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Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
Sponsor: Genetix Biotherapeutics Inc.
Summary
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.
Official title: 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
Key Details
Gender
All
Age Range
2 Years - 53 Years
Study Type
OBSERVATIONAL
Enrollment
85
Start Date
2020-10-21
Completion Date
2038-01
Last Updated
2025-03-20
Healthy Volunteers
No
Conditions
Interventions
Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
Locations (16)
University of Alabama
Birmingham, Alabama, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Cohen Children's Medical Center
New Hyde Park, New York, United States
Columbia University Medical Center
New York, New York, United States
The University of North Carolina
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Baylor College of Medicine
Houston, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Hospital Necker
Paris, France