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 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
Phase of Trial: Phase III
Latest Information Update: 17 Sep 2017
At a glance
- Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
- Indications Beta-thalassaemia
- Focus Registrational; Therapeutic Use
- Acronyms Northstar-2
- Sponsors bluebird bio
- 02 Aug 2017 According to a bluebird bio media release, recruitment completed for the adult and adolescent patient cohort in Jun 2017.
- 25 Jun 2017 Trial design and recent update (n=2) on this study, presented at the 22nd Congress of the European Haematology Association.
- 23 Jun 2017 According to a bluebird bio media release, Alexis Thompson is a primary investigator on the study. As of June 2, 2017, drug product had been manufactured for six patients.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History