Congenital Adrenal Hyperplasia
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-06-09EMA Authorised: Efmody (hydrocortisone) — Immedica Pharma AB
Efmody (hydrocortisone) by Immedica Pharma AB has been authorised for congenital adrenal hyperplasia in the EU.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Natural History Study of Patients With Excess Androgen
A NICHD-sponsored natural history study examining excess androgen in congenital adrenal hyperplasia and familial male-limited precocious pub…
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CLINICAL_TRIALS research · 2026-04-17Clinical Trial Update: Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old [Phase 2]
A Phase 2 clinical trial evaluating the safety, tolerability, and pharmacokinetics/pharmacodynamics of crinecerfont in children under 4 with…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH) [Phase 2, Phase 3]
A pediatric Phase 2/3 clinical trial evaluating Crinecerfont for congenital adrenal hyperplasia (classic CAH) is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: Parenting and CAH - 21-hydroxylase Deficiency
This clinical trial update shows that NCT06900153 in Paris is currently recruiting to study parenting in families affected by congenital adr…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia [Phase 3]
This clinical trial update shows a Phase 3 study evaluating Atumelnant in adults with congenital adrenal hyperplasia, including the classic …
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old [Phase 2]
A Phase 2 clinical trial update evaluating the pharmacokinetics, safety and tolerability of Crinecerfont in infants under 2 years with conge…
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: Syndromes With Neonatal Salt Loss: Not Only Congenital Adrenal Hyperplasia Due to 21-hydroxylase Deficiency (21OH-ISC)
A clinical trial investigating syndromes causing neonatal salt loss beyond congenital adrenal hyperplasia due to 21-hydroxylase deficiency i…
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CLINICAL_TRIALS trial_result · 2026-03-12Clinical Trial Update: Retrospective Multicentre Study on the Impact of Neonatal Screening for Congenital Adrenal Hyperplasia in Italy
A retrospective multicentre Italian study evaluating the impact of neonatal screening for congenital adrenal hyperplasia on clinical outcome…
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CLINICAL_TRIALS research · 2026-03-09Clinical Trial Update: A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia [Phase 1, Phase 2]
A Phase 1/Phase 2 clinical trial of Lu AG13909 for congenital adrenal hyperplasia is currently recruiting.
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EMA approval · 2026-02-23EMA Authorised: Efmody (hydrocortisone) — Immedica Netherlands B.V.
EMA authorised Efmody (hydrocortisone) by Immedica Netherlands B.V. for congenital adrenal hyperplasia.
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CLINICAL_TRIALS trial_result · 2026-02-04Clinical Trial Update: Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia [Phase 3]
This document reports the completion of a Phase 3 long-term safety study evaluating Chronocort for congenital adrenal hyperplasia.
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH [Phase 2]
Phase 2 study evaluating safety, efficacy, and pharmacokinetics of tildacerfont in children aged 2-17 with congenital adrenal hyperplasia (2…
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CLINICAL_TRIALS research · 2025-12-23Clinical Trial Update: A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [Phase 1, Phase 2]
A status update for an ongoing Phase 1/2 gene therapy study for classic congenital adrenal hyperplasia, sponsored by Adrenas Therapeutics In…
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CLINICAL_TRIALS research · 2025-12-11Clinical Trial Update: Prenatal Dex Study [NA]
Clinical trial NCT02795871 evaluating prenatal dexamethasone for congenital adrenal hyperplasia has completed.
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CLINICAL_TRIALS withdrawal · 2025-12-10Clinical Trial Update: A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia [NA]
The clinical trial evaluating a virtual education-based transition intervention for adolescents and young adults with congenital adrenal hyp…
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CLINICAL_TRIALS trial_result · 2025-11-04Clinical Trial Update: Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1 [Phase 1]
A Phase 1 trial investigating androgen reduction in congenital adrenal hyperplasia was terminated, with results posted.
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CLINICAL_TRIALS trial_result · 2025-10-22Clinical Trial Update: Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia [Phase 2]
Phase 2 trial of SPR001 in adults with classic congenital adrenal hyperplasia has completed and results have been posted.
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CLINICAL_TRIALS trial_result · 2025-10-01Clinical Trial Update: A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH [Phase 2]
Phase 2b study of tildacerfont in adults with congenital adrenal hyperplasia was terminated, with results posted.
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CLINICAL_TRIALS trial_result · 2025-09-23Clinical Trial Update: Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn) [Phase 2]
Phase 2 clinical trial evaluating CRN04894 for safety, efficacy, and pharmacokinetics in congenital adrenal hyperplasia has completed enroll…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: An Extension Study to Evaluate Safety and Efficacy in Participants Treated With CRN04894 [Phase 2]
Extension phase 2 clinical trial to assess safety and efficacy of CRN04894 in participants with congenital adrenal hyperplasia; currently re…
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CLINICAL_TRIALS research · 2025-09-16Clinical Trial Update: Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment [Phase 4]
A Phase 4 clinical trial is evaluating once-daily hydrocortisone for congenital adrenal hyperplasia, with recruitment currently ongoing.
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CLINICAL_TRIALS trial_result · 2025-09-08Clinical Trial Update: Investigation of the Etiology of Hypertension and Endothelial Damage in Patients With Cytochrome P450 Oxidoreductase Deficiency
A completed clinical trial investigating the causes of hypertension and endothelial damage in individuals with cytochrome P450 oxidoreductas…
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CLINICAL_TRIALS trial_result · 2025-08-12Clinical Trial Update: Baby Detect : Genomic Newborn Screening
Clinical trial update on the Baby Detect genomic newborn screening program, reporting completion of a study assessing screening for multiple…
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CLINICAL_TRIALS research · 2025-08-01Clinical Trial Update: Fertility And Sexual Function In CAH: CALLIOPE
A CALLIOPE study evaluating fertility and sexual function in congenital adrenal hyperplasia is currently recruiting, sponsored by the Univer…