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Classic congenital adrenal hyperplasia

MONDO:0060783

A severe form of congenital adrenal hyperplasia characterized by very low or absent activity of an enzyme in the steroidogenic pathway typically presenting early in life, and requiring life-long cortisol replacement.

Also known as: classic CAH, classic congenital adrenal hyperplasia

6 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Metabolic disease (233) Inherited lipid metabolism disorder (189) Hereditary disease (176) Endocrine system disorder (72) Inborn errors of metabolism (45) Congenital adrenal hyperplasia (31) Adrenocortical insufficiency (26) Hereditary endocrine growth disease (24) Human disease (14)
Trials to join now! 4 Not yet recruiting 1 Completed 1
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  • New pill could tame rare hormone disorder

    Disease control Completed

    This study tested a new oral drug called atumelnant in 38 adults with classic congenital adrenal hyperplasia, a genetic condition causing hormone imbalances. The drug works by blocking a hormone receptor to lower excess adrenal hormones. Researchers measured safety, side effects,…

    Phase: PHASE2 • Sponsor: Crinetics Pharmaceuticals Inc. • Aim: Disease control

    Last updated Jun 27, 2026 07:51 UTC

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