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New treatment option for Congenital Adrenal Hyperplasia
Official title A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
ClinicalTrials.gov ID: NCT07159841
What this study is testing
What is Atumelnant?
Atumelnant is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for congenital adrenal hyperplasia.
Also referred to as CRN04894.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 17
You may be able to join 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...
- 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.
You likely can't join 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...
- 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.
See the full eligibility criteria
- 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...
- 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 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 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 (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.
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Minneapolis, Minnesota, United States
- New Brunswick, New Jersey, United States
- Philadelphia, Pennsylvania, United States
- Fort Worth, Texas, United States
- Charlottesville, Virginia, United States
- Buenos Aires, Buenos Aires, Argentina
- Córdoba, Córdoba Province, Argentina
- Buenos Aires, Argentina
- Westmead, New South Wales, Australia
- South Brisbane, Queensland, Australia
- Clayton, Victoria, Australia
- Nedlands, Western Australia, Australia
- Edegem, Antwerp, Belgium
- Brussels, Brussels Capital, Belgium
- Ghent, East Flanders, Belgium
- Leuven, Flemish Brabant, Belgium
- São Paulo, São Paulo, Brazil
+ 14 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Boston, Massachusetts, United States; Ann Arbor, Michigan, United States; Minneapolis, Minnesota, United States; New Brunswick, New Jersey, United States; Philadelphia, Pennsylvania, United States and 28 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.