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

Primary Purpose

Hepatolenticular Degeneration; Wilson

Status
Recruiting
Phase
Not Applicable
Locations
International
Study Type
Interventional
Intervention
Low-volume continuous veno-venous haemodialysis
Sponsored by
Mexbrain
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hepatolenticular Degeneration; Wilson focused on measuring Wilson's Disease, Low-volume continuous veno-venous hemodialysis, Copper

Eligibility Criteria

10 Years - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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. Exclusion Criteria: 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

Sites / Locations

  • Hôpital Femme Mère Enfant, Service des urgences et la réanimation pédiatriques
  • Hôpital Croix Rousse, Service d'hépatologie et gastroentérologie
  • Hospital Universitario Vall d'Hebron, Unitat de Trasplantament Hepàtic Pediàtric
  • Hospital Clinic Barcelona, Liver ICURecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MEX-CD1 Low volume CVVHD

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Pulse measurement for safety purposes
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Temperature measurement for safety purposes
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Arterial blood pressure measurement every hour during treatment phase for safety purposes
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Weight measurement for Safety purposes
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study. Weight measurement before and after each treatment.
AE recording
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Number of participants with abnormal laboratory test results
Safety measurements via blood sample analysis before and after each 4-hour dialysis session
Responder rate
The responder rate is defined as the proportion of patients with >50% of the baseline net amount of exchangeable copper extracted throughout the study.
Copper kinetics
Kinetics of Copper measurement at time 0h, time 1h, time 2h, time 3h and time 4h
Changes in copper concentration between screening visit and last visit
Assessment of the change in non-ceruloplasmin-bound copper (NCC) concentration between the baseline and the patient release
Hepatic function evolution
Assessment of the stability or improvement of hepatic function between the enrolment and the last visit of the patient according to the history of the disease. Hepatic function is assessed through medical imaging, transient elastography (FibroScan or FibroTest), LiverMultiScan™, assessment of presence/absence of jaundice, assessment of presence/absence of haemolysis, ascites detection per sonography.
Neurologic and psychiatric status evolution
Assessment of the stability or improvement of the neurological and psychiatric status between the enrolment and the last visit of the patient. Neurological and psychiatric status is evaluated with UWDRS scores, Clinical Global Impression-Improvement Scale (CGI-S versus CGI-I), MRI. (no units for all the scales)
Wilson's Disease evolution
Assessment of the stability or improvement of the WD by Global Assessment Scale for WD between the enrolment and the last visit of the patient.

Full Information

First Posted
April 6, 2023
Last Updated
July 6, 2023
Sponsor
Mexbrain
Collaborators
Integrated Scientific Services (ISS) AG
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1. Study Identification

Unique Protocol Identification Number
NCT05917327
Brief Title
Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease
Acronym
MEXWILS
Official Title
MEXWILS - Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 1, 2023 (Anticipated)
Primary Completion Date
April 1, 2025 (Anticipated)
Study Completion Date
April 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mexbrain
Collaborators
Integrated Scientific Services (ISS) AG

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hepatolenticular Degeneration; Wilson
Keywords
Wilson's Disease, Low-volume continuous veno-venous hemodialysis, Copper

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
Prospective, multinational, multicentric, single-arm, open label, pivotal/registration clinical trial.
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
MEX-CD1 Low volume CVVHD
Arm Type
Experimental
Arm Description
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
Intervention Type
Device
Intervention Name(s)
Low-volume continuous veno-venous haemodialysis
Intervention Description
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.
Primary Outcome Measure Information:
Title
Performance of MEX-CD1
Description
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.
Time Frame
4 hours; from treatment start (0 hours) to treatment end (4 hours)
Secondary Outcome Measure Information:
Title
Pulse measurement for safety purposes
Description
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Time Frame
From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.
Title
Temperature measurement for safety purposes
Description
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Time Frame
From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.
Title
Arterial blood pressure measurement every hour during treatment phase for safety purposes
Description
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Time Frame
Once at screening and last visit and every hour during treatment phase, assessed up to 2 weeks.
Title
Weight measurement for Safety purposes
Description
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study. Weight measurement before and after each treatment.
Time Frame
From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.
Title
AE recording
Description
Assessment of the safety of the MEX-CD1 medical device in low-volume continuous veno-venous hemodialysis. Safety end point measurements are evaluated throughout the study.
Time Frame
From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.
Title
Number of participants with abnormal laboratory test results
Description
Safety measurements via blood sample analysis before and after each 4-hour dialysis session
Time Frame
From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.
Title
Responder rate
Description
The responder rate is defined as the proportion of patients with >50% of the baseline net amount of exchangeable copper extracted throughout the study.
Time Frame
4 hours; from treatment start (0 hours) to treatment end (4 hours)
Title
Copper kinetics
Description
Kinetics of Copper measurement at time 0h, time 1h, time 2h, time 3h and time 4h
Time Frame
4 hours; from treatment start (0 hours) to treatment end (4 hours)
Title
Changes in copper concentration between screening visit and last visit
Description
Assessment of the change in non-ceruloplasmin-bound copper (NCC) concentration between the baseline and the patient release
Time Frame
Between the screening visit and the last visit, assessed up to 2 weeks.
Title
Hepatic function evolution
Description
Assessment of the stability or improvement of hepatic function between the enrolment and the last visit of the patient according to the history of the disease. Hepatic function is assessed through medical imaging, transient elastography (FibroScan or FibroTest), LiverMultiScan™, assessment of presence/absence of jaundice, assessment of presence/absence of haemolysis, ascites detection per sonography.
Time Frame
Between the screening visit and the last visit, assessed up to 2 weeks.
Title
Neurologic and psychiatric status evolution
Description
Assessment of the stability or improvement of the neurological and psychiatric status between the enrolment and the last visit of the patient. Neurological and psychiatric status is evaluated with UWDRS scores, Clinical Global Impression-Improvement Scale (CGI-S versus CGI-I), MRI. (no units for all the scales)
Time Frame
Between the screening visit and the last visit, assessed up to 2 weeks.
Title
Wilson's Disease evolution
Description
Assessment of the stability or improvement of the WD by Global Assessment Scale for WD between the enrolment and the last visit of the patient.
Time Frame
Between the screening visit and the last visit, assessed up to 2 weeks.
Other Pre-specified Outcome Measures:
Title
non-ceruloplasmin-bound copper elimination and restoration during the treatment
Description
Assessment of the non-ceruloplasmin-bound copper elimination and restoration along the dialysis session.
Time Frame
4 hours; from treatment start (0 hours) to treatment end (4 hours)
Title
non-ceruloplasmin-bound copper restoration between treatment
Description
Assessment of the non-ceruloplasmin-bound copper restoration between dialysis sessions.
Time Frame
End of treatment session (n) to beginning of next treatment session (n+1), assessed up to 2 weeks.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Karen Gillant
Phone
+33 (0) 9 83 00 05 85
Email
karen.gillant@mexbrain.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Edouardo COUCHONNAL-BEDOYA
Organizational Affiliation
Hôpital Femme Mère Enfant, Service Hépato-Gastroentérologie et Nutrition Pédiatrique
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hôpital Femme Mère Enfant, Service des urgences et la réanimation pédiatriques
City
Bron
State/Province
Rhône-Alpes
ZIP/Postal Code
69500
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Etienne Javouhey
First Name & Middle Initial & Last Name & Degree
Etienne Javouhey, MD
First Name & Middle Initial & Last Name & Degree
Aurélie Portefaix, MD
Facility Name
Hôpital Croix Rousse, Service d'hépatologie et gastroentérologie
City
Lyon
State/Province
Rhône-Alpes
ZIP/Postal Code
69317
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Teresa Antonini
First Name & Middle Initial & Last Name & Degree
Teresa Antonini, MD
First Name & Middle Initial & Last Name & Degree
Céline Guichon, MD
Facility Name
Hospital Universitario Vall d'Hebron, Unitat de Trasplantament Hepàtic Pediàtric
City
Barcelona
State/Province
Catalonia
ZIP/Postal Code
08035
Country
Spain
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jesus Quintero
First Name & Middle Initial & Last Name & Degree
Jesus Quintero, MD
Facility Name
Hospital Clinic Barcelona, Liver ICU
City
Barcelona
State/Province
Catalonia
ZIP/Postal Code
08036
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Enric Reverter
First Name & Middle Initial & Last Name & Degree
Enric Reverter, MD
First Name & Middle Initial & Last Name & Degree
Zoé Mariño, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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

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