Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector


6 United States sites

2 Germany sites

1 Australia site

1 France site

1 Italy site

1 United Kingdom site

1 Thailand site


< 50 Years

Study type








About the study

This is a multi-center, long-term safety and efficacy follow-up study for subjects with
hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with
ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After
completing the parent clinical study (approximately 2 years), eligible subjects will be
followed for an additional 13 years for a total of 15 years post-drug product infusion. No
investigational drug product will be administered in the study.

participation requirements

– Provision of written informed consent for this study by subjects, or as applicable,
subject’s parent(s)/legal guardian(s)

– Treated with drug product for therapy of a hemoglobinopathy in a bluebird
bio-sponsored clinical study

– Able to comply with study requirements

participation restrictions

– There are no exclusion criteria for this study


  • Oakland, California, United States
  • Chicago, Illinois, United States
  • Bethesda, Maryland, United States
  • New York, New York, United States
  • Philadelphia, Pennsylvania, United States
  • Charleston, South Carolina, United States
  • Sydney, Australia
  • Paris, France
  • Hannover, Germany
  • Heidelberg, Germany
  • Rome, Italy
  • Bangkok, Thailand
  • London, United Kingdom