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A Study Evaluating the Safety and Efficacy of bb1111 in Severe Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease

Clinical Trial Summary

This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

Clinical Trial Description

Subject participation for this study will be 2 years post-transplant. 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 an additional 13 years for a total of 15 years post-drug product infusion. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02140554
Study type Interventional
Source bluebird bio
Contact
Status Completed
Phase Phase 1/Phase 2
Start date February 2, 2015
Completion date January 30, 2024
View Details
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