Deep Brain Stimulation in Patients With Dystonia (STN DBS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00773604
Recruitment Status : Completed
First Posted : October 16, 2008
Last Update Posted : August 7, 2015
Information provided by (Responsible Party):
University of California, San Francisco

Brief Summary:
The purpose of this study is to evaluate the safety and effectiveness of deep brain stimulation (DBS) of the subthalamic nucleus (STN)for primary dystonia.

Condition or disease Intervention/treatment Phase
Dystonia Device: Medtronic implantable deep brain stimulation (DBS) system Phase 1 Phase 2

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). Deep brain stimulation (DBS) has been shown to be an effective treatment in patients with medically refractory dystonia and is currently approved for both the GPi and STN targets under a humanitarian device exemption (HDE) for use in segmental and generalized primary dystonia as well as focal cervical dystonia. 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 are to 1) evaluate the safety and efficacy of STN DBS for dystonia; 2) determine the time course of STN potential efficacy and optimal stimulation parameters; and 3) determine the frequency and severity of stimulation-induced motor adverse effects in previously non-dystonic body regions. Twenty-five patients will be screened, consented, and enrolled in this study. All patients will undergo bilateral STN DBS for dystonia. Participants will be evaluated pre- and postoperatively with standard dystonia rating scales including the Burke-Fahn-Marsden Dystonia rating scale (BFMDRS), Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), the Jankovic Rating Scale (JRS), and Clinical Global Improvement (CGI) rating scale. 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Subthalamic Nucleus (STN) Deep Brain Stimulation (DBS) in Patients With Dystonia
Study Start Date : June 2008
Actual Primary Completion Date : February 2015
Actual Study Completion Date : February 2015

Intervention Details:
  • Device: Medtronic implantable deep brain stimulation (DBS) system
    surgical placement of deep brain stimulation system for treatment of dystonia

Primary Outcome Measures :
  1. The primary outcome measure is the change in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) movement score from baseline to 12 months. [ Time Frame: 36 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Refractory primary dystonia diagnosed by a movement disorders neurologist
  • Severe functional impairment despite optimal medical management, including failed botulinum toxin therapy
  • Age 7-80 years (UCSF patients) and 18-80 (VA patients)

Exclusion Criteria:

  • Patients considered at high risk for elective neurosurgery because of co-morbid conditions
  • Brain MRI showing extensive brain atrophy or small vessel ischemic disease
  • Pregnancy
  • Inability to tolerate awake microelectrode-guided neurosurgery
  • Inability to follow up with post-operative study visits
  • Inability to speak or read English
  • Patients with a score of 4.5 or lower on the BFMDRS movement scale
  • Patients with Secondary dystonia

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00773604

United States, California
San Francisco Veterans Administration Medical Center
San Francisco, California, United States, 94121
University of California, San Francisco
San Francisco, California, United States, 94143
Sponsors and Collaborators
University of California, San Francisco
Principal Investigator: Jill Ostrem, M.D. University of California, San Francisco

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: University of California, San Francisco Identifier: NCT00773604     History of Changes
Other Study ID Numbers: Private donor
First Posted: October 16, 2008    Key Record Dates
Last Update Posted: August 7, 2015
Last Verified: August 2015

Keywords provided by University of California, San Francisco:
Deep brain Stimulation
cervical dystonia
cranial dystonia
generalized dystonia
subthalamic nucleus

Additional relevant MeSH terms:
Dystonic Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases