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NCT07159841PHASE2, PHASE3Recruiting

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

Crinetics Pharmaceuticals Inc.

Start Date

1/22/2026

Completion Date

3/1/2030

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).

Detailed Description

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.

Eligibility Criteria

Age Range: 1 years to 17 years

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.

Interventions

DRUG

Atumelnant

DRUG

Placebo

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Conditions

Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia

Locations

UCSF Ron Conway Gateway Medical Building

San Francisco, California 94158

United States

Boston's Children's Hospital

Boston, Massachusetts 02115

United States

University of Michigan

Ann Arbor, Michigan 48109

United States

University of Minnesota

Minneapolis, Minnesota 55454

United States

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey 08901

United States

Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital

New York, New York 10029

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Cook Children's Health Care System

Fort Worth, Texas 76104

United States

University of Virginia Health System

Charlottesville, Virginia 22903

United States

Instituto de Investigaciones Metabólicas

Buenos Aires, Buenos Aires C1012AAR

Argentina

Hospital de Niños de la Santísima Trinidad

Córdoba, Córdoba Province X5000

Argentina

Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB

Argentina

Instituto Médico Especializado (IME)

Buenos Aires, C1405BCH

Argentina

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

Buenos Aires, C1425EFD

Argentina

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

Westmead, New South Wales 2145

Australia

Queensland Children's Hospital

South Brisbane, Queensland 4101

Australia

Monash Children's Hospital, Monash Health

Clayton, Victoria 3168

Australia

Perth Children's Hospital

Nedlands, Western Australia 6009

Australia

UZA (Antwerp University Hospital)

Edegem, Antwerp 2650

Belgium

Cliniques Universitaires Saint-Luc

Brussels, Brussels Capital 1200

Belgium

UZ Gent (University Hospital Ghent)

Ghent, East Flanders 9000

Belgium

UZ Leuven (Universitair Ziekenhuis Leuven)

Leuven, Flemish Brabant 3000

Belgium

Nucleo de Pesquisa Clinica do Rio Grande do Sul

Porto Alegre, Rio Grande do Sul 90430-001

Brazil

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

Ribeirão Preto, São Paulo 14051-140

Brazil

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

São Paulo, São Paulo 05403-000

Brazil

Centre Hospitalier Universitaire (CHU) d'Angers

Angers, 49100

France

Hopital Kremlin-Bicétre - APHP Paris Saclay

Le Kremlin-Bicêtre, 94270

France

Hopital Jeanne de Flandre - CHU de Lille

Lille, 59037

France

APHM -Hopital La Timone Enfants

Marseille, 13385 Cedex 5

France

Hopital Necker - Enfants Malades

Paris, 75015

France

Hopital Robert Debre

Paris, 75019

France

Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin

Tübingen, Baden 72076

Germany

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

Berlin, 13353

Germany

AOU Federico II

Naples, Campania 80131

Italy

IRCCS Istituto Giannina Gaslini

Genoa, Liguria 16147

Italy

IRCCS Ospedale San Raffaele

Milan, Lombardy 20132

Italy

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, Tuscany 50139

Italy

Radboudumc Amalia Children's Hospital, Department of Pediatrics

Nijmegen, Gelderland 6525 GA

Netherlands

lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych

Lodz, Poland 93-338

Poland

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

Szczecin, West Pomeranian Voivodeship 71-252

Poland

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

London, WC1N 3JH

United Kingdom

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

Manchester, M13 9WL

United Kingdom

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

Sheffield, S10 2TH

United Kingdom