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Clinical Trials in the UK / NCT07159841
Recruiting Phase 2/3

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

NCT07159841 · tracked via the Priya Life Science UK tracker
Sponsor
Crinetics Pharmaceuticals Inc.
Phase
Phase 2/3
Started
2026-01-22
Last updated
2026-09-02

Condition(s) studied

Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia

Investigational drug(s) / intervention(s)

AtumelnantPlacebo

Atumelnant: Atumelnant, tablets, once daily by mouth, weight-based dosing

Placebo: Placebo, tablets, once daily by mouth, weight-based dosing

Study summary

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).

Eligibility

Sex
ALL
Min age
1 Year
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (48)

FacilityCityRegionStatus
UCSF Ron Conway Gateway Medical Building San Francisco California Recruiting
Boston's Children's Hospital Boston Massachusetts Recruiting
University of Michigan Ann Arbor Michigan Recruiting
University of Minnesota Minneapolis Minnesota Recruiting
Rutgers Robert Wood Johnson Medical School New Brunswick New Jersey Recruiting
Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital New York New York Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Cook Children's Health Care System Fort Worth Texas Recruiting
University of Virginia Health System Charlottesville Virginia Recruiting
Instituto de Investigaciones Metabólicas Buenos Aires Buenos Aires Recruiting
Hospital de Niños de la Santísima Trinidad Córdoba Córdoba Province Recruiting
Hospital Italiano de Buenos Aires Buenos Aires Argentina Recruiting
Instituto Médico Especializado (IME) Buenos Aires Argentina Recruiting
CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez Buenos Aires Argentina Recruiting
Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead Westmead New South Wales Not Yet Recruiting
Queensland Children's Hospital South Brisbane Queensland Not Yet Recruiting
Monash Children's Hospital, Monash Health Clayton Victoria Recruiting
Perth Children's Hospital Nedlands Western Australia Not Yet Recruiting
UZA (Antwerp University Hospital) Edegem Antwerp Recruiting
Cliniques Universitaires Saint-Luc Brussels Brussels Capital Recruiting
UZ Gent (University Hospital Ghent) Ghent East Flanders Recruiting
UZ Leuven (Universitair Ziekenhuis Leuven) Leuven Flemish Brabant Recruiting
Nucleo de Pesquisa Clinica do Rio Grande do Sul Porto Alegre Rio Grande do Sul Recruiting
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo Ribeirão Preto São Paulo Recruiting
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP) São Paulo São Paulo Recruiting
Instituto de Investigaciones Materno Infantil IDIMI, Facultad de Medicina de la Universidad de Chile Santiago Santiago Metropolitan Recruiting
Hospital las Hilgueras de Talcahuano Talcahuano Chile Recruiting
Centre Hospitalier Universitaire (CHU) d'Angers Angers France Recruiting
Hopital Kremlin-Bicétre - APHP Paris Saclay Le Kremlin-Bicêtre France Recruiting
Hopital Jeanne de Flandre - CHU de Lille Lille France Recruiting
APHM -Hopital La Timone Enfants Marseille France Recruiting
Hopital Necker - Enfants Malades Paris France Recruiting
Hopital Robert Debre Paris France Recruiting
Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin Tübingen Baden Recruiting
Universitatsklinikum Koln Klinik und Poliklinik fur Kinder- und Jugendmedizin Cologne North Rhine-Westphalia Recruiting
Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie Berlin Germany Recruiting
AOU Federico II Naples Campania Recruiting
IRCCS Istituto Giannina Gaslini Genoa Liguria Recruiting
IRCCS Ospedale San Raffaele Milan Lombardy Recruiting
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Tuscany Recruiting

+ 8 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07159841 on ClinicalTrials.gov ↗ ← All trials in the UK