Clinical trial NCT02583074: Clinical Trial of STN-DBS for Primary Cranial-Cervical Dystonia
Official title: Subthalamic Deep Brain Stimulation in Patients With Medication-Refractory Primary Cranial-Cervical Dystonia: A Randomised, Sham-controlled Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Dystonia
Keywords: deep brain stimulation
Subthalamic Nucleus
dystonia
Lead sponsor: Beijing Tiantan Hospital
Sponsor class: OTHER
Collaborators: name: Peking Union Medical College Hospital
name: The First Affiliated Hospital of Dalian Medical University
name: China-Japan Friendship Hospital
First posted: 2015-10-21
Enrollment: 40
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Primary cranial-cervical dystonia is managed mainly by repeated botulinum toxin injections.This study is to establish whether subthalamic nucleus neurostimulation could improve symptoms in patients not adequately responding to chemodenervation or oral drug treatment. Investigators compared this surgical treatment with sham stimulation in a randomized, controlled clinical trial.

Detailed description: In this randomised, sham-controlled trial, investigators will recruit forty patients with primary cranial-cervical dystonia to receive an implanted device for STN-DBS, and participants will be randomly assigned to receive either neurostimulation or sham stimulation for 3 months.The primary end point was the change from baseline to 3 months in the severity of symptoms, according to the Burke-Fahn-Marsden Dystonia Rating Scale. Two masked dystonia experts who unaware of treatment status will assess the severity of dystonia by reviewing standardised videos.Subsequently, all patients will receive open-label neurostimulation; blinded assessment will be repeated after 6 months of active treatment.

Eligibility criteria: Inclusion Criteria:

1. Clinical diagnosis of primary cranial-cervical dystonia: blepharospasm combined with dystonia of the lower facial muscles. Cervical muscles can be involved but did not dominate the clinical picture.
2. Medical intractable：including various drugs and local botulinum toxin injections.
3. patients deteriorated in activities of daily living and showed social withdrawal during the course of their disease as they suffered from apparent hyperkinesia of facial muscles and functional blindness secondary to severe blepharospasm.

Exclusion Criteria:

1. All other topographical areas remained unaffected.
2. Secondary causes will be excluded by standard cranial magnetic resonance imaging (MRI) scans.
3. Cognitive impairment, dementia,severe psychiatric diseases as well as higher surgical risks due to comorbidities will also be exclusion criteria.

Primary outcomes:
measure: Change of motor and disability scores
timeFrame: Baseline,3 months after surgery, 6 months after active stimulation
Secondary outcomes:
measure: Quality of life
timeFrame: Baseline,3 months after surgery,6 months after active stimulation
measure: Depression
timeFrame: Baseline,3 months after surgery,6 months after active stimulation
Other outcomes:
measure: Adverse event
timeFrame: 3 months after surgery,6 months after active stimulation
Locations:
city: Beijing
country: China
facility: Beijing Tiantan Hospital Affiliated to Capital Medical University
state: Beijing
city: Beijing
country: China
facility: Beijing Tiantan Hospital Affiliated to Capital Medical University
Study officials:
affiliation: Beijing Tiantan Hospital
name: Zhang Jian-guo, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Skogseid IM. Pallidal deep brain stimulation is effective, and improves quality of life in primary segmental and generalized dystonia. Acta Neurol Scand Suppl. 2008;188:51-5. doi: 10.1111/j.1600-0404.2008.01032.x.
citation: Limotai N, Go C, Oyama G, Hwynn N, Zesiewicz T, Foote K, Bhidayasiri R, Malaty I, Zeilman P, Rodriguez R, Okun MS. Mixed results for GPi-DBS in the treatment of cranio-facial and cranio-cervical dystonia symptoms. J Neurol. 2011 Nov;258(11):2069-74. doi: 10.1007/s00415-011-6075-0. Epub 2011 May 7.
citation: Reese R, Gruber D, Schoenecker T, Bazner H, Blahak C, Capelle HH, Falk D, Herzog J, Pinsker MO, Schneider GH, Schrader C, Deuschl G, Mehdorn HM, Kupsch A, Volkmann J, Krauss JK. Long-term clinical outcome in meige syndrome treated with internal pallidum deep brain stimulation. Mov Disord. 2011 Mar;26(4):691-8. doi: 10.1002/mds.23549. Epub 2011 Feb 10.
citation: Ostrem JL, Markun LC, Glass GA, Racine CA, Volz MM, Heath SL, de Hemptinne C, Starr PA. Effect of frequency on subthalamic nucleus deep brain stimulation in primary dystonia. Parkinsonism Relat Disord. 2014 Apr;20(4):432-8. doi: 10.1016/j.parkreldis.2013.12.012. Epub 2014 Jan 5.
citation: Cao C, Pan Y, Li D, Zhan S, Zhang J, Sun B. Subthalamus deep brain stimulation for primary dystonia patients: a long-term follow-up study. Mov Disord. 2013 Nov;28(13):1877-82. doi: 10.1002/mds.25586. Epub 2013 Jul 16.