Steroid inherited metabolic disorder
MONDO:0005523Errors in metabolic processing of steroids resulting from inborn genetic mutations that are inherited or acquired in utero.
39 clinical trials for this condition and its sub-types, 0 tagged with Steroid inherited metabolic disorder itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Steroid inherited metabolic disorder
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Congenital adrenal hyperplasia 36 trials · 38 incl. sub-types
8 sub-types
- Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency 20 trials Sub-types →
- Classic congenital adrenal hyperplasia 5 trials
- Congenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency 1 trial
- Congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency 1 trial
- Congenital adrenal hyperplasia due to 3-beta-hydroxysteroid dehydrogenase deficiency 1 trial
- Congenital adrenal hyperplasia due to cytochrome P450 oxidoreductase deficiency 1 trial
- Congenital lipoid adrenal hyperplasia due to STAR deficency 1 trial Sub-types →
- Non-classic congenital adrenal hyperplasia 0 trials
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Congenital bile acid synthesis defect 1 trial · 2 incl. sub-types
6 sub-types
Most studied deeper sub-types
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New hope for kids with rare hormone disorder: drug may cut steroid doses
Disease control Stopped earlyThis 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 2 • 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 Stopped earlyThis 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 1 • 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 Stopped earlyThis 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 2 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC