Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A phase II, multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease

This important study is investigating a medication called Isturisa for children and teenagers who have Cushing's disease. The main goal is to understand how the body handles this medicine (how it's absorbed, used, and removed), what effects it has, and to check if it's safe and well-tolerated. Researchers will be giving different strengths of Isturisa tablets. They will also be looking to see if the medicine helps keep urine cortisol levels (a marker for Cushing's) normal and how these levels change over time. Your safety and comfort throughout the study are very important to the research team.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Recordati AG

Enrolment target

Start

31 Oct 2024

Cushing's disease

What is this study about?

This study is about a medicine called Isturisa, which is already used for adults with Cushing's disease. Now, researchers want to see if it can help children and teenagers who have this condition. Cushing's disease is a health problem where your body makes too much of a hormone called cortisol, which can lead to various health issues.

The study aims to understand how the body processes Isturisa in younger patients – how much medicine gets into the bloodstream, how long it stays there, and how it leaves the body. They also want to see what effects the medicine has on the body and, most importantly, to check if it's safe and if patients can tolerate taking it without too many side effects. This is a "Phase II" study, which means it's an early stage but very important step in testing new treatments.

Finding safe and effective treatments for children and teenagers with Cushing's disease is really important. If this study shows that Isturisa is well-tolerated and works for younger patients, it could potentially offer a new treatment option in the future. The results from this research will help doctors understand the best way to use Isturisa, including the right dose, to help children and teenagers manage their Cushing's disease.

Key takeaways

This study evaluates Isturisa for children and teenagers with Cushing's disease.
It aims to understand how the body uses the medicine and check its safety.
Patients will receive Isturisa tablets and have regular health checks.
Participation involves clinic visits and taking the study medication.
You can withdraw from the study at any time.

Who may be eligible?

This study is looking for children and teenagers who have Cushing's disease. Unlike some studies that have strict age limits, this one says "any to any," meaning they are open to considering patients across a wide age range, as long as they are children or teenagers with Cushing's disease.

Both boys and girls can take part in this study. The most important thing is that you have been diagnosed with Cushing's disease and meet the specific health requirements set out by the research team.

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.

Do you have Cushing's disease?
Are you a child or teenager?
Are you able to take tablets by mouth?
Are you able to attend regular clinic appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be given Isturisa tablets at different strengths (1mg, 5mg, or 10mg). You and your doctors would decide the best starting dose. You would have regular visits to the clinic where doctors would check your health, take blood and urine samples, and ask you about how you are feeling and any side effects you might be having. These checks help the medical team understand how the medicine is working and ensure you are safe. The total length of time you would be involved in the study would depend on how your body responds to the medicine and what the study doctors think is best for you, but it would involve several months of regular checks and taking the medication.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit of receiving a medicine that could help manage your Cushing's disease. However, like all medicines, Isturisa can have side effects, and some might be unpleasant. The study team will carefully monitor you for any side effects and will explain these risks in detail. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
France
—
Unverified
Belgium
—
Unverified
Italy

Common questions

What is Cushing's disease?

Cushing's disease is a condition where your body makes too much of a hormone called cortisol, which can cause various health problems.

What is Isturisa?

Isturisa is a medicine designed to help lower the amount of cortisol your body makes. It's currently used for adults, and this study is testing it for children and teenagers.

Will I get fake medicine (a placebo)?

No, this is an 'open-label' study, which means everyone taking part will receive Isturisa. There won't be a placebo group.

How often will I need to visit the clinic?

You will need to attend regular clinic visits throughout the study. The exact schedule will be explained by the study team.

Can I stop taking part if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your medical care.