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Study of Trientine Dihydrochloride (Cufence) to Evaluate Its Effects and Safety in Patients with Wilson’s Disease

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What is this study about?

This clinical trial focuses on patients with Wilson’s Disease, a genetic condition that causes copper to accumulate in the body’s tissues. The study investigates a medication called Cufence (trientine dihydrochloride), which is taken as hard capsules by mouth. This medicine helps remove excess copper from the body in people with Wilson’s Disease.

The main purpose of this research is to understand how different doses of Cufence affect copper levels in the body and how the medication works in patients with Wilson’s Disease. The study will measure how the drug moves through the body and its effects on various markers of copper levels in blood and urine.

During this 24-month study, participants will take Cufence capsules daily, with a maximum daily dose of 1600 mg. Throughout the study, doctors will monitor copper levels in patients’ blood and urine, and check how well the medication is working. They will also track any changes in liver function and assess participants’ neurological and mental health status to ensure the treatment is safe and effective.

1 Initial visit

You will start taking Cufence (trientine dihydrochloride) in the form of 200 mg hard capsules by mouth

The medical team will perform initial assessments of your condition, including blood tests to measure copper levels and liver function

You will undergo evaluation of your neurological status using a special rating scale

2 Regular monitoring

Your blood will be tested regularly to measure the levels of copper and ceruloplasmin (a copper-binding protein)

You will need to collect 24-hour urine samples to measure copper excretion

Regular assessments of your liver health will be performed using blood tests and Fibroscan (a non-invasive scan)

Your neurological and mental health status will be monitored using various questionnaires

3 Blood sampling

Blood samples will be taken to measure the levels of Cufence and its breakdown products in your body

These measurements help determine how your body processes the medication

4 Safety monitoring

Any side effects or health changes will be recorded throughout the study period

Regular physical examinations and health assessments will be performed

5 Study completion

The study will continue until September 2025

Final assessments will include blood tests, urine collection, and evaluation of your overall health status

Who Can Join the Study?

  • Patient or their representative must provide written informed consent before starting any study procedures
  • Must be male or female aged 5 years or older at the time of consent
  • Must have a confirmed diagnosis of Wilson’s Disease with a Leipzig score of 4 or higher (Leipzig score is a diagnostic scoring system that considers various symptoms and test results)
  • Adult patients (18 years and older) must have previous treatment with D-penicillamine (a medication used to treat Wilson’s Disease)
  • Younger patients (under 18 years) must have previous treatment with either D-penicillamine or zinc
  • Female patients who can become pregnant must:
    • Have a negative pregnancy test at both Screening and Baseline visits
    • Use highly effective birth control (less than 1% failure rate) during the study, such as:
      • Partner who had vasectomy (at least 6 months before)
      • Hormonal contraceptives (oral, patch, injection, or vaginal) used for at least 3 months
      • Implanted or intrauterine devices used for at least 6 months
      • Sexual abstinence
  • Must be able to complete study requirements and attend study visits, as determined by the study doctor

Who Cannot Join the Study?

  • Known hypersensitivity (severe allergic reaction) to Cufence or any of its components
  • Pregnant women or those planning pregnancy during the study period
  • Women who are breastfeeding
  • Patients with severe liver disease (poor liver function)
  • Patients with serious kidney problems (poor kidney function)
  • Patients taking other medications for Wilson’s Disease
  • Patients who have had major surgery within 3 months before the study
  • Patients with uncontrolled psychiatric conditions
  • Patients who cannot follow study procedures or provide reliable information
  • Patients participating in other clinical trials
  • Patients with serious medical conditions that could interfere with study participation
  • Patients with history of substance abuse in the past 6 months

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Fondation A De Rothschild Paris France
Instytut Pomnik Centrum Zdrowia Dziecka Warsaw Poland
Instytut Psychiatrii I Neurologii Warsaw Poland
Centre Hospitalier Lyon Sud Pierre Benite France
Azjqaa Uuvhsjdady Hcwvlrmf Aarhus Denmark
Ugesiruljxpqavgqumruo Mhgyjtiw Afy Munster Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Denmark Denmark
Not recruiting
16.09.2021
France France
Not recruiting
16.09.2021
Germany Germany
Not recruiting
16.09.2021
Poland Poland
Not recruiting
16.09.2021

Trial locations

Investigated drugs:

Cufence (trientine dihydrochloride) is a medication used to treat Wilson’s disease, a rare genetic condition where copper builds up in the body. It works by binding to excess copper in the body and helping to remove it through urine. This medication helps control copper levels in patients who cannot take other treatments or who are intolerant to them. The medication helps maintain healthy copper levels by increasing the amount of copper that is removed from the body through urine.

Wilson’s Disease – A rare genetic disorder that causes copper to accumulate in the liver, brain, and other vital organs. The body cannot properly regulate and remove excess copper, leading to its buildup in tissues. The disease is caused by mutations in the ATP7B gene, which normally helps remove excess copper from the body. As copper accumulates, it can affect liver function and cause neurological symptoms. The condition typically appears between ages 5 and 35, though it can manifest at any age. The disease progresses gradually as copper continues to accumulate in various organs if left unmanaged.

Trial ID:
2024-516431-27-00
Protocol code:
TR-004
Trial Phase:
Therapeutic confirmatory (Phase III)

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