Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Locations

2 United States sites

1 France site

Age

2 to 53 Years

Study type

Observational

Gender

All

Interventions

Other

Compensation

Unknown

About the study

This is a multi-center, long-term safety and efficacy follow-up study for subjects with
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 subject, or as applicable,
subject’s parent(s)/legal guardian(s)

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

– Able to comply with study requirements

participation restrictions

– There are no exclusion criteria for this study

Locations

  • New York, New York, United States, Columbia University Medical Center, 10032
  • Charleston, South Carolina, United States, Medical University of South Carolina, 29425
  • Paris, France, Hospital Necker, 75015