Congenital Adrenal HyperplasiaPhase 2/3NCT07159841
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
A Phase 2/3 study testing Atumelnant in people with Congenital Adrenal Hyperplasia and Classic Congenital Adrenal Hyperplasia, run by Crinetics Pharmaceuticals Inc. at 51 sites in 13 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
- What is being tested?
- Atumelnant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 153 participants
- Where
- 51 sitesin 13 countries
- Study dates
- 22 Jan 2026to Mar 2030
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
- 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.
Who cannot join
- 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.
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- 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 (including atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose.
- Individuals in Part C who do not meet the Part C Inclusion Criteria.
Trial locations and contacts
51 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
51 sites in 13 countries
United States10 sites, 10 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
Boston's Children's Hospital
Boston, Massachusetts, 02115
- Recruiting
University of Virginia Health System
Charlottesville, Virginia, 22903
- Recruiting
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
- Recruiting
Cook Children's Health Care System
Fort Worth, Texas, 76104
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55454
- Recruiting
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
- Recruiting
Icahn School of Medicine at Mount Sinai-Mount Sinai Hospital
New York, New York, 10029
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
UCSF Ron Conway Gateway Medical Building
San Francisco, California, 94158
France6 sites, 6 recruiting
- Recruiting
Centre Hospitalier Universitaire (CHU) d'Angers
Angers, 49100
- Recruiting
Hopital Kremlin-Bicétre - APHP Paris Saclay
Le Kremlin-Bicêtre, 94270
- Recruiting
Hopital Jeanne de Flandre - CHU de Lille
Lille, 59037
- Recruiting
APHM -Hopital La Timone Enfants
Marseille, 13385 Cedex 5
- Recruiting
Hopital Necker - Enfants Malades
Paris, 75015
- Recruiting
Hopital Robert Debre
Paris, 75019
Argentina5 sites, 5 recruiting
- Recruiting
Instituto de Investigaciones Metabólicas
Buenos Aires, Buenos Aires, C1012AAR
- Recruiting
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
- Recruiting
Instituto Médico Especializado (IME)
Buenos Aires, C1405BCH
- Recruiting
CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez
Buenos Aires, C1425EFD
- Recruiting
Hospital de Niños de la Santísima Trinidad
Córdoba, Córdoba Province, X5000
Australia4 sites, 4 recruiting
- Recruiting
Monash Children's Hospital, Monash Health
Clayton, Victoria, 3168
- Not yet recruiting
Perth Children's Hospital
Nedlands, Western Australia, 6009
- Not yet recruiting
Queensland Children's Hospital
South Brisbane, Queensland, 4101
- Not yet recruiting
Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Belgium4 sites, 4 recruiting
- Recruiting
Cliniques Universitaires Saint-Luc
Brussels, Brussels Capital, 1200
- Recruiting
UZA (Antwerp University Hospital)
Edegem, Antwerp, 2650
- Recruiting
UZ Gent (University Hospital Ghent)
Ghent, East Flanders, 9000
- Recruiting
UZ Leuven (Universitair Ziekenhuis Leuven)
Leuven, Flemish Brabant, 3000
Italy4 sites, 4 recruiting
- Recruiting
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Tuscany, 50139
- Recruiting
IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147
- Recruiting
IRCCS Ospedale San Raffaele
Milan, Lombardy, 20132
- Recruiting
AOU Federico II
Naples, Campania, 80131
United Kingdom4 sites, 4 recruiting
- Recruiting
Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital
Liverpool, L12 2AP
- Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street Hospital
London, WC1N 3JH
- Recruiting
Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital
Manchester, M13 9WL
- Recruiting
Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank
Sheffield, S10 2TH
Brazil3 sites, 3 recruiting
- Recruiting
Nucleo de Pesquisa Clinica do Rio Grande do Sul
Porto Alegre, Rio Grande do Sul, 90430-001
- Recruiting
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
Ribeirão Preto, São Paulo, 14051-140
- Recruiting
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, São Paulo, 05403-000
Germany3 sites, 3 recruiting
- Recruiting
Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie
Berlin, 13353
- Recruiting
Universitatsklinikum Koln Klinik und Poliklinik fur Kinder- und Jugendmedizin
Cologne, North Rhine-Westphalia, 50937
- Recruiting
Universitätsklinikum Tübingen Klinik fur Kinder und Jugendmedizin
Tübingen, Baden, 72076
Poland3 sites, 3 recruiting
- Recruiting
Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku, Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii
Bialystok, Podlaskie Voivodeship, 15-274
- Recruiting
lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych
Lodz, Poland, 93-338
- 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
Chile2 sites, 2 recruiting
- Recruiting
Instituto de Investigaciones Materno Infantil IDIMI, Facultad de Medicina de la Universidad de Chile
Santiago, Santiago Metropolitan, 8360160
- Recruiting
Hospital las Hilgueras de Talcahuano
Talcahuano, 4270918
Saudi Arabia2 sites, 2 recruiting
- Recruiting
King Faisal Specialist Hospital & Research Center
Riyadh, 11211
- Recruiting
King Saud University Medical City (KSU-MC}
Riyadh, 11451
Netherlands1 site, 1 recruiting
- Recruiting
Radboudumc Amalia Children's Hospital, Department of Pediatrics
Nijmegen, Gelderland, 6525 GA
About this study
Official title: A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension
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).
Read the 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.
- Being tested
- Atumelnant, Placebo
- Keywords
- Congenital Adrenal Hyperplasia, CAH, CRN04894, Atumelnant, Pediatric, Balance-CAH
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtumelnantDrug
Also called CRN04894
Atumelnant, tablets, once daily by mouth, weight-based dosing
PlaceboDrug
Placebo, tablets, once daily by mouth, weight-based dosing
| Study group | Role | What they receive |
|---|---|---|
| Treatment (Part A) | Experimental | Open-label, semi-sequential cohorts. Receives: Drug: Atumelnant |
| Active Treatment (Part B) | Experimental | Randomized, Parallel Arms, Double-Blind Receives: Drug: Atumelnant |
| Placebo (Part B) | Placebo comparator | Randomized, Parallel Arms, Double-Blind Receives: Drug: Placebo |
| Open-Label Treatment (Part C) | Experimental | Open-label treatment period for participants entering Part C from Part A and B. Receives: Drug: Atumelnant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in morning serum androstenedione (A4) (Part A)
Measured: Week 8
Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)
Measured: Week 28
Change from baseline in serum early morning A4 over time (Part C)
Measured: Up to Week 260
Secondary outcomes (8)
Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A)
Measured: Week 8
Plasma and/or blood concentrations of atumelnant (Part A)
Measured: Up to Week 8
Change from baseline in serum early morning A4 (Part B)
Measured: Week 4
Change from baseline in serum early morning 17-OHP (Part B)
Measured: Week 4
Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN (Part B)
Measured: Week 28
Change from baseline in serum early morning 17-OHP over time (Part C)
Measured: Up to Week 260
Percent change from baseline in GC daily dose over time (Part C)
Measured: Up to Week 260
Proportion of participants with physiologic GC dose while serum early morning A4 ≤ULN over time (Part C)
Measured: Up to Week 260
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start22 Jan 2026
- Primary completionMar 2030 (estimated)
- Study completionMar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 153 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Crinetics Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07159841
- Sponsor's protocol ID
- CRN04894-13
- Other IDs
- 2024-519578-38-00
- Status last verified
- Jun 2026
- First submitted
- 29 Aug 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No