ClinicalTrials.gov record NCT07159841
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2/3 study evaluates once-daily oral atumelnant in children with classic congenital adrenal hyperplasia (CAH) to study safety, efficacy, and how the drug behaves in the body. The study has three parts: Part A is open-label semi-sequential cohorts, Part B is randomized and triple-masked with a placebo control, and Part C is an open-label long-term extension. About 153 participants ages 1 to <18 may be enrolled.
What it is studying. The study is testing atumelnant's safety and effects on hormone control in pediatric classic CAH, and characterizing its pharmacokinetics (PK) and pharmacodynamics (PD). Key measures include morning serum androstenedione and other adrenal hormones, and changes in glucocorticoid daily dose over time.
What the researchers hope to learn. To evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of atumelnant in children with classic CAH; to measure changes in morning serum androstenedione and 17-hydroxyprogesterone; and to assess whether glucocorticoid dosing can be changed and how outcomes evolve long-term in the extension.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Atumelnant
- Placebo
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
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:
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After screening, participants take study tablets by mouth once daily (weight‑based dosing). Part B is randomized and double‑blind, so some participants may receive placebo. Visits include regular clinic assessments and blood tests for hormone levels (such as A4 and 17‑OHP), safety monitoring, and drug level measurements; participants who finish Part A or B may enter a longer open‑label follow‑up (Part C) with assessments recorded up to Week 260.
How the study is structured. An interventional Phase 2/3 drug study with three parts: Part A (open-label, semi-sequential cohorts), Part B (randomized, triple-masked, placebo-controlled), and Part C (open-label extension for participants from Parts A or B). Participants receive daily oral tablets and undergo scheduled hormone testing and PK/PD assessments.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Argentina, Australia, Belgium, Brazil, Chile, France, Germany, Italy, Netherlands, Poland, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on congenital adrenal hyperplasia
Important dates
- Study start
- Jan 22, 2026
- Primary completion
- March 2030 (estimated)
- Study completion
- March 2030 (estimated)
- Target enrollment
- 153 participants (estimated)
- Last updated on registry
- Sep 30, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Crinetics Pharmaceuticals Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Crinetics Clinical Trials 📞 833-827-9741 ✉ clinicaltrials@crinetics.com
Participating sites (49)
- UCSF Ron Conway Gateway Medical BuildingRecruiting
San Francisco, California, 94158 · United States
- Ann and Robert H. Lurie Children's Hospital of ChicagoRecruiting
Chicago, Illinois, 60611 · United States
- Boston's Children's HospitalRecruiting
Boston, Massachusetts, 02115 · United States
- University of MichiganRecruiting
Ann Arbor, Michigan, 48109 · United States
- University of MinnesotaRecruiting
Minneapolis, Minnesota, 55454 · United States
- Rutgers Robert Wood Johnson Medical SchoolRecruiting
New Brunswick, New Jersey, 08901 · United States
- Icahn School of Medicine at Mount Sinai-Mount Sinai HospitalRecruiting
New York, New York, 10029 · United States
- Children's Hospital of PhiladelphiaRecruiting
Philadelphia, Pennsylvania, 19104 · United States
- Cook Children's Health Care SystemRecruiting
Fort Worth, Texas, 76104 · United States
- University of Virginia Health SystemRecruiting
Charlottesville, Virginia, 22903 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- pharmacokinetics
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- Informed Consent Form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- during Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- glucocorticoid
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- investigational drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- open-label extension
- A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
- Phase 2
- A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- Phase 3
- A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- placebo controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
← Back to clinical trials