NCT02151526

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Genetix Biotherapeutics Inc. · Industry
• Sex: All
• Age: 5 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).

Conditions

• Beta-Thalassemia Major
• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	LentiGlobin BB305 Drug Product	LentiGlobin BB305 Drug Product was administered by intravenous (IV) infusion.

Timeline

• Start date: 2013-06-07
• Primary completion: 2019-02-26
• Completion: 2019-02-26
• First posted: 2014-05-30
• Last updated: 2022-04-26
• Results posted: 2020-03-10

Locations

1 site across 1 country: France

Regulatory

• FDA-regulated drug study