The purpose of this study is to evaluate the effect of a potential new treatment for Congenital Adrenal Hyperplasia (CAH) called atumelnant and compare it to placebo treatment.
CAH is a disease that affects the adrenal glands. The adrenal glands produce important hormones like cortisol. In people with CAH, the body produces too little of the cortisol hormone which results in the body producing an excess of another hormone, adrenocorticotropic hormone, which ultimately causes the effects of CAH. This study aims to determine whether participants receiving atumelnant experience improved quality of life and a reduction in their glucocorticoid use.
Anticipated enrolment close date: 31 October 2026
Who can take part
You may be eligible to participate in this research project if you:
- Are aged 18-75 years of age;
- Have been formally diagnosed with Classic Congenital Adrenal Hyperplasia (CAH); and
- Are able and willing to take part in the study for up to 42 weeks
You may be ineligible to participate in this research project if you:
- Are currently on stable doses of medication for your CAH treatment
- Do not have any history of diabetes treated with insulin, or major cardiovascular disease
What's involved
- This is a randomised, double-blind research project where participants will receive a new medication for the treatment of Congenital Adrenal Hyperplasia in the form of a tablet taken once daily for 32 weeks with an additional 4 week follow up period.
- The visits will involve collection of blood and urine, as well as medical assessment by the study team.
- Other tests will include ECG, blood pressure, weight, eye examination, and surveys.
Ethics
All research in Australia involving human participants is reviewed by an independent group called a Human Research Ethics Committee (HREC). The ethical aspects of this research study have been approved by the Royal Melbourne Hospital HREC.
This study is being carried out according to the National Statement on Ethical Conduct in Human Research (2007). This statement protects the interests of people who agree to participate in human research studies.