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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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.

Where it's running

• Children's Hospital Los Angeles
  Los Angeles, California, United States Recruiting
• UCSF Ron Conway Gateway Medical Building
  San Francisco, California, United States Recruiting
• Ann and Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Boston's Children's Hospital
  Boston, Massachusetts, United States Recruiting
• University of Michigan
  Ann Arbor, Michigan, United States Recruiting
• University of Minnesota
  Minneapolis, Minnesota, United States Recruiting

+3 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Crinetics Clinical Trials
• Email: clinicaltrials@crinetics.com
• Phone: 833-827-9741

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Atumelnant for adults with classic congenital adrenal hyperplasia

  This will test whether taking once-daily atumelnant helps adults with classic 21‑hydroxylase deficiency CAH who are already on stable glucocorticoid therapy.

  Recruiting at 82 sites Phase 3 Crinetics Pharmaceuticals Inc. · Los Angeles, California and 81 other locations · NCT07144163

• Long-term evaluation of atumelnant for congenital adrenal hyperplasia

  This study is testing if a new treatment called atumelnant is safe and effective for people with congenital adrenal hyperplasia who have already participated in a related study.

  Recruiting at 10 sites Phase 2 Crinetics Pharmaceuticals Inc. · Minneapolis, Minnesota and 12 other locations · NCT06712823

• Pediatric Congenital Adrenal Hyperplasia: Long-Term Outcomes and Feelings of Regret

  This study looks at adults who had classic CAH from 21-hydroxylase deficiency as children to see if early surgeries and management choices are linked to long‑term satisfaction or regret.

  Recruiting University Hospital, Lille · Lille · NCT07781267

• Crinecerfont safety testing in babies with classic CAH under 4 years old

  This study tests whether crinecerfont is safe and tolerable in children under 4 years with classic CAH who have been on hydrocortisone.

  Recruiting at 11 sites Phase 2 Neurocrine Biosciences · San Francisco, California and 10 other locations · NCT07536269

• Registry for male patients with congenital adrenal hyperplasia

  This study is creating a registry for boys with congenital adrenal hyperplasia to better understand the condition and track how common it is in the Emilia Romagna region.

  Recruiting IRCCS Azienda Ospedaliero-Universitaria di Bologna · Bologna · NCT06754423

• Registry for Rare Diseases

  This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.

  Recruiting Hospital Italiano de Buenos Aires · Buenos Aires, Buenos Aires · NCT06573723

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.