Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

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

Verified siteRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

11 criteria

  • 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 trial?

12 criteria

  • 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
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

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.

What is already known about the treatment

Cufence (Trientine Dihydrochloride) - An oral medication administered in capsule form that is used for treating Wilson's Disease, a rare genetic disorder affecting copper metabolism in the body. This copper-chelating agent works by binding to excess copper in the body and helping to remove it through urination, effectively reducing copper accumulation in tissues and organs. Trientine dihydrochloride belongs to the pharmacological class of metal chelating agents and represents a second-line treatment option for patients who cannot tolerate or respond to D-penicillamine therapy, with its effectiveness supported by clinical studies showing significant improvement in copper excretion and disease symptoms.

Investigated diseases

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 detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516431-27-00Protocol codeTR-004Estimated enrolment51 patientsSponsorUnivar Solutions B.V.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.