A Phase 3 Gene Therapy Study

• Research type

  Research Study

• Full title

  A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who have a 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

• IRAS ID

  224195

• Contact name

  John Porter

• Contact email

  j.porter@ucl.ac.uk

• Sponsor organisation

  bluebird bio, Inc.

• Eudract number

  2016-003611-35

• Duration of Study in the UK

  3 years, 6 months, days

• Research summary

  Research Summary:
  This study is evaluating a treatment for transfusion-dependent â-thalassemia (an inherited blood disease that can cause severe anaemia). Anaemia is a condition where the blood does not have enough healthy red blood cells.

  To treat the disease, participants usually require life long transfusions of donated blood. This treatment is effective but has some limitations.

  The only option currently available to help treat transfusion-dependent β-thalassemia is a medical procedure called a hematopoietic stem cell transplant (HSCT) in which a participant receives blood forming stem cells from a donor.

  Gene transfer attempts to add working genes to help people who have a gene that is not working properly.
  The goal of gene transfer is to cure the disease by transferring a working copy of the gene into a patient’s cells to correct the genetic defect. This is called gene transfer or gene addition.

  In this study, the blood stem cells will be collected from the participants blood and sent to a laboratory. At the laboratory, a gene transfer agent will be used to transfer a working copy of the beta globin gene into the DNA of blood stem cells. These modified blood stem cells will then be returned to the participants body, where we hope that they grow and produce new cells that will contain the functioning copy of the beta globin gene. In theory, over time corrected stem cells will produce normal red blood cells to correct the anaemia.

  This research study is being conducted in the hope that the working beta globin gene will be able to correct the abnormalities causing the disease. It is hoped that by inserting a changed beta globin gene into the stem cells, the participants body will start making some amount of haemoglobin and may reduce their anaemia, which may lead to fewer transfusions or no need for transfusions. The goal of this investigational treatment is for your changed blood stem cells to become a permanent source of blood cells with a working beta globin gene.

  About 15 participants will be in this study. Some will be adults and at least 10 will be young children and adolescents.

  Lay Summary of Results:
  Link to Clinicaltrials.gov results https://www.clinicaltrials.gov/study/NCT03207009?term=NCT03207009&rank=1&tab=results

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  17/LO/0494

• Date of REC Opinion

  3 May 2017

• REC opinion

  Favourable Opinion