NCT03275051

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

A Long-term Safety and Efficacy follow-on Study in Participants With Transfusion Dependent Beta-thalassemia Who Have Previously Received OTL-300 (Formerly Know as GSK2696277)) and Completed the TIGET-BTHAL Study

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: IRCCS San Raffaele · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).

Conditions

• Beta Thalassaemia

Interventions

Type	Name	Description
OTHER	Safety and Efficacy assessments	Safety and efficacy assessment of OTL-300 in subjects with transfusion dependent beta-thalassemia will be performed.

Timeline

• Start date: 2017-10-04
• Primary completion: 2025-12-01
• Completion: 2026-06-01
• First posted: 2017-09-07
• Last updated: 2022-11-23

Locations

1 site across 1 country: Italy