Congenital adrenal hyperplasia
MONDO:0018479Congenital adrenal hyperplasia (CAH) is an inherited endocrine disorder caused by a steroidogenic enzyme deficiency that is characterized by adrenal insufficiency and variable degrees of hyper or hypo androgyny manifestations, depending of the type and the severity of the disease.
Also known as: adrenal hyperplasia, adrenogenital disorder, adrenogenital syndrome, CAH, adrenal hyperplasia, congenital, congenital adrenal gland hyperplasia, congenital lipoid adrenal hyperplasia, lipoid CAH
40 clinical trials for this condition and its sub-types, 36 tagged with Congenital adrenal hyperplasia itself.
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Sub-types of Congenital adrenal hyperplasia
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A pill that quiets overactive adrenal glands? first human test begins
Disease control Not yet recruitingResearchers are testing an experimental oral drug called OMS1620 in 84 healthy adults. The drug blocks the receptor that adrenocorticotrophic hormone (ACTH) uses to signal the adrenal glands. The trial measures whether single and repeated doses are safe, how the drug moves throug…
Phase 1 • Sponsor: OMass Therapeutics Australia Proprietary Ltd • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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Could a drug tame excess cortisol in a common hormone disorder?
Disease control Not yet recruitingThis study tests whether the drug metyrapone can safely lower cortisol levels in people with mild autonomous cortisol secretion (MACS), a condition where the adrenal glands produce too much cortisol. Participants will receive either metyrapone or a placebo for a short period, and…
Phase 2 • Sponsor: Mayo Clinic • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New cortisol timing therapy may improve life for adults with rare hormone disorder
Disease control Not yet recruitingThis study follows 100 adults with congenital adrenal hyperplasia (CAH) who switch from standard steroids to a timed-release hydrocortisone (Efmody). The goal is to see if better mimicking the body's natural cortisol rhythm improves hormone levels, metabolic health, and quality o…
Sponsor: IRCCS San Raffaele • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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New program aims to smooth healthcare transition for teens with rare hormone disorder
Knowledge-focused Not yet recruitingThis study tests a structured program to help 40 adolescents and young adults with congenital adrenal hyperplasia (CAH) move from pediatric to adult healthcare. Participants will learn about their condition, emergency preparedness, and self-management. The goal is to see if the p…
Sponsor: University of Alabama at Birmingham • Aim: Knowledge-focused
Last updated Aug 23, 2026 00:00 UTC
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Could a simple saliva test replace blood draws for hormone monitoring?
Knowledge-focused Not yet recruitingThis study explores whether at-home saliva samples can reliably measure key steroid hormones in people with congenital adrenal hyperplasia (CAH), a condition requiring lifelong hormone therapy. Currently, monitoring involves morning blood tests that miss daily hormone fluctuation…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jul 23, 2026 00:00 UTC