A Multicenter, Phase 2/3, Dose Range Finding Study to Evaluate the Efficacy and Safety of Atumelnant in Adults with ACTH-Dependent Cushing’s Syndrome (ADCS) Including an Open-Label Extension for Long-Term Assessment. (EQUILIBRIUM-ADCS)
Cushing's syndrome happens when your body produces too much cortisol, often called the stress hormone. In ACTH-dependent Cushing's syndrome, this is triggered by high levels of another hormone called ACTH, usually caused by a small, non-cancerous pituitary tumour. High cortisol can cause weight gain, muscle weakness, mood changes, and high blood pressure. This study tests an experimental tablet called atumelnant. Researchers want to see which dose works best and whether it is safe. They will measure whether atumelnant can lower cortisol levels in your urine, blood, and saliva back down to a healthy range by week 12. Some participants may receive a dummy pill (placebo) or different doses of the medicine. The study also offers a longer-term treatment phase and provides backup steroid medicines if cortisol levels drop too low. Taking part may also help show if the medicine improves your quality of life.
At a glance
What is this study about?
Cortisol is a vital hormone that helps control your blood pressure, immune system, and energy levels. However, having too much cortisol over a long period leads to a condition called Cushing's syndrome. In ACTH-dependent Cushing's syndrome (often called Cushing's disease), the pituitary gland in the brain releases too much ACTH hormone, signalling the adrenal glands to make excess cortisol. Current treatments include surgery or existing medicines, but these do not work for everyone and can cause troublesome side effects.
This clinical trial is looking at a new medicine called atumelnant. Atumelnant is a tablet designed to block the signals that cause your body to overproduce cortisol. Researchers are conducting a combined Phase 2 and Phase 3 study, which means they are checking both which dose is most effective and how safe the treatment is compared to a placebo (a dummy pill with no active medicine).
During the first 12 weeks of the trial, doctors will track your cortisol levels through regular blood tests, night-time saliva swabs, and 24-hour urine collections. They will also ask you to fill out questionnaires to see if your day-to-day energy, mood, and overall quality of life improve. After the initial testing period, eligible participants may enter an open-label extension, where everyone receives the active medicine to assess its long-term safety and benefits.
Key takeaways
- The EQUILIBRIUM-ADCS trial tests a new daily tablet called atumelnant for adults with ACTH-dependent Cushing's syndrome.
- The main goal is to see if the medicine can safely bring high cortisol levels down to a healthy range within 12 weeks.
- The study includes safety backup medicines in case cortisol levels drop lower than expected.
- Participants will have regular check-ups, blood tests, saliva tests, and urine collections.
- Taking part is voluntary, and you can withdraw at any point without losing access to standard medical care.
Who may be eligible?
This study is open to adults aged 18 and over who have been diagnosed with ACTH-dependent Cushing's syndrome, including pituitary-dependent Cushing's disease. Men and women of any background can join if they meet the medical criteria.
To take part, your cortisol levels must be high enough to measure before treatment begins, and you must be willing to attend regular hospital appointments. You will also need to be able to swallow tablets and collect saliva and urine samples at home as instructed by the clinic team.
You may not be able to join if you have certain other serious medical conditions, uncontrolled health problems, or if you are taking specific medications that could interfere with the study drug. Your hospital specialist or the study doctor will carry out detailed tests to ensure taking part is safe for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am an adult aged 18 or older.
- I have been diagnosed with ACTH-dependent Cushing's syndrome or Cushing's disease.
- I currently have high cortisol levels that need medical treatment.
- I am able and willing to attend regular hospital check-ups and swallow tablets daily.
- I can collect urine and saliva samples at home following instructions from the clinic team.
What does participation involve?
Taking part in this study involves several stages. First, you will attend a screening visit to check your general health, blood tests, and hormone levels. If you join, you will be assigned to take tablets (either atumelnant at a specific dose or a matching placebo) once a day. You will have regular hospital visits over the first 12 weeks for physical check-ups, morning blood tests, and health questionnaires. You will also collect saliva samples at home late at night and collect your urine over a 24-hour period before visits. If your cortisol drops too low at any point, the study team provides safety steroid medicine (such as hydrocortisone) to restore your balance. After the initial 12-week period, you may have the option to join a longer extension phase where all participants receive atumelnant to see how well it works over time.
Potential risks and benefits
Locations (8)
- —UnverifiedSweden
- —UnverifiedAustria
- —UnverifiedSpain
- —UnverifiedPoland
- —UnverifiedNetherlands
- —UnverifiedItaly
- —UnverifiedGermany
- —UnverifiedFrance
Common questions
What is ACTH-dependent Cushing's syndrome?
It is a form of Cushing's syndrome where excess cortisol is driven by high levels of ACTH, a hormone usually produced by a small tumour in the pituitary gland.
Will I definitely receive the active study medicine?
Not necessarily during the first 12 weeks. Some participants may receive a placebo (dummy pill) so doctors can clearly see if atumelnant works. However, active treatment may be available in the extension phase.
What happens if my cortisol levels drop too quickly?
The study team closely monitors your blood and symptoms. If your cortisol drops too low, backup steroid medicines like hydrocortisone are provided to keep you safe.
Do I have to pay for the study treatment?
No. All study medication, tests, and trial-related hospital visits are provided free of charge, and travel expenses are often reimbursed.
Can I stop taking part if I change my mind?
Yes. You can leave the study at any time and for any reason. Your regular medical team will continue to look after you using standard treatments.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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