Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

Enrolling by invitation

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT04628585

Run by

Genetix Biotherapeutics Inc.

People needed

85

Starts

2020-10-21

Expected to finish

2038-01-01

Last updated by the study team

2025-03-20

Who can join

Age: 2 and older, up to 53. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• There are no exclusion criteria for this study

Where it is running

• 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.