Ongoing, recruitingTherapeutic use (Phase IV)Interventional

Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients

This research is focused on Cufence, a medicine used for Wilson's Disease. The main goals are to understand how the body absorbs, uses, and gets rid of Cufence, and to see how effective and safe it is for patients. We'll be looking closely at levels of the medicine in the blood, as well as several markers for copper levels, which are important in Wilson's Disease. We'll also be monitoring for any side effects and how the medicine affects symptoms of the condition, including liver, brain, and mental health changes. This study aims to provide more information about how Cufence works in a real-world setting.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic use (Phase IV)

Sponsor

Univar Solutions B.V.

Enrolment target

Start

11 Sep 2024

Wilson’s Disease

What is this study about?

This study is about a medicine called Cufence, which is already used to treat Wilson's Disease. Wilson's Disease is a rare genetic condition where too much copper builds up in the body, which can damage organs like the liver, brain, and eyes. Because Cufence is already approved and in use, this study is a 'Phase IV' study. This means we're looking to gather more information about how it works in everyday clinical practice, rather than testing if it's safe and effective for the very first time.

The main aim is to understand how your body handles Cufence. This includes how much of the medicine gets into your blood, how long it stays there, and how your body processes it. We'll also be checking various copper levels in your body, as these are key to monitoring Wilson's Disease. These checks will help us see how well Cufence is controlling the copper build-up. We'll also be keeping a close eye on any side effects you might experience.

Beyond understanding the medicine's behaviour and safety, the study will also look at how Cufence affects your health. This includes assessing any changes in your liver health, how your thinking and memory might be, and any changes in your mood or mental well-being. By collecting all this information, doctors can get a better picture of how Cufence helps people with Wilson's Disease in their daily lives.

Key takeaways

The study investigates Cufence, a medicine for Wilson's Disease.
It aims to understand how the body handles Cufence and its effectiveness.
Safety and side effects will be closely monitored.
It's for adults (18 years and older) with Wilson's Disease, both men and women.
Participation involves taking Cufence and attending regular check-ups and tests.

Who may be eligible?

To be able to take part in this study, you need to have Wilson's Disease. There's no upper age limit, meaning that adults of any age are welcome to participate, as long as they are at least 18 years old.

Both men and women can join this study. If you think you might be eligible, it's always best to discuss it with your doctor or the study 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.

Are you 18 years old or older?
Do you have Wilson's Disease?
Are you able to attend regular clinic visits?
Are you willing to take Cufence capsules as part of the study?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be taking Cufence 200 mg capsules. You'll have regular visits to the study clinic, where doctors and nurses will check your health. They will take blood samples to measure the levels of Cufence in your body and to check your copper levels and liver function. You might also have urine tests to check copper levels.

There will be assessments to check your brain function, memory, and mood, using various questionnaires and tests. Your liver health might also be monitored using scans like Fibroscan, and other blood tests. The study will continue for a certain period, and you'll have follow-up visits after you finish taking the study medicine to make sure you're well. The total duration of your participation will be explained to you by the study team.

Potential risks and benefits

Taking part in this study might offer a better understanding of how Cufence works for you, and your health will be closely monitored by medical professionals. This extra care could be a benefit. However, like all medicines, Cufence can have side effects, and some of the tests, like blood draws, carry small risks, such as bruising. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

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

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Unverified
Germany
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Unverified
France
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Unverified
Denmark
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Unverified
Poland

Common questions

What is Wilson's Disease?

Wilson's Disease is a rare inherited condition where too much copper builds up in the body, which can cause damage to organs like the liver and brain.

What is Cufence?

Cufence is a medicine that helps remove the extra copper from the body in people with Wilson's Disease.

Why is this study being done?

This study aims to learn more about how Cufence works in people with Wilson's Disease, including how the body uses it and how safe and effective it is in a real-world setting.

Will I get Cufence if I join?

Yes, if you join this study, you will be given Cufence 200 mg capsules as part of the research.

Can I stop participating in the study at any time?

Yes, you have the right to leave the study at any point, and your decision will not affect your future medical care.