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

NCT07159841 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CRN04894-132024-519578-38-00
• Study Type: interventional
• Target: 153
• Locations: 10 countries
• Sites: 32

Brief 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).

Conditions

Congenital Adrenal Hyperplasia; Classic Congenital Adrenal Hyperplasia

Keywords

Congenital Adrenal Hyperplasia; CAH; CRN04894; Atumelnant; Pediatric; Balance-CAH

Outcome Measures

Primary Outcomes (3)

• Change from baseline in morning serum androstenedione (A4) (Part A)

  Week 8

• Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)

  Week 28

• Change from baseline in serum early morning A4 over time (Part C)

  Up to Week 260

Secondary Outcomes (8)

• Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A)

  Week 8

• Plasma and/or blood concentrations of atumelnant (Part A)

  Up to Week 8

• Change from baseline in serum early morning A4 (Part B)

  Week 4

• Change from baseline in serum early morning 17-OHP (Part B)

  Week 4

• Proportion of participants with physiologic GC dose while serum early morning A4 <ULN (Part B)

  Week 28

Study Arms (4)

Treatment (Part A)

EXPERIMENTAL

Open-label, semi-sequential cohorts.

Drug: Atumelnant

Active Treatment (Part B)

EXPERIMENTAL

Randomized, Parallel Arms, Double-Blind

Drug: Atumelnant

Placebo (Part B)

PLACEBO COMPARATOR

Randomized, Parallel Arms, Double-Blind

Drug: Placebo

Open-Label Treatment (Part C)

EXPERIMENTAL

Open-label treatment period for participants entering Part C from Part A and B.

Drug: Atumelnant

Interventions

Atumelnant DRUG

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

Also known as: CRN04894

Active Treatment (Part B); Open-Label Treatment (Part C); Treatment (Part A)

Placebo DRUG

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

Placebo (Part B)

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Part A and B participants are eligible to be included in the study only if all of the following criteria apply:
• Male or female at birth, between 1 to \<18 years of chronological age at the time of signing the Informed Consent Form (ICF).
• 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.
• Participants must have an elevated morning serum A4 level \>ULN during Screening obtained prior to morning glucocorticoid (GC) administration.
• Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.
• Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.
• Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range.

You may not qualify if:

• 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:
• Diagnosis of any form of CAH other than classic 21-OHD.
• Participants treated with other GCs within 30 days of Screening.
• 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.
• 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.
• Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening.
• History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ.
• Abnormal sleep/wake cycles (as determined by the Investigator).
• Female participants who are pregnant or lactating.
• Participants who have been dosed with an investigational drug (other than atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.
• Part C:

Sponsors & Collaborators

• Crinetics Pharmaceuticals Inc.: lead

Study Sites (32)

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55454, United States

RECRUITING

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Cook Children's Health Care System

Fort Worth, Texas, 76104, United States

RECRUITING

University of Virginia Health System

Charlottesville, Virginia, 22903, United States

RECRUITING

Instituto de Investigaciones Metabólicas

Buenos Aires, Buenos Aires, C1012AAR, Argentina

RECRUITING

Hospital de Niños de la Santísima Trinidad

Córdoba, Córdoba Province, X5000, Argentina

RECRUITING

Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB, Argentina

RECRUITING

Instituto Médico Especializado (IME)

Buenos Aires, C1405BCH, Argentina

RECRUITING

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

Buenos Aires, C1425EFD, Argentina

RECRUITING

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

Westmead, New South Wales, 2145, Australia

NOT YET RECRUITING

Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia

NOT YET RECRUITING

Monash Children's Hospital, Monash Health

Clayton, Victoria, 3168, Australia

RECRUITING

UZA (Antwerp University Hospital)

Edegem, Antwerp, 2650, Belgium

RECRUITING

UZ Gent (University Hospital Ghent)

Ghent, East Flanders, 9000, Belgium

RECRUITING

UZ Leuven (Universitair Ziekenhuis Leuven)

Leuven, Flemish Brabant, 3000, Belgium

RECRUITING

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

São Paulo, São Paulo, 05403-000, Brazil

RECRUITING

Centre Hospitalier Universitaire (CHU) d'Angers

Angers, 49100, France

RECRUITING

Hopital Kremlin-Bicétre - APHP Paris Saclay

Le Kremlin-Bicêtre, 94270, France

RECRUITING

Hopital Jeanne de Flandre - CHU de Lille

Lille, 59037, France

RECRUITING

APHM -Hopital La Timone Enfants

Marseille, 13385 Cedex 5, France

RECRUITING

Hopital Necker - Enfants Malades

Paris, 75015, France

RECRUITING

Hopital Robert Debre

Paris, 75019, France

RECRUITING

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

Berlin, 13353, Germany

RECRUITING

AOU Federico II

Naples, Campania, 80131, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genoa, Liguria, 16147, Italy

RECRUITING

IRCCS Ospedale San Raffaele

Milan, Lombardy, 20132, Italy

RECRUITING

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, Tuscany, 50139, Italy

RECRUITING

lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych

Lodz, Poland, 93-338, Poland

RECRUITING

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

RECRUITING

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

Sheffield, S10 2TH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Central Study Contacts

Crinetics Clinical Trials

CONTACT

833-827-9741; clinicaltrials@crinetics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2025
• First Posted: September 8, 2025
• Study Start: January 22, 2026
• Primary Completion (Estimated): March 1, 2030
• Study Completion (Estimated): March 1, 2030
• Last Updated: April 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1); GB (1); BR (1); US (6); PL (2); FR (6); IT (4); BE (3); AR (5); AU (3)