A Phase 3 Single Arm 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 do not 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

  202539

• Contact name

  John Porter

• Contact email

  j.porter@ucl.ac.uk

• Sponsor organisation

  bluebird bio, Inc.

• Eudract number

  2015-004122-33

• 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). To treat the disease, participants regularly receive transfusions of red blood cells. This treatment is effective but has some limitations. The only option currently available to help treat transfusion-dependent â-thalassemia is a stem cell transplant in which a participant receives blood forming stem cells from a donor.
  Gene transfer attempts to add functioning genes to help people who have a gene that is not working properly. The gene transfer in this study involves transferring a corrected copy of the beta globin gene into the participants blood stem cells. 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 functioning 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.
  The purpose of this study is to evaluate if gene transfer is a safe and effective way to treat transfusion-dependent â-thalassemia. This research study is being conducted in the hope that the functioning beta globin gene will be able to correct the abnormalities causing the disease. It is hoped that by inserting a functioning beta globin gene into the participants stem cells, their body will start making some amount of haemoglobin and may reduce anaemia, which may lead to fewer transfusions or no need for transfusions.
  About 15 participants from around the world will be in this study. Some will be adults and at least 5 will be adolescents. Participants will be in the study for about 2 years and 4 months.

  Lay summary of study results:
  Not available. If you would like to find more details on this study, please visit:
  https://www.clinicaltrials.gov/
  and search for the study identifier NCT02906202
  or
  https://www.clinicaltrialsregister.eu/
  and search for the study identifier 2015-004122-33

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  16/LO/1066

• Date of REC Opinion

  5 Sep 2016

• REC opinion

  Further Information Favourable Opinion