NCT02906202

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: All
• Age: 0 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	LentiGlobin BB305 Drug Product	LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.

Timeline

• Start date: 2016-08-08
• Primary completion: 2022-03-31
• Completion: 2022-03-31
• First posted: 2016-09-20
• Last updated: 2023-06-18
• Results posted: 2023-06-18

Locations

8 sites across 6 countries: United States, France, Germany, Italy, Thailand, United Kingdom