A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

Recruiting now · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia

In brief

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).

Key facts

Study ID

NCT07159841

Run by

Crinetics Pharmaceuticals Inc.

People needed

153

Starts

2026-01-22

Expected to finish

2030-03-01

Last updated by the study team

2026-07-24

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Part A and B participants are eligible to be included in the study only if all of the following criteria apply:
• 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.
• Biochemical euthyroidism as determined by the Investigator.
• 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.

You may not qualify if…

• 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:
• 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.
• Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.
• History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ.
• Abnormal sleep/wake cycles (as determined by the Investigator).
• Female participants who are pregnant or lactating.
• Participants who have been dosed with an investigational drug (including atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.
• Individuals in Part C who do not meet the Part C Inclusion Criteria.

Where it is running

• Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank — Sheffield, United Kingdom (enrolling)
• Boston's Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• Rutgers Robert Wood Johnson Medical School — New Brunswick, New Jersey, United States (enrolling)
• Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital — New York, New York, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Cook Children's Health Care System — Fort Worth, Texas, United States (enrolling)
• University of Virginia Health System — Charlottesville, Virginia, United States (enrolling)
• Instituto de Investigaciones Metabólicas — Buenos Aires, Buenos Aires, Argentina (enrolling)
• Hospital de Niños de la Santísima Trinidad — Córdoba, Córdoba Province, Argentina (enrolling)
• Hospital Italiano de Buenos Aires — Buenos Aires, Argentina (enrolling)
• Instituto Médico Especializado (IME) — Buenos Aires, Argentina (enrolling)
• CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez — Buenos Aires, Argentina (enrolling)
• Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital — London, United Kingdom (enrolling)
• Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital — Manchester, United Kingdom (enrolling)
• UCSF Ron Conway Gateway Medical Building — San Francisco, California, United States (enrolling)
• Monash Children's Hospital, Monash Health — Clayton, Victoria, Australia (enrolling)
• UZA (Antwerp University Hospital) — Edegem, Antwerp, Belgium (enrolling)
• Cliniques Universitaires Saint-Luc — Brussels, Brussels Capital, Belgium (enrolling)
• UZ Gent (University Hospital Ghent) — Ghent, East Flanders, Belgium (enrolling)
• UZ Leuven (Universitair Ziekenhuis Leuven) — Leuven, Flemish Brabant, Belgium (enrolling)
• Nucleo de Pesquisa Clinica do Rio Grande do Sul — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo — Ribeirão Preto, São Paulo, Brazil (enrolling)
• Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP) — São Paulo, São Paulo, Brazil (enrolling)

Full record on ClinicalTrials.gov

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