Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency
MONDO:0008728The most common form of congenital adrenal hyperplasia (CAH), characterized by simple virilizing or salt wasting forms that can manifest with genital ambiguity in females and with adrenal insufficiency (in both sexes), and that presents with dehydration, hypoglycemia in the neonatal period (that can be lethal if untreated), and hyperandrogenia.
Also known as: 21-OHD, classic 21-OHD CAH, classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, 21 hydroxylase deficiency, 21-hydroxylase deficiency, CYP21 deficiency, adrenal hyperplasia 3, adrenal hyperplasia, congenital, due to 21-HYDROXYLASE deficiency
58 clinical trials for this condition and its sub-types.
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New hope for kids with rare hormone disorder: drug may cut steroid doses
Disease control TerminatedThis study tested an experimental drug called tildacerfont in 67 children aged 2 to 17 with congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The goal was to see if adding this once-daily pill to standard steroid treatment could improve d…
Phase: PHASE2 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Prostate cancer drug trialed to reduce steroids in kids with rare hormone disorder
Disease control TerminatedThis early-phase study tested a drug called abiraterone acetate (normally used for prostate cancer) in 4 children with congenital adrenal hyperplasia (CAH). The goal was to see if it could lower high male hormone levels and reduce the need for strong steroid medications that can …
Phase: PHASE1 • Sponsor: University of Texas Southwestern Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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New drug aims to cut steroid use in rare hormone disorder
Disease control TerminatedThis study tested a daily tablet called Tildacerfont in 100 adults with classic congenital adrenal hyperplasia, a condition where the body can't make certain hormones properly. The goal was to see if the drug could safely reduce the high doses of steroids patients need to take. T…
Phase: PHASE2 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC