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

NCT ID NCT07159841

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • AOU Federico II

    RECRUITING

    Naples, Campania, 80131, Italy

  • APHM -Hopital La Timone Enfants

    RECRUITING

    Marseille, 13385 Cedex 5, France

  • 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

  • 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 de Niños de la Santísima Trinidad

    RECRUITING

    Córdoba, Córdoba Province, X5000, Argentina

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genoa, Liguria, 16147, Italy

  • IRCCS Ospedale San Raffaele

    RECRUITING

    Milan, Lombardy, 20132, Italy

  • 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 Metabólicas

    RECRUITING

    Buenos Aires, Buenos Aires, C1012AAR, Argentina

  • Monash Children's Hospital, Monash Health

    RECRUITING

    Clayton, Victoria, 3168, Australia

  • Perth Children's Hospital

    NOT_YET_RECRUITING

    Nedlands, Western Australia, 6009, Australia

  • Queensland Children's Hospital

    NOT_YET_RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • 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

  • 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

  • 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

  • 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