Actively Recruiting
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
Led by Crinetics Pharmaceuticals Inc. · Updated on 2026-05-29
153
Participants Needed
33
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating the safety, effectiveness, how the body processes, and the effects of atumelnant treatment in children with classic congenital adrenal hyperplasia (CAH). This Phase 2/3 study includes an open-label extension and involves pediatric participants aged 1 to under 18 years. The study is designed to assess these factors through multiple parts, including initial open-label cohorts, a randomized placebo-controlled phase, and a long-term extension. The study has three parts: Part A includes open-label, semi-sequential cohorts for different age groups with safety reviews before progressing; Part B is a double-blind, randomized, placebo-controlled phase comparing atumelnant to placebo; Part C is an open-label extension where participants from Parts A and B can continue treatment. Atumelnant and placebo are given as once-daily oral tablets with dosing based on weight. Participants will undergo various assessments including measuring morning serum androstenedione and 17-hydroxyprogesterone levels, pharmacokinetic and pharmacodynamic studies, and monitoring glucocorticoid doses. Safety and efficacy are evaluated at multiple time points up to 260 weeks. The study also includes long-term monitoring of hormone levels and glucocorticoid dosing while tracking participant adherence and health status throughout the study duration.
CONDITIONS
Brief Title
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female at birth, aged 1 to under 18 years at consent.
- Medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
- Elevated morning serum androstenedione above the upper limit of normal before morning glucocorticoid dose.
- On stable supraphysiologic glucocorticoid replacement therapy for at least one month before screening.
- Compliance with glucocorticoid and mineralocorticoid replacement regimens as judged by investigator.
- Biochemical euthyroidism as determined by the investigator.
- For Part C, completion of treatment in Part A or B and investigator's opinion that continuing is beneficial regardless of age.
You will not qualify if you...
- Diagnosis of any form of CAH other than classic 21-hydroxylase deficiency.
- Treatment with other glucocorticoids within 30 days before screening.
- Stress dose glucocorticoid therapy within 2 weeks before screening.
- Use of growth hormones within 1 week for short acting or 6 weeks for long acting prior to screening.
- Use of corticotropin-releasing factor receptor antagonists within 14 days before screening.
- History of cancer except treated dermal squamous, basal cell carcinoma, or cervical carcinoma in situ.
- Abnormal sleep/wake cycles as determined by investigator.
- Female participants who are pregnant or breastfeeding.
- Participation in another investigational drug study within 60 days or 5 half-lives before first dose.
- For Part C, individuals not meeting Part C inclusion criteria.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 8 weeks for Part A and up to 28 weeks for Part B
Participants receive atumelnant or placebo tablets once daily by mouth with weight-based dosing to evaluate safety, efficacy, and pharmacokinetics.
Multiple visits during treatment period
Duration - Up to 260 weeks
Participants who completed Parts A or B may continue receiving open-label atumelnant treatment to assess long-term safety and efficacy.
Regular visits throughout extension period
Trial Site Locations
Total: 33 locations
1
Boston's Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
3
University of Minnesota
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
4
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 08901
Actively Recruiting
5
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
6
Cook Children's Health Care System
Fort Worth, Texas, United States, 76104
Actively Recruiting
7
University of Virginia Health System
Charlottesville, Virginia, United States, 22903
Actively Recruiting
8
Instituto de Investigaciones Metabólicas
Buenos Aires, Buenos Aires, Argentina, C1012AAR
Actively Recruiting
9
Hospital de Niños de la Santísima Trinidad
Córdoba, Córdoba Province, Argentina, X5000
Actively Recruiting
10
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina, C1199ABB
Actively Recruiting
11
Instituto Médico Especializado (IME)
Buenos Aires, Argentina, C1405BCH
Actively Recruiting
12
CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez
Buenos Aires, Argentina, C1425EFD
Actively Recruiting
13
Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Not Yet Recruiting
14
Queensland Children's Hospital
South Brisbane, Queensland, Australia, 4101
Not Yet Recruiting
15
Monash Children's Hospital, Monash Health
Clayton, Victoria, Australia, 3168
Actively Recruiting
16
UZA (Antwerp University Hospital)
Edegem, Antwerp, Belgium, 2650
Actively Recruiting
17
UZ Gent (University Hospital Ghent)
Ghent, East Flanders, Belgium, 9000
Actively Recruiting
18
UZ Leuven (Universitair Ziekenhuis Leuven)
Leuven, Flemish Brabant, Belgium, 3000
Actively Recruiting
19
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, São Paulo, Brazil, 05403-000
Actively Recruiting
20
Centre Hospitalier Universitaire (CHU) d'Angers
Angers, France, 49100
Actively Recruiting
21
Hopital Kremlin-Bicétre - APHP Paris Saclay
Le Kremlin-Bicêtre, France, 94270
Actively Recruiting
22
Hopital Jeanne de Flandre - CHU de Lille
Lille, France, 59037
Actively Recruiting
23
APHM -Hopital La Timone Enfants
Marseille, France, 13385 Cedex 5
Actively Recruiting
24
Hopital Necker - Enfants Malades
Paris, France, 75015
Actively Recruiting
25
Hopital Robert Debre
Paris, France, 75019
Actively Recruiting
26
Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie
Berlin, Germany, 13353
Actively Recruiting
27
AOU Federico II
Naples, Campania, Italy, 80131
Actively Recruiting
28
IRCCS Istituto Giannina Gaslini
Genoa, Liguria, Italy, 16147
Actively Recruiting
29
IRCCS Ospedale San Raffaele
Milan, Lombardy, Italy, 20132
Actively Recruiting
30
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Tuscany, Italy, 50139
Actively Recruiting
31
lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych
Lodz, Poland, Poland, 93-338
Actively Recruiting
32
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, Poland, 71-252
Actively Recruiting
33
Sheffield Children's Hospital NHS Trust, Sheffield Children's Hospital, Western Bank
Sheffield, United Kingdom, S10 2TH
Actively Recruiting
Research Team
C
Crinetics Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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