New pill could help kids with rare hormone disorder cut back on steroids
NCT ID NCT07159841
First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 5 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AOU Federico II
RECRUITINGNaples, Campania, 80131, Italy
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APHM -Hopital La Timone Enfants
RECRUITINGMarseille, 13385 Cedex 5, France
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Azienda Ospedaliera Universitaria Meyer IRCCS
RECRUITINGFlorence, Tuscany, 50139, Italy
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Boston's Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez
RECRUITINGBuenos Aires, C1425EFD, Argentina
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Centre Hospitalier Universitaire (CHU) d'Angers
RECRUITINGAngers, 49100, France
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Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie
RECRUITINGBerlin, 13353, Germany
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Cliniques Universitaires Saint-Luc
RECRUITINGBrussels, Brussels Capital, 1200, Belgium
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Cook Children's Health Care System
RECRUITINGFort Worth, Texas, 76104, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hopital Jeanne de Flandre - CHU de Lille
RECRUITINGLille, 59037, France
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Hopital Kremlin-Bicétre - APHP Paris Saclay
RECRUITINGLe Kremlin-Bicêtre, 94270, France
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Hopital Necker - Enfants Malades
RECRUITINGParis, 75015, France
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Hopital Robert Debre
RECRUITINGParis, 75019, France
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Hospital Italiano de Buenos Aires
RECRUITINGBuenos Aires, C1199ABB, Argentina
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
RECRUITINGSão Paulo, São Paulo, 05403-000, Brazil
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Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
RECRUITINGRibeirão Preto, São Paulo, 14051-140, Brazil
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Hospital de Niños de la Santísima Trinidad
RECRUITINGCórdoba, Córdoba Province, X5000, Argentina
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Hospital las Hilgueras de Talcahuano
RECRUITINGTalcahuano, 4270918, Chile
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IRCCS Istituto Giannina Gaslini
RECRUITINGGenoa, Liguria, 16147, Italy
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IRCCS Ospedale San Raffaele
RECRUITINGMilan, Lombardy, 20132, Italy
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Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital
RECRUITINGNew York, New York, 10029, United States
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Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead
NOT_YET_RECRUITINGWestmead, New South Wales, 2145, Australia
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Instituto Médico Especializado (IME)
RECRUITINGBuenos Aires, C1405BCH, Argentina
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Instituto de Investigaciones Metabólicas
RECRUITINGBuenos Aires, Buenos Aires, C1012AAR, Argentina
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Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital
RECRUITINGManchester, M13 9WL, United Kingdom
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Monash Children's Hospital, Monash Health
RECRUITINGClayton, Victoria, 3168, Australia
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Nucleo de Pesquisa Clinica do Rio Grande do Sul
RECRUITINGPorto Alegre, Rio Grande do Sul, 90430-001, Brazil
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Perth Children's Hospital
NOT_YET_RECRUITINGNedlands, Western Australia, 6009, Australia
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Queensland Children's Hospital
NOT_YET_RECRUITINGSouth Brisbane, Queensland, 4101, Australia
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Radboudumc Amalia Children's Hospital, Department of Pediatrics
RECRUITINGNijmegen, Gelderland, 6525 GA, Netherlands
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Rutgers Robert Wood Johnson Medical School
RECRUITINGNew Brunswick, New Jersey, 08901, United States
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Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank
RECRUITINGSheffield, S10 2TH, United Kingdom
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UCSF Ron Conway Gateway Medical Building
RECRUITINGSan Francisco, California, 94158, United States
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UZ Gent (University Hospital Ghent)
RECRUITINGGhent, East Flanders, 9000, Belgium
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UZ Leuven (Universitair Ziekenhuis Leuven)
RECRUITINGLeuven, Flemish Brabant, 3000, Belgium
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UZA (Antwerp University Hospital)
RECRUITINGEdegem, Antwerp, 2650, Belgium
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
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University of Virginia Health System
RECRUITINGCharlottesville, Virginia, 22903, United States
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Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin
RECRUITINGTübingen, Baden, 72076, Germany
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Uniwersytecki Szpital Kliniczny Nr 1 im. Prof. Tadeusza Sokotowskiego PUM w Szczecinie, Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
RECRUITINGSzczecin, West Pomeranian Voivodeship, 71-252, Poland
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lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych
RECRUITINGLodz, Poland, 93-338, Poland
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could a simple saliva test replace blood draws for hormone monitoring?
- New program aims to smooth healthcare transition for teens with rare hormone disorder
- New hope for CAH: drug may slash steroid use
- New hope for toddlers with rare hormone disorder?
- Italian study checks how well newborn screening detects rare hormone disorder
- New hormone treatment shows promise for rare adrenal disorder