Clinical trial NCT03017586: Efficacy and Safety of DBS in Patients With Primary Dystonia
Official title: Subthalamic Nucleus (STN) and Globus Pallidus Internus (GPi) Deep Brain Stimulation (DBS) in Patients With Primary Dystonia（RELAX Study）
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Dystonia
Lead sponsor: Beijing Pins Medical Co., Ltd
Sponsor class: INDUSTRY
First posted: 2017-01-11
Enrollment: 72
Sex: ALL
Minimum age (years): 6
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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 exclusion Criteria by researchers

Primary outcomes:
measure: change in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) movement score
timeFrame: 3，6 months
Secondary outcomes:
measure: Short-Form General Health Survey (SF-36)
timeFrame: 3，6 monthsthe Beck Anxiety Inventory31 (with scores ranging from 0 to 63 and higher scores indicating more severe anxiety)
measure: Visual analogue scale
timeFrame: 3，6 months
measure: Measurements of walking
timeFrame: 3，6 months
measure: The Beck Depression Inventory
timeFrame: 3，6 months
measure: Mini-Mental State Examination
timeFrame: 3，6 months
measure: The rate of improvement of BFMDRS score
timeFrame: 3，6 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Union Medical College Hospital
state: Beijing
city: Wuhan
country: China
facility: Union Hospital Tongji Medical College Huazhong University of Science and Technology
state: Hubei
city: Nanjing
country: China
facility: Nanjing Brain Hospital
state: Jiangsu
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Shanghai
country: China
facility: Tongji Hospital of Tongji Univeristy
state: Shanghai
city: Chengdu
country: China
facility: West China Hospital Sichuan University
state: Sichuan
city: Hangzhou
country: China
facility: Sir Run Run Shaw Hospital,School of Medicine,Zhejiang University
state: Zhejiang
Study officials:
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