A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH) (NCT07159841) | Clinical Trial Compass
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
United States153 participantsStarted 2026-01-22
Plain-language summary
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
Who can participate
Age range1 Year – 17 Years
SexALL
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Inclusion criteria
✓. Male or female at birth, between 1 to \<18 years of chronological age at the time of signing the Informed Consent Form (ICF).
✓. Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency (21-OHD) based on standard medically accepted criteria such as elevated 17-OHP level, confirmed CYP21A2 genetic testing, positive newborn screening with confirmatory second tier testing, or cosyntropin stimulation.
✓. Participants must have an elevated morning serum A4 level \>ULN during Screening obtained prior to morning glucocorticoid (GC) administration.
✓. Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.
✓. Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.
✓. Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range.
Exclusion criteria
✕. Diagnosis of any form of CAH other than classic 21-OHD.
✕. Participants treated with other GCs within 30 days of Screening.
✕. Stress dose of GC therapy within 2 weeks of start of Screening, defined as any dose above the normal maintenance dose, including but not limited to intravenous (IV) or intramuscular (IM) hydrocortisone.
✕. Use of growth hormones within 1 week of start of Screening for short acting, or within 6 weeks of start of Screening for long acting.
What they're measuring
1
Change from baseline in morning serum androstenedione (A4) (Part A)
Timeframe: Week 8
2
Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)
Timeframe: Week 28
3
Change from baseline in serum early morning A4 over time (Part C)