CUSHMAH - Benefit of steroidogenesis inhibitors in Mild Cushing syndrome (Mild Autonomous Cortisol Secretion): a randomized trial in patients with Primary Bilateral Macronodular Adrenocortical Hyperplasia
This research, called CUSHMAH, is investigating a new treatment for a specific type of mild Cushing's syndrome where both adrenal glands have benign growths. These growths cause the body to make too much of the stress hormone, cortisol. The study aims to see if a medication called METYRAPONE ESTEVE 250 mg can help improve health problems often linked to this condition, such as high blood pressure and diabetes. Participants will either receive the active medication or a placebo (a dummy pill with no active ingredient). This is a Phase III study, which means it's one of the final steps before a treatment might become more widely available. Researchers will monitor changes in blood pressure, diabetes, and other health markers over six months to understand how effective the treatment is.
At a glance
What is this study about?
This study is called CUSHMAH, and it's looking into a health condition where your body produces too much cortisol, often called the 'stress hormone'. Specifically, it focuses on people who have a mild form of Cushing's syndrome. This type of Cushing's occurs when both of your small adrenal glands, which sit on top of your kidneys, develop growths. These growths, called 'macronodules', aren't cancerous but cause the glands to work overtime, leading to health issues like high blood pressure and diabetes.
The main goal of this research is to test a medication called METYRAPONE ESTEVE 250 mg. The study wants to find out if this medication can help improve these common health problems – namely, blood pressure control and diabetes – in people with this condition. To do this, some participants will receive the active medication, while others will receive a placebo, which looks identical but has no active drug inside. This is a common and important way to fairly compare the effects of a new treatment.
This is a 'Phase III' study, which means it's a very important stage of research. It's designed to confirm if the new treatment is safe and effective in a larger group of people before it can potentially be approved for wider use. Researchers will carefully monitor participants for six months, looking at various health markers, including blood pressure, blood sugar, and overall well-being. They will also look at how hormone levels change and assess quality of life.
Key takeaways
- This study is testing METYRAPONE ESTEVE for mild Cushing's syndrome.
- It aims to see if the drug improves blood pressure and diabetes.
- Half of participants will get the drug, half will get a dummy pill (placebo).
- The study lasts for 6 months with regular health checks.
- It's open to adults aged 18 and over with the specific condition.
- Participation could help improve understanding of this condition.
Who may be eligible?
To be considered for this study, you need to be an adult, 18 years old or older. The study is open to both men and women.
The main requirement is that you have a specific condition called "Primary Bilateral Macronodular Adrenocortical Hyperplasia" which is causing a moderate form of Cushing Syndrome, often referred to as "Mild Autonomous Cortisol Secretion."
This means that both of your adrenal glands have harmless growths, and these growths are causing your body to make too much cortisol, but not to an extreme level. Your doctor will be able to tell you if your specific health condition fits these requirements.
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.
- Are you 18 years old or older?
- Has your doctor told you that you have Primary Bilateral Macronodular Adrenocortical Hyperplasia?
- Are you experiencing a 'mild' form of Cushing's syndrome (Mild Autonomous Cortisol Secretion)?
- Are you currently dealing with high blood pressure and/or diabetes?
What does participation involve?
If you decide to take part in this study, you will first have some tests to make sure you meet all the requirements. Once confirmed, you will be randomly assigned to either receive the study medication (METYRAPONE ESTEVE 250 mg) or a placebo (a dummy pill).
You will take the assigned medication as instructed for a total of six months. Throughout these six months, you will have several scheduled visits to the clinic or hospital. During these visits, the study team will conduct various assessments. These may include blood and urine tests to check your hormone levels and other health markers, measurements of your blood pressure and weight, and possibly questionnaires to assess your quality of life. The doctors will also closely monitor how your diabetes may be affected. The total duration of the active treatment and monitoring period for this study is six months.
Potential risks and benefits
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Common questions
What is Cushing's syndrome?
It's a condition where your body makes too much of a hormone called cortisol, which can cause various health problems.
What is a placebo?
A placebo is a 'dummy pill' that looks like the real medication but has no active ingredient. It helps researchers fairly compare treatments.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'blinded' study, meaning neither you nor your study doctor will know which treatment you are receiving until after the study is over.
How long will I be in the study?
You'll be in the study for six months, taking the medication and having regular check-ups.
Can I leave the study at any time?
Yes, you are free to leave the study at any time, and it won't affect your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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