NCT03207009

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia 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: 19 (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 approximately 18 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. 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: 2017-06-08
• Primary completion: 2022-11-15
• Completion: 2022-11-15
• First posted: 2017-07-02
• Last updated: 2024-03-07
• Results posted: 2023-12-29

Locations

9 sites across 6 countries: United States, France, Germany, Greece, Italy, United Kingdom

Regulatory

• FDA-regulated drug study