A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
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
Interventions
Atumelnant
Placebo
Conditions
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