Hepato-lenticular degeneration – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:54 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Hepato-lenticular degeneration – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study comparing different zinc doses for the treatment of Wilson disease in healthy participants. https://clinicaltrials.eu/trial/a-study-comparing-different-zinc-doses-for-the-treatment-of-wilson-disease-in-healthy-participants/ Wed, 29 Apr 2026 15:09:16 +0000 https://clinicaltrials.eu/trial/a-study-comparing-different-zinc-doses-for-the-treatment-of-wilson-disease-in-healthy-participants/ This study focuses on Wilson Disease, a rare condition where too much copper builds up in the body. The purpose of the study is to investigate how different doses of zinc affect the way the body absorbs copper through the intestines. Participants may receive Galzin, a medication used to manage copper levels, or a different dose known as ET-700. Some may also receive a placebo.

To understand how the medications work, a substance called copper (64cu) chloride is used during the study. This is followed by a PET/CT, which is a specialized type of imaging scan that uses a radioactive tracer to create detailed pictures of the body’s internal organs. By using this scan, researchers can measure the amount of copper that reaches the liver to see how much absorption has changed due to the different treatments.

]]> Study of Trientine Dihydrochloride (Cufence) to Evaluate Its Effects and Safety in Patients with Wilson’s Disease https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-trientine-dihydrochloride-in-patients-with-wilsons-disease/ Wed, 29 Apr 2026 14:59:38 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-trientine-dihydrochloride-in-patients-with-wilsons-disease/ 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.

]]> Study on Gene Therapy with VTX-801 and Copper (64Cu) Chloride for Adults with Wilson’s Disease https://clinicaltrials.eu/trial/study-on-gene-therapy-with-vtx-801-and-copper-64cu-chloride-for-adults-with-wilsons-disease/ Wed, 29 Apr 2026 14:30:06 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-with-vtx-801-and-copper-64cu-chloride-for-adults-with-wilsons-disease/ This clinical trial is focused on studying Wilson’s Disease, a rare genetic disorder that affects how the body processes copper. The study involves a new treatment called VTX-801, which is a type of gene therapy. Gene therapy is a method that uses genes to treat or prevent diseases. In this case, VTX-801 is designed to help the body manage copper levels better by using a special viral vector to deliver a shortened form of the human gene ATP7B, which is important for copper regulation.

The purpose of the study is to assess the safety and tolerability of VTX-801 when given to adult patients with Wilson’s Disease. The study will involve a single dose of the treatment, administered through an intravenous infusion, which means it is given directly into a vein. Participants will be monitored over a period of five years to observe how they respond to the treatment and to ensure it is safe. The study will also involve the use of 64Cu, a form of copper, to help track how the body processes copper during the trial.

Throughout the study, various health checks will be conducted, including clinical examinations and tests like ECG (a test that records the electrical activity of the heart) and MRI (a type of scan that uses magnetic fields to create detailed images of the body). These checks will help researchers understand the effects of the treatment on the participants’ health. The study aims to provide valuable information that could lead to better treatment options for people with Wilson’s Disease in the future.

]]> Study on the Safety and Effects of UX701 Gene Therapy for Adults with Wilson Disease https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ux701-gene-therapy-for-adults-with-wilson-disease/ Wed, 29 Apr 2026 14:24:20 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ux701-gene-therapy-for-adults-with-wilson-disease/ This clinical trial is focused on studying Wilson disease, a rare genetic disorder that affects the body’s ability to regulate copper. The study is testing a new treatment called UX701, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a modified version of a gene into the body. In this case, the virus is designed to help correct the copper metabolism in people with Wilson disease by transferring a modified protein known as ATP7B.

The purpose of the study is to evaluate the safety and effects of UX701 in adults with Wilson disease. Participants will receive either the gene therapy or a placebo. The study will be conducted in two stages. In the first stage, the focus will be on determining the safest dose of UX701 and understanding its effects over a period of 52 weeks. The second stage will compare the effects of UX701 with a placebo, particularly looking at how well it helps regulate copper levels in the body.

Throughout the study, participants will undergo regular assessments to monitor their health and the effects of the treatment. This includes checking copper levels in the body and evaluating any changes in their current medication for Wilson disease. The study aims to provide valuable information on the potential benefits and risks of using UX701 as a treatment for Wilson disease.

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