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

October 7, 2008
October 16, 2008
August 7, 2015
June 2008
February 2015   (Final data collection date for primary outcome measure)
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 ]
The primary outcome measure is the change in Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) movement score from baseline to 6 months. [ Time Frame: 6 months ]
Complete list of historical versions of study NCT00773604 on Archive Site
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Deep Brain Stimulation in Patients With Dystonia
Subthalamic Nucleus (STN) Deep Brain Stimulation (DBS) in Patients With Dystonia
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.
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.
Phase 1
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Device: Medtronic implantable deep brain stimulation (DBS) system
surgical placement of deep brain stimulation system for treatment of dystonia
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Ostrem JL, San Luciano M, Dodenhoff KA, Ziman N, Markun LC, Racine CA, de Hemptinne C, Volz MM, Heath SL, Starr PA. Subthalamic nucleus deep brain stimulation in isolated dystonia: A 3-year follow-up study. Neurology. 2017 Jan 3;88(1):25-35. doi: 10.1212/WNL.0000000000003451. Epub 2016 Nov 30.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
February 2015
February 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
7 Years to 80 Years   (Child, Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
Private donor
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University of California, San Francisco
University of California, San Francisco
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Principal Investigator: Jill Ostrem, M.D. University of California, San Francisco
University of California, San Francisco
August 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP