Gene therapy aims to free patients from daily steroids for rare adrenal disorder
NCT ID NCT04783181
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a gene therapy called BBP-631 for classic congenital adrenal hyperplasia (CAH), a genetic condition that forces patients to take daily steroids. The therapy uses a harmless virus to deliver a working copy of the faulty gene. Only 8 adults are enrolled to check safety and whether it can lower stress hormones and raise natural cortisol levels.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- BBP-631 (a gene therapy using a harmless virus to deliver a working copy of the CYP21A2 gene)
- What this could lead to
- If successful, this could reduce or eliminate the need for daily steroid medication in people with classic congenital adrenal hyperplasia.
- What could go wrong
- This is a very early, small trial (8 people) testing safety first. Gene therapy is complex and may not work as hoped, and there are risks like immune reactions or liver issues.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New program aims to smooth healthcare transition for teens with rare hormone disorder
- New hope for CAH: drug may slash steroid use
- New hope for toddlers with rare hormone disorder?
- Italian study checks how well newborn screening detects rare hormone disorder
- New hormone treatment shows promise for rare adrenal disorder