MEXWILS - Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Mexbrain
- 入组人数
- 1
- 试验地点
- 8
- 主要终点
- Performance of MEX-CD1
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
结局指标
主要结局
Performance of MEX-CD1
时间窗: 4 hours; from treatment start (0 hours) to treatment end (4 hours)
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.
次要结局
- 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.)
- AE recording(From the start of the first MEX-CD1 treatment until the last visit, 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.)
- Number of participants with abnormal laboratory test results(From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.)
- Responder rate(4 hours; from treatment start (0 hours) to treatment end (4 hours))
- Neurologic and psychiatric status evolution(Between the screening visit and the last visit, assessed up to 2 weeks.)
- Hepatic function evolution(Between the screening visit and the last visit, assessed up to 2 weeks.)
- Changes in copper concentration between screening visit and last visit(Between the screening visit and the last visit, assessed up to 2 weeks.)
- Pulse measurement for safety purposes(From the start of the first MEX-CD1 treatment until the last visit, assessed up to 2 weeks.)
- Wilson's Disease evolution(Between the screening visit and the last visit, assessed up to 2 weeks.)
- Copper kinetics(4 hours; from treatment start (0 hours) to treatment end (4 hours))