WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)
France5 participantsStarted 2016-01
Plain-language summary
Dystonia in Wilson's disease represent a major issue. The persistence of disabling motor symptoms despite medical treatments justifies conducting a study on deep brain stimulation (DBS) in Wilson's disease (WD). For bradykinetic patients, subthalamic nucleus (STN) could be considered as a better target than the globus pallidus (GPi). For patients with hyperkinetic dystonia, the internal globus pallidus (GPi) will be chosen as the target of DBS.
The investigators hypothesize that STN DBS will improve Wilson's disease patients, who, despite copper chelators drugs, are still impaired by severe dystonia and akinesia (more or less associated with other movement disorders).
The investigators primary objective is to demonstrate the efficacy of STN/GPi DBS on dystonia associated with Wilson's disease.
Secondary objectives:
* To evaluate the impact of STN/GPi DBS on other movements disorders (tremor, Parkinsonism, chorea) observed in Wilson's disease.
* To describe cognitive status of patients and to evaluate the consequences of STN/GPi DBS on cognition and behavioral aspects of the disease.
* To evaluate the consequences of the stimulation on speech and swallowing.
* To evaluate the social impact of STN/GPi DBS in Wilson's disease.
* To evaluate the safety of STN/GPi DBS in the specific context of Wilson's disease.
Who can participate
Age range
18 Years – 60 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Age \> 18 and \< 60 years.
* Severe neurological form of Wilson's disease with predominant dystonia and akinetic-rigid syndrome, despite optimized treatment stabilized for at least 6 months.
* Important disability due to abnormal movements (Rankin score=2 to 4).
* Absence of dementia (MMS \> 24 and BREF \> 15).
* Stable psychiatric status and absence of severe depression (BDI \<28).
* Social security coverage.
* Signature of informed consent. (signature of legal guardian for subjects under protection)
Exclusion Criteria:
* Severe hepatopathy with coagulation disorders (Platelet count \< 100 G / l; INR \> 1.5; V factor deficit; low level of fibrinogen \< 1g/dL; increased of fibrin degradation products; low level of antithrombin).
* Liver transplanted patients \< 2 years
* Patients under immunosupressive drugs and corticoids regimen.
* Participation to another biomedical research involving any drugs.
* Severe and uncontrolled psychosis or depression.
* Major atrophy on brain MRI that could represent a problem for leads implantation.
* Necrosis of the STN/GPi on brain MRI.
* Female subjects who are pregnant or lactating.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in movement disorder evaluated by the Canadian Occupational Performance Measure (COPM) performance and satisfaction scores