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Looking for participantsPhase2/Phase3

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

Sponsor: Crinetics Pharmaceuticals Inc.

NCT ID: NCT07159841

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Atumelnant (drug), Placebo (drug)
How long the study runs
Study runs about 50 months (dates as stated)
About the drug or intervention
Atumelnant — drug: Atumelnant, tablets, once daily by mouth, weight-based dosing · Placebo — drug: Placebo, tablets, once daily by mouth, weight-based dosing
Patient visit burden
Not specified by the sponsor

In plain English

This study, called Balance-CAH, is looking at children and teenagers aged 1 to under 18 with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency (a condition where the body cannot make certain hormones properly). The study is run by Crinetics Pharmaceuticals Inc. and has three parts (A, B and C).

Who can take part

  • Aged 1 to under 18 years at the time of signing the consent form (Parts A and B)
  • A confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency, based on tests such as raised 17-OHP levels, genetic testing, newborn screening with a confirmatory test, or a stimulation test
  • A raised morning blood level of a hormone called androstenedione (A4), measured before the morning glucocorticoid (a type of steroid medicine) dose
  • Already taking a stable glucocorticoid replacement medicine for at least one month before screening
  • Following their glucocorticoid and, if needed, mineralocorticoid medicine routine as judged by the study doctor
  • Having normal thyroid function, as judged by the study doctor
  • For Part C: having finished treatment in Part A or Part B, and the study doctor believing it would benefit the person to continue, whatever their age

Who may not be able to

  • Having any form of CAH other than classic 21-hydroxylase deficiency
  • Being treated with other glucocorticoid medicines within 30 days before screening
  • Having had a 'stress dose' of glucocorticoid medicine (a higher-than-normal dose, including by injection) within 2 weeks before screening started
  • Using growth hormone medicines (short-acting within 1 week, or long-acting within 6 weeks before screening started)
  • Using a type of medicine called a corticotropin-releasing factor receptor antagonist within 14 days before screening
  • A history of cancer, apart from certain fully treated skin cancers (basal or squamous cell carcinoma) or an early-stage cervical cancer that was removed or treated
  • Unusual sleep and wake patterns, as judged by the study doctor
  • Participants who are pregnant or breastfeeding
  • Having taken an experimental drug (including one called atumelnant) in another study within 60 days or 5 half-lives (whichever is longer) before the first dose
  • For Part C: not meeting the Part C entry criteria

What taking part involves

  • • Not stated — ask the trial team

Time commitment: Not stated — ask the trial team (the study involves screening checks, and children who finish Part A or B may be invited to continue into Part C).

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
1 Year to 17 Years
Who
All
Number of participants
153
Started
2026-01-22
Last checked
2026-06

Plain English Summary

What is this study?

  • • Testing a new treatment for congenital adrenal hyperplasia
  • • Phase2/Phase3 - 153 participants
  • • 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)

Who can take part?

  • • Ages 1 Year to 17 Years
  • • Diagnosed with congenital adrenal hyperplasia

Where?

  • • Liverpool - Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital
  • • London - Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital
  • • Manchester - Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital
  • • Sheffield - Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank

This is a simplified summary. Always discuss with your doctor before making any decisions.

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).

More detail

This Phase 2/3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE). A total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to \< 18 years old. The first 3 cohorts in Part A are for ages 12 to \<18 years and will be semi-sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.

Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia

How this trial compares with your answers

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 1 Year - 17 Years
  • Who can join: All genders

Treatment history

Treatments you must have had:

  • ✓ an elevated morning serum A4 level \>ULN during Screening obtained

What the study is looking for

  • ✓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 agreement to take part...
  • ✓Participants must have an elevated morning serum A4 level \>ULN during Screening obtained prior to morning...
  • ✓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...

Who cannot take part

  • ✗Part A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from...
  • ✗Diagnosis of any form of CAH other than classic 21-OHD.
  • ✗Participants treated with other GCs within 30 days of Screening.
  • ✗Use of growth hormones within 1 week of start of Screening for short acting, or within 6 weeks of start of Screening...
  • ✗Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.
See the full 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. 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 Is This Study? (4 UK sites)

Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital

Liverpool L12 2AP, United Kingdom

Recruiting
Hospital R&D contact (matched)

Kelly Davies

research@alderhey.nhs.uk0151 2525570

Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital

London WC1N 3JH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank

Sheffield S10 2TH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Alessia Dunn

STH.ResearchAdministration@nhs.net0114 2712550

How to Get in Touch

Crinetics Clinical Trials

Sponsor contact

CONTACT

833-827-9741 clinicaltrials@crinetics.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-06