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New pill could help kids with rare hormone disorder cut back on steroids

NCT ID NCT07159841

Disease control Sponsor: Crinetics Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 03, 2026 · Updated 7 times

Summary

This study tests a daily oral medication called atumelnant in children aged 1 to 18 with classic congenital adrenal hyperplasia, a genetic condition that disrupts hormone production. The goal is to see if the drug can safely normalize hormone levels and allow patients to lower their daily steroid doses. The trial includes a placebo group and a long-term extension to monitor lasting effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
atumelnant (CRN04894), a daily oral tablet
What this could lead to
If successful, this could offer a new treatment option that helps children with congenital adrenal hyperplasia better control their hormone levels and reduce their dependence on high-dose steroids.
What could go wrong
This is an early-to-mid-stage trial with only 153 participants, so results may not apply to all patients. The drug may cause side effects or fail to show clear benefit over placebo.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 153 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2026

Expected to finish

Mar 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

classic congenital adrenal hyperplasia classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency congenital adrenal hyperplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Classic congenital adrenal hyperplasia Congenital adrenal hyperplasia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    48 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AOU Federico II

    RECRUITING

    Naples, Campania, 80131, Italy

  • APHM -Hopital La Timone Enfants

    RECRUITING

    Marseille, 13385 Cedex 5, France

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

    RECRUITING

    Liverpool, L12 2AP, United Kingdom

  • Azienda Ospedaliera Universitaria Meyer IRCCS

    RECRUITING

    Florence, Tuscany, 50139, Italy

  • Boston's Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez

    RECRUITING

    Buenos Aires, C1425EFD, Argentina

  • Centre Hospitalier Universitaire (CHU) d'Angers

    RECRUITING

    Angers, 49100, France

  • Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie

    RECRUITING

    Berlin, 13353, Germany

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Cliniques Universitaires Saint-Luc

    RECRUITING

    Brussels, Brussels Capital, 1200, Belgium

  • Cook Children's Health Care System

    RECRUITING

    Fort Worth, Texas, 76104, United States

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

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hopital Jeanne de Flandre - CHU de Lille

    RECRUITING

    Lille, 59037, France

  • Hopital Kremlin-Bicétre - APHP Paris Saclay

    RECRUITING

    Le Kremlin-Bicêtre, 94270, France

  • Hopital Necker - Enfants Malades

    RECRUITING

    Paris, 75015, France

  • Hopital Robert Debre

    RECRUITING

    Paris, 75019, France

  • Hospital Italiano de Buenos Aires

    RECRUITING

    Buenos Aires, C1199ABB, Argentina

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)

    RECRUITING

    São Paulo, São Paulo, 05403-000, Brazil

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo

    RECRUITING

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital de Niños de la Santísima Trinidad

    RECRUITING

    Córdoba, Córdoba Province, X5000, Argentina

  • Hospital las Hilgueras de Talcahuano

    RECRUITING

    Talcahuano, 4270918, Chile

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genoa, Liguria, 16147, Italy

  • IRCCS Ospedale San Raffaele

    RECRUITING

    Milan, Lombardy, 20132, Italy

  • Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital

    RECRUITING

    New York, New York, 10029, United States

  • Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead

    NOT_YET_RECRUITING

    Westmead, New South Wales, 2145, Australia

  • Instituto Médico Especializado (IME)

    RECRUITING

    Buenos Aires, C1405BCH, Argentina

  • Instituto de Investigaciones Materno Infantil IDIMI, Facultad de Medicina de la Universidad de Chile

    RECRUITING

    Santiago, Santiago Metropolitan, 8360160, Chile

  • Instituto de Investigaciones Metabólicas

    RECRUITING

    Buenos Aires, Buenos Aires, C1012AAR, Argentina

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

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Monash Children's Hospital, Monash Health

    RECRUITING

    Clayton, Victoria, 3168, Australia

  • Nucleo de Pesquisa Clinica do Rio Grande do Sul

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90430-001, Brazil

  • Perth Children's Hospital

    NOT_YET_RECRUITING

    Nedlands, Western Australia, 6009, Australia

  • Queensland Children's Hospital

    NOT_YET_RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Radboudumc Amalia Children's Hospital, Department of Pediatrics

    RECRUITING

    Nijmegen, Gelderland, 6525 GA, Netherlands

  • Rutgers Robert Wood Johnson Medical School

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

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

    RECRUITING

    Sheffield, S10 2TH, United Kingdom

  • UCSF Ron Conway Gateway Medical Building

    RECRUITING

    San Francisco, California, 94158, United States

  • UZ Gent (University Hospital Ghent)

    RECRUITING

    Ghent, East Flanders, 9000, Belgium

  • UZ Leuven (Universitair Ziekenhuis Leuven)

    RECRUITING

    Leuven, Flemish Brabant, 3000, Belgium

  • UZA (Antwerp University Hospital)

    RECRUITING

    Edegem, Antwerp, 2650, Belgium

  • Universitatsklinikum Koln Klinik und Poliklinik fur Kinder- und Jugendmedizin

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

  • University of Virginia Health System

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin

    RECRUITING

    Tübingen, Baden, 72076, Germany

  • Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku, Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii

    RECRUITING

    Bialystok, Podlaskie Voivodeship, 15-274, Poland

  • Uniwersytecki Szpital Kliniczny Nr 1 im. Prof. Tadeusza Sokotowskiego PUM w Szczecinie, Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie

    RECRUITING

    Szczecin, West Pomeranian Voivodeship, 71-252, Poland

  • lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych

    RECRUITING

    Lodz, Poland, 93-338, Poland

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Other studies related to the condition(s) this trial covers.