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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
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
Where Is This Study? (4 UK sites)
Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital
Liverpool L12 2AP, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital
Manchester M13 9WL, United Kingdom
Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank
Sheffield S10 2TH, United Kingdom
How to Get in Touch
Crinetics Clinical Trials
Sponsor contactCONTACT
