Recruiting
Phase 2
Phase 3

Atumelnant

Sponsor:

Crinetics Pharmaceuticals Inc.

Code:

NCT07159841

Conditions

Congenital Adrenal Hyperplasia

Classic Congenital Adrenal Hyperplasia

Eligibility Criteria

Sex: All

Age: 1 - 17

Healthy Volunteers: Not accepted

Interventions

Atumelnant

Placebo

Study Details

Brief 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).

Conditions

Congenital Adrenal Hyperplasia

Classic Congenital Adrenal Hyperplasia

Study ID

NCT07159841

Start date

Jan 22, 2026

Status verified date

Jun, 2026

Completion date

Mar, 2030

Anticipated

Primary completion date

Mar, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 17

Healthy Volunteers: Not accepted

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. 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.

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 (including atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.
10. Individuals in Part C who do not meet the Part C Inclusion Criteria.

Study Design

Enrollment

153 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (Part A)

Open-label, semi-sequential cohorts.

experimental: Active Treatment (Part B)

Randomized, Parallel Arms, Double-Blind

placebo comparator: Placebo (Part B)

Randomized, Parallel Arms, Double-Blind

experimental: Open-Label Treatment (Part C)

Open-label treatment period for participants entering Part C from Part A and B.

Interventions

Atumelnant

Atumelnant, tablets, once daily by mouth, weight-based dosing

Placebo

Placebo, tablets, once daily by mouth, weight-based dosing

Primary outcome measure

  • Change from baseline in morning serum androstenedione (A4) (Part A) [ Time Frame: Week 8 ]
  • Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B) [ Time Frame: Week 28 ]
  • Change from baseline in serum early morning A4 over time (Part C) [ Time Frame: Up to Week 260 ]

Central Contacts and Locations

Central contacts

Locations

UCSF Ron Conway Gateway Medical Building

Recruiting

San Francisco, California, United States, 94158

Boston's Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55454

Rutgers Robert Wood Johnson Medical School

Recruiting

New Brunswick, New Jersey, United States, 08901

Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital

Recruiting

New York, New York, United States, 10029

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Cook Children's Health Care System

Recruiting

Fort Worth, Texas, United States, 76104

University of Virginia Health System

Recruiting

Charlottesville, Virginia, United States, 22903

More Information

Sponsor

Crinetics Pharmaceuticals Inc.

Last update posted

Sep 2, 2026

Last verified

Jun, 2026

Keywords

  • Congenital Adrenal Hyperplasia
  • CAH
  • CRN04894
  • Atumelnant
  • Pediatric
  • Balance-CAH

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Crinetics Pharmaceuticals Inc. on 2026-09-02.