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RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form 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. Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range. 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 Should NOT Join This Trial: 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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. Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range. 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 (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: 10. Individuals in Part C who do not meet the Part C Inclusion Criteria.

Treatments Being Tested

DRUG

Atumelnant

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

DRUG

Placebo

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

Locations (18)

University of Michigan
Ann Arbor, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
University of Virginia Health System
Charlottesville, Virginia, United States
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
Instituto Médico Especializado (IME)
Buenos Aires, Argentina
Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead
Westmead, New South Wales, Australia
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Monash Children's Hospital, Monash Health
Clayton, Victoria, Australia
UZ Leuven (Universitair Ziekenhuis Leuven)
Leuven, Flemish Brabant, Belgium
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, São Paulo, Brazil
Hopital Kremlin-Bicétre - APHP Paris Saclay
Le Kremlin-Bicêtre, France
Hopital Robert Debre
Paris, France
AOU Federico II
Naples, Campania, Italy
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Tuscany, Italy
lnstytut Centrum Zdrowia Matki Polki, Klinika Endokrynologii i Chor6b Metabolicznych
Lodz, Poland, 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, Poland