTHER

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."

Trial Locations (15)

Unknown

Oakland

Chicago

Bethesda

New York

Philadelphia

Charleston

Sydney

Marseille

Paris

Hanover

Heidelberg

Thessaloniki

Rome

Bangkok

London

Sponsors
All Listed Sponsors
lead

Genetix Biotherapeutics Inc.

INDUSTRY

NCT02633943 - Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy | Biotech Hunter | Biotech Hunter