A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Beta-Hemoglobinopathies (Sickle Cell Disease and Beta-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral Beta-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)
Active, no longer recruiting
Phase of Trial: Phase I/II
Latest Information Update: 01 Nov 2017
At a glance
- Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
- Indications Beta-thalassaemia; Sickle cell anaemia
- Focus Adverse reactions; Proof of concept; Therapeutic Use
- Sponsors bluebird bio
- 01 Nov 2017 According to a bluebird bio media release, data from the study will be presented at the American Society of Hematology (ASH) Annual Meeting 2017.
- 02 Aug 2017 According to a bluebird bio media release, the company presented new data from this study at European Hematology Association (EHA) Annual Meeting.
- 23 Jun 2017 According to a bluebird bio media release, as of June 2, 2017 four patients with Transfusion-Dependent Beta-Thalassemia and three patients with severe Severe Sickle Cell Disease undergone infusion in this trial.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History