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Atumelnant for children with classic congenital adrenal hyperplasia (Balance-CAH)

A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension

Phase2; Phase3 Interventional Crinetics Pharmaceuticals Inc. · NCT07159841

This study will test whether atumelnant is safe and helps control hormone levels in children aged 1 to under 18 with classic congenital adrenal hyperplasia.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment153 (estimated)
Ages1 Year to 17 Years
SexAll
SponsorCrinetics Pharmaceuticals Inc. Industry-sponsored
Locations32 sites (Ann Arbor, Michigan and 31 other locations)
Trial IDNCT07159841 on ClinicalTrials.gov

What this trial studies

This Phase 2/3 program includes an open-label, semi-sequential Phase 2 portion (Part A), a double-blind, randomized, placebo-controlled Phase 3 confirmation portion (Part B), and an open-label extension (Part C) for ongoing treatment. Approximately 153 pediatric participants ages 1 to <18 may be enrolled, with initial cohorts enrolling ages 12 to <18 sequentially and a later cohort for ages 1 to 11 after safety reviews. The study will measure safety, pharmacokinetics (PK), pharmacodynamics (PD), and effects on adrenal hormone control while participants remain on stable supraphysiologic glucocorticoid replacement. A Safety Review Committee will review interim data before progression between cohorts, and participants from Parts A and B may roll into the open-label extension.

Who should consider this trial

Good fit: Ideal candidates are children aged 1 to <18 with a confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency, an elevated morning serum androstenedione (A4) above the normal range, and who have been on a stable supraphysiologic glucocorticoid replacement regimen for at least one month.

Not a fit: Patients who have non-classic CAH, are outside the 1 to <18 age range, cannot maintain a stable glucocorticoid regimen, or have other major medical contraindications are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, atumelnant could improve hormonal control in children with classic CAH and potentially allow lower glucocorticoid doses, reducing long-term steroid side effects.

How similar studies have performed: This is a relatively new targeted therapy approach in pediatric CAH with limited prior pediatric data, and early-phase findings supported advancing to this combined Phase 2/3 program.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

Part A and B participants are eligible to be included in the study only if all of the following criteria apply:

1. Male or female at birth, between 1 to \<18 years of chronological age at the time of signing the Informed Consent Form (ICF).
2. 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.
3. Participants must have an elevated morning serum A4 level \>ULN during Screening obtained prior to morning glucocorticoid (GC) administration.
4. Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.
5. Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.
6. Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range.

Part C inclusion criteria require participants to complete treatment in either Part A or Part B and in the Investigator's opinion it would benefit the participant to continue in Part C, regardless of age.

Exclusion Criteria:

Part A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from participation in this study:

1. Diagnosis of any form of CAH other than classic 21-OHD.
2. Participants treated with other GCs within 30 days of Screening.
3. 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.
4. 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.
5. Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.
6. History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ.
7. Abnormal sleep/wake cycles (as determined by the Investigator).
8. Female participants who are pregnant or lactating.
9. Participants who have been dosed with an investigational drug (other than atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.

   Part C:
10. Individuals in Part C who do not meet the Part C Inclusion Criteria.

Where this trial is running

Ann Arbor, Michigan and 31 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Congenital Adrenal HyperplasiaClassic Congenital Adrenal HyperplasiaCAHCRN04894AtumelnantPediatricBalance-CAH
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.