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

NCT05917327 | Terminated DMC

• 1 other identifier: PJ2104-0003
• Study Type: interventional
• Target: 1
• Locations: 2 countries
• Sites: 4

Brief 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.

Conditions

Hepatolenticular Degeneration; Wilson

Keywords

Wilson's Disease; Low-volume continuous veno-venous hemodialysis; Copper

Outcome Measures

Primary Outcomes (1)

• Performance of MEX-CD1

  The primary objective is to determine the performance of MEX-CD1 in terms of copper extraction in low-volume continuous veno-venous hemodialysis. This will be measured by the mean net amount of copper extracted per unit time relative to baseline, i.e., a proportion.

  4 hours; from treatment start (0 hours) to treatment end (4 hours)

Secondary Outcomes (12)

• Pulse measurement for safety purposes

  From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.

• Temperature measurement for safety purposes

  From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.

• Arterial blood pressure measurement every hour during treatment phase for safety purposes

  Once at screening and last visit and every hour during treatment phase, assessed up to 2 weeks.

• Weight measurement for Safety purposes

  From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.

• AE recording

  From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.

Other Outcomes (2)

• non-ceruloplasmin-bound copper elimination and restoration during the treatment

  4 hours; from treatment start (0 hours) to treatment end (4 hours)

• non-ceruloplasmin-bound copper restoration between treatment

  End of treatment session (n) to beginning of next treatment session (n+1), assessed up to 2 weeks.

Study Arms (1)

MEX-CD1 Low volume CVVHD

EXPERIMENTAL

Patients enrolled in the treatment arm will receive MEX-CD1 treatment depending on the severity of their symptoms in addition to standard of care: * 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

Device: Low-volume continuous veno-venous haemodialysis

Interventions

Low-volume continuous veno-venous haemodialysis DEVICE

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.

MEX-CD1 Low volume CVVHD

Eligibility Criteria

• Age: 10 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females aged between 10 years and 80 years and weighing 30 kg and more
• Established diagnosis of Wilson disease (current Leipzig score ≥ 4). (For patients to whom Leipzig score can't be calculated at time of screening (while waiting for the genetic results), we assume a score of 4 (mutation detected on 2 chromosomes by default) if the two parents are Wilsonian.
• Adequate venous access to allow the setting up of recirculated low-volume continuous veno-venous hemodialysis (dialysis catheter ≥11.5 F, medium blood flow rate 100-200 mL/min) and the collection of blood samples.
• Both the patients already under Standard Of Care (SOC) or not under SOC.
• Patients must present at least one moderate hepatic or Neuropsychiatric symptom(s). (please refer 3.4 for the severity criteria)
• Patient, or parent or guardian in the case of minor, must have been informed about the nature of the clinical investigation, and must have agreed to participate in the clinical investigation, and signed the Informed Consent Form (ICF) prior to participation in any clinical investigation-related activities. Minors under the age of 14 must provide oral consent to participate in the clinical investigation.

You may not qualify if:

• Males and females weighing less than 30 kg
• Patients suffering from copper deficiency
• Patients who are unwilling or unable to comply with clinical investigation procedures
• Seafood allergy and prior allergy to one of the MEX-CD1 product components
• Allergy or contraindication to heparin or citrate
• Inadequate venous access
• Participation in another investigation with an investigational drug or another Medical Device (MD) within 30 days preceding, and during the present investigation
• Pregnant or breastfeeding women according to Article 66 of the Regulations (EU) 2017/745 on Medical Devices

Sponsors & Collaborators

• Mexbrain: lead
• Integrated Scientific Services (ISS) AG: collaborator

Study Sites (4)

Hôpital Femme Mère Enfant, Service des urgences et la réanimation pédiatriques

Bron, Auvergne-Rhône-Alpes, 69500, France

Location

Hôpital Croix Rousse, Service d'hépatologie et gastroentérologie

Lyon, Auvergne-Rhône-Alpes, 69317, France

Location

Hospital Universitario Vall d'Hebron, Unitat de Trasplantament Hepàtic Pediàtric

Barcelona, Catalonia, 08035, Spain

Location

Hospital Clinic Barcelona, Liver ICU

Barcelona, Catalonia, 08036, Spain

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Edouardo COUCHONNAL-BEDOYA

  Hôpital Femme Mère Enfant, Service Hépato-Gastroentérologie et Nutrition Pédiatrique

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Prospective, multinational, multicentric, single-arm, open label, pivotal/registration clinical trial.

Study Record Dates

• First Submitted: April 6, 2023
• First Posted: June 23, 2023
• Study Start: January 24, 2024
• Primary Completion: September 15, 2025
• Study Completion: September 15, 2025
• Last Updated: December 5, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); ES (2)