One-Shot gene therapy trial aims to fix rare hormone disorder
NCT ID NCT04783181
Summary
This is the first-ever study in people to test a gene therapy for congenital adrenal hyperplasia (CAH), a genetic disorder where the body can't make enough cortisol. Eight adults with classic CAH will receive a single intravenous dose of the experimental therapy, called BBP-631. The main goals are to see if the treatment is safe and if it can help the body produce its own cortisol, potentially reducing the need for lifelong steroid medication.
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Contacts and locations
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Locations
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Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Lucas Research, Inc.
Morehead City, North Carolina, 28557, United States
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892-1932, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
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