Gene therapy could ease rare hormone disorder
NCT ID NCT04783181
First seen Jan 06, 2026 · Last updated May 13, 2026 · Updated 13 times
Summary
This study tests a new gene therapy for adults with classic congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The treatment delivers a working copy of the CYP21A2 gene using a harmless virus. Researchers aim to see if it is safe and can reduce abnormal hormone levels, potentially allowing patients to lower their daily steroid medications.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for CONGENITAL ADRENAL HYPERPLASIA are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Lucas Research, Inc.
Morehead City, North Carolina, 28557, United States
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892-1932, United States
-
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
Explore the condition pages connected to this study.