A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age1-17
SponsorCrinetics Pharmaceuticals Inc.
About this trial
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).
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Atumelnant
- Placebo
Treatment groups
153 Participants
are divided into 4 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov