Dystonia
Conditions
Brief summary
The purpose of this study is to evaluate the safety and effectiveness of deep brain stimulation (DBS) of the subthalamic nucleus (STN) and globus pallidus internus (GPi) for primary dystonia.
Detailed description
Dystonia is an uncommon brain disorder in which there is abnormal muscle tone producing twisting, writhing movements and abnormal postures. It is associated with abnormal electrical activity in two groups of nerve cells in the brain called the globus pallidus internus (GPi) and the subthalamic nucleus (STN). GPi DBS appears to be effective for medication-refractory focal and segmental dystonia affecting the cranial and cervical regions in open-label series, but recently GPi stimulation has been associated with subtle motor disturbances in previously non-dystonic body regions (i.e., arms and legs) in this population of patients. DBS of the STN has also been reported to be effective for treating generalized and cervical dystonia in small open label trials. STN DBS for cranial and cervical regions may provide similar efficacy in the treatment of dystonia as GPi DBS, but without unwanted stimulation-induced motor effects. Objectives of this study is to evaluate the safety and efficacy of STN-DBS and GPi-DBS for dystonia; Participants will be evaluated pre- and postoperatively with standard dystonia rating scales including the Burke-Fahn-Marsden Dystonia rating scale (BFMDRS), Changes in cognitive function will be assessed with neuropsychological testing. Stimulation parameters will be documented, and a patient questionnaire will be administered postoperatively to determine if patients are experiencing stimulation-induced motor adverse effects. Patient weight will be recorded at study visits. This pilot study will provide preliminary open label efficacy outcomes for STN DBS in the treatment of primary dystonia and will help determine if this target should be compared to GPi DBS in a larger double-blind trial.
Interventions
DEVICEBeijing Pins Medical Co., Ltd. Deep Brain Stimulator
Deep Brain Stimulation for primary dystonia subjects
Sponsors
Beijing Pins Medical Co., Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
1. Refractory primary (systemic, segmental) dystonia diagnosed by a movement disorders neurologist 2. Severe functional impairment despite optimal medical management, including failed botulinum toxin therapy 3. Ability to follow up with post-operative study visits 4. Patients and their relatives have reasonable surgery expectations 5. Volunteer to participate in clinical trials, and signed consent form 6. Age 6-60 years
Exclusion criteria
1. Pregnancy or plan a pregnancy 2. Good treatment with Non-invasive therapy 3. Dopamine reactive dystonia ，Genetic degeneration ，Paroxysmal dystonia ，Secondary dystonia， Psychogenic dystonia 4. Brain MRI showing extensive brain atrophy or small vessel ischemic disease 5. Cognitive impairment(MMSE\<24) 6. Severe depression or other serious mental illness 7. History of traumatic brain injury, tumor, or severe cerebrovascular disease 8. Severe brain atrophy (diagnosed by CT or MRI) 9. Hyperthermia therapy in implant parts 10. Abnormal in blood inspection, blood clotting disorders, liver and kidney dysfunction, or other clinical judgment cannot tolerate surgery 11. High blood pressure, serious heart diseases, or respiratory diseases 12. Diabetes 13. Long-term treatment of immunosuppressive or hormones 14. Implant pacemakers, defibrillators, cochlear and other nerve stimulators 15. Other diseases need frequent MRI examinations 16. Participated in any other clinical trials within 3 months 17. Reluctant or unable to implant surgery 18. Reluctant or unable to cooperate with follow-up 19. Other
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| change in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) movement score | 3，6 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Visual analogue scale | 3，6 months | The severity of dystonia and pain was assessed with the use of a visual analogue scale, with scores ranging from 0 to 10 and higher scores indicating greater severity |
| Measurements of walking | 3，6 months | Measurements of walking (the duration and number of steps taken in a 14-m walk with one turn) |
| Short-Form General Health Survey (SF-36) | 3，6 monthsthe Beck Anxiety Inventory31 (with scores ranging from 0 to 63 and higher scores indicating more severe anxiety) | Quality of life was assessed with the Medical Outcomes Study 36-item Short-Form General Health Survey (SF-36), which evaluates both physical and mental components of functioning on a scale of 0 to 100, with higher scores indicating a higher level of function. |
| Mini-Mental State Examination | 3，6 months | Cognitive were assessed by mini-mental state examination |
| The rate of improvement of BFMDRS score | 3，6 months | The rate of improvement of BFMDRS score that \>25%、\>50%、\>75% |
| The Beck Depression Inventory | 3，6 months | The Beck Depression Inventory (with scores ranging from 0 to 63 and higher scores indicating more severe depression) |
Countries
China
Outcome results
None listed