Efficacy and Safety of DBS in Patients With Primary Dystonia

Dystonia Clinical Trial

Official title:

Subthalamic Nucleus (STN) and Globus Pallidus Internus (GPi) Deep Brain Stimulation (DBS) in Patients With Primary Dystonia（RELAX Study）

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03017586
• Other study ID #: PINS-023
• Status: Completed
• Phase: N/A
• Start date: December 27, 2017
• Est. completion date: December 17, 2021

Study information

• Verified date: March 2018
• Source: Beijing Pins Medical Co., Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 72
• Est. completion date: December 17, 2021
• Est. primary completion date: September 13, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 60 Years

Eligibility

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

Related Conditions & MeSH terms

• Dystonia
• Dystonic Disorders

Intervention

Device:
• Beijing Pins Medical Co., Ltd. Deep Brain Stimulator
Deep Brain Stimulation for primary dystonia subjects

Locations

Country	Name	City	State
China	Beijing Union Medical College Hospital	Beijing	Beijing
China	West China Hospital Sichuan University	Chengdu	Sichuan
China	Sir Run Run Shaw Hospital,School of Medicine,Zhejiang University	Hangzhou	Zhejiang
China	Qilu Hospital of Shandong University	Jinan	Shandong
China	Nanjing Brain Hospital	Nanjing	Jiangsu
China	Tongji Hospital of Tongji Univeristy	Shanghai	Shanghai
China	Union Hospital Tongji Medical College Huazhong University of Science and Technology	Wuhan	Hubei

Sponsors (1)

Lead Sponsor	Collaborator
Beijing Pins Medical Co., Ltd

Country where clinical trial is conducted

China, 

References & Publications (18)

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Volkmann J, Wolters A, Kupsch A, Muller J, Kuhn AA, Schneider GH, Poewe W, Hering S, Eisner W, Muller JU, Deuschl G, Pinsker MO, Skogseid IM, Roeste GK, Krause M, Tronnier V, Schnitzler A, Voges J, Nikkhah G, Vesper J, Classen J, Naumann M, Benecke R; DBS study group for dystonia. Pallidal deep brain stimulation in patients with primary generalised or segmental dystonia: 5-year follow-up of a randomised trial. Lancet Neurol. 2012 Dec;11(12):1029-38. doi: 10.1016/S1474-4422(12)70257-0. Epub 2012 Nov 1. — View Citation

* Note: There are 18 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	change in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) movement score		3,6 months
Secondary	Short-Form General Health Survey (SF-36)	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.	3,6 monthsthe Beck Anxiety Inventory31 (with scores ranging from 0 to 63 and higher scores indicating more severe anxiety)
Secondary	Visual analogue scale	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	3,6 months
Secondary	Measurements of walking	Measurements of walking (the duration and number of steps taken in a 14-m walk with one turn)	3,6 months
Secondary	The Beck Depression Inventory	The Beck Depression Inventory (with scores ranging from 0 to 63 and higher scores indicating more severe depression)	3,6 months
Secondary	Mini-Mental State Examination	Cognitive were assessed by mini-mental state examination	3,6 months
Secondary	The rate of improvement of BFMDRS score	The rate of improvement of BFMDRS score that >25%?>50%?>75%	3,6 months