NCT05917327

Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease

MEXWILS - Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Mexbrain · Industry
• Sex: All
• Age: 10 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to test the MEX-CD1 hemodialysis medical device in patients suffering from Wilson's Disease. The main questions it aims to answer are: * Does the device work as expected by removing the excess of free copper from the blood? * Is the device safe when used according to the instructions for use? Depending on the severity of their symptoms, patients will receive either 5 or 10 treatments on consecutive days with the MEX-CD1 hemodialysis medical device.

Detailed description

This study investigates the performance and safety of the MEX-CD1 hemodialysis device in patients suffering from Wilson's Disease. Wilson's Disease is a rare genetic disease (1'000 to 2'000 patients in France) linked to a problem in copper homeostasis. The direct consequence is a progressive accumulation of copper, first in the liver and then in the whole body with two major implications: (i) at the hepatic level and (ii) at the neurological level. The disease is globally well known and managed in developed countries. It can present itself in several manners: An acute decompensation of the disease is possible. This concerns mainly big children or young adults, presenting themselves with an acute hepatic deficiency that may need intensive care and a liver transplant. In most cases, the clinical picture is one of chronic hepatic and/or neurological disease. Treatment must be adapted to the clinical situation. Two phases can be distinguished: * A primary treatment phase, whose goal it is to eliminate the excess copper deposited in the body. This phase generally takes 1 to 2 years with chelating treatments; * A maintenance phase, corresponding to the treatment which will allow the copper balance to be maintained and equilibrated. This lifelong treatment is to be taken daily (with doses of chelators and/or zinc salts). Finally, during the maintenance phase, periods of lesser observance or escape phases can be observed, those are responsible for severe aggravation of the liver (fulminant hepatitis) or of neurological symptoms that can lead to death. The proposed medical device allows, by combining dialysis to a hyper-chelating colloidal dialysate (MEX-CD1) to specifically extract copper from the blood (and particularly the exchangeable copper). All patients enrolled in this study will, depending on the severity of their symptoms, receive 4-hour long treatments with MEX-CD1: * Patients with moderate liver injury not requiring extracorporeal blood epuration therapies as standard of care: 5 treatments with MEX-CD1 on consecutive days * Patients requiring extracorporeal blood epuration therapies as standard of care: 10 treatments with MEX-CD1 on consecutive days During the MEX-CD1 treatment, the patient's condition will be closely monitored. Additionally, enrolled patients will have a thorough assessment of their Wilson's Disease at the screening visit and at the last visit. Between the last day of treatment and the last visit, enrolled patients will have two rest days.

Conditions

• Hepatolenticular Degeneration; Wilson

Interventions

Type	Name	Description
DEVICE	Low-volume continuous veno-venous haemodialysis	MEX-CD1 is a hyper-chelating colloidal solution that can be added to the dialysate to be used in low-volume continuous veno-venous hemodialysis. One treatment will last 4 hours. For non-hospitalized patients, the treatment is performed on an outpatient basis.

Timeline

• Start date: 2024-01-24
• Primary completion: 2025-09-15
• Completion: 2025-09-15
• First posted: 2023-06-23
• Last updated: 2025-12-05

Locations

4 sites across 2 countries: France, Spain