Tracking gene therapy kids for 15 years: does the fix last?
NCT ID NCT04049084
Summary
This study is checking on children who were previously treated with an experimental gene therapy for a rare, severe immune system disorder called ADA-SCID. The goal is to see how safe and effective the treatment remains over 15 years by tracking their health, survival, and whether they need any additional treatments. No new therapy is given in this study; it only observes and collects data from about 70 participants during their regular doctor visits.
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Contacts and locations
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Locations
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Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095, United States
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UCL Great Ormond Street Institute of Child Health
London, WC1N 1EH, United Kingdom
Conditions
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