Gene therapy could ease daily steroid dependence for rare hormone disorder
NCT ID NCT04783181
First seen Jan 06, 2026 · Last updated May 23, 2026 · Updated 15 times
Summary
This study tests a new gene therapy for adults with classic congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production and requires lifelong steroid medication. The treatment uses a harmless virus to deliver a working copy of the faulty gene, aiming to reduce abnormal hormone levels and potentially lower the need for daily steroids. Eight adults are enrolled in this early-phase trial to check safety and find the best dose.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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
Explore the condition pages connected to this study.