NCT01745120

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: All
• Age: 12 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product \[autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene\].

Detailed description

Subject participation for this study will be 2 years. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant.

Conditions

• β-thalassemia Major

Interventions

Type	Name	Description
GENETIC	LentiGlobin BB305 Drug Product	Transplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector.

Timeline

• Start date: 2013-08-01
• Primary completion: 2018-02-08
• Completion: 2018-02-21
• First posted: 2012-12-07
• Last updated: 2019-05-08
• Results posted: 2019-05-08

Locations

6 sites across 3 countries: United States, Australia, Thailand