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)
Phase of Trial: Phase I/II
Latest Information Update: 13 Jun 2019
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At a glance
- Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
- Indications Beta-thalassaemia; Haemoglobinopathies; Sickle cell anaemia
- Focus Adverse reactions; Pharmacodynamics; Proof of concept; Therapeutic Use
- Sponsors bluebird bio
- 10 Jun 2019 Status changed from active, no longer recruiting to completed.
- 03 Jun 2019 The European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO for patients 12 years and older with transfusion-dependent beta-thalassemia for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. This approval is supported by data from HGB-205 ,HGB-204, HGB-207, HGB-212 and LTF-303 studies.
- 16 May 2019 This trial has been completed in France.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History