Pilot Study Evaluating Safety of Unilateral Gamma Knife Subthalamotomy for Parkinson's Disease (GKSTN)

This is a pilot study to evaluate the safety and efficacy of unilateral gamma knife subthalamotomy for Parkinson's disease in patients deemed poor candidates for deep brain stimulation.

Study Overview

• Status: Terminated
• Conditions: Parkinsons Disease

Detailed Description

The goal of the study is to enroll 20 subjects who could benefit from unilateral subthalamotomy due to the presence of unilateral rigidity, tremor, bradykinesia and/or dyskinesias (as opposed to axial symptoms such as walking) but who are suboptimal candidates for deep brain stimulation (DBS) because of advanced age (>74), medical comorbidities precluding surgery, or patient aversion to DBS, and who show more than 30% improvement in Unified Parkinson's Disease Rating Scale (UPDRS) score in the ON versus OFF medication state.

Secondary objectives will focus on the efficacy of Gamma Knife (GK) subthalamotomy for Parkinson's disease (PD), as determined by changes in the patient's clinical status and any improvement following the treatment. A full neurologic exam as well as a screen for depression will be administered at every postoperative visit. The patient's neurologic function will be assessed with a full neurological examination and objectively quantified using the UPDRS focusing on the contralateral side, as well as overall UPDRS.

The clinician will complete a Clinical Global Impression (CGI) and Mini-Mental State Examination (MMSE) at each visit to record overall impression of the patient's disease progression and severity, and to evaluate cognitive function. Pre- and post-treatment assessment of the Levodopa Equivalent Daily Dose (LEDD) (quantification of medication regimen in levodopa equivalents) will be obtained and recorded to evaluated the efficacy of gamma knife subthalamotomy (GKS) as a treatment for Parkinson's disease (PD). A Parkinson's Disease Questionnaire-39 (PDQ-39) to determine the patient's independence and quality of life will be filled out at each visit. Finally, the patient will complete a Beck Depression Inventory at each clinic visit to monitor for development or progression of depressive symptoms.

• Study Type: Observational
• Enrollment (Actual): 4

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10016
      • NYU Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 74 years and older (Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Primary care clinic

Description

Inclusion Criteria:

1. Otherwise appropriate DBS candidates but deemed suboptimal for DBS on account of one or more of the following:

   • advanced age greater than 74 years of age

   • medical comorbidities

     • need for continual anticoagulation
     • poor candidates for general anesthesia based on cardiac, hepatic ,renal or pulmonary status
     • high infection risk if internal DBS device hardware is placed (ie. Poor diabetes mellitus control)
   • patient aversion to DBS

2. Patients with asymmetric disease symptoms including:

   • rigidity
   • tremor
   • bradykinesia
   • dyskinesia

3. More than 30% improvement in UPDRS score in ON versus OFF medication state, except:

   • in the presence of disabling contralateral dyskinesias/dystonia limiting dose tolerance
   • tremors that are medically refractory
4. Patients who are on a stable medication regimen and demonstrating stable disease symptoms (absence of significant improvement or deterioration) for at least 3 months at the time of GKS, as determined by review of medical record
5. MMSE score greater than or equal to 26

Exclusion Criteria:

• Patients with prominent axial symptoms including postural instability and gait disturbances, defined as a score of 3 or greater on Questions 28, 29 & 30 on the UPDRS
• Patients whose symptoms do not improve by more than 30% in UPDRS score with medication, unless patient experiences dose-limiting side effects
• The presence of dementia or inability to provide informed consent for participation in the study, as determined by a mini-mental status examination score below 25
• Patients who have had prior brain radiation
• Patients with improvement and satisfactory symptom management will not be offered this procedure. We will only include patients without satisfactory symptom management.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
GK subthalamotomy; Evaluate the safety and efficacy of unilateral GK subthalamotomy for PD in patients deemed poor candidates for DBS.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of performing GK lesions in the Subthalamic Nucleus (STN) for PD patients	Safety of GK subthalamotomy in the management of PD as measured by stable findings on neurologic exam at each clinic visit (hemiballismus, dyskinesias, sensory changes) and the absence of Serious Adverse Events (SAE) attributable to treatment.; Because this study does not have a control group, we will compare the non-inferiority of GK subthalamotomy to published outcomes in PD patients undergoing other forms of management.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of GK subthalamotomy for PD	To be determined by changes in the patient's clinical status and any improvement following the treatment.; Neurologic function as determined by UPDRS, MMSE, neurologic exam results, CGI assessment and LEDD score.; Quality of life, as determined by PDQ-39 score and Beck's Depression Inventory (BDI) score.	12 months

Collaborators and Investigators

• Sponsor: NYU Langone Health
• Investigators: Principal Investigator: Douglas Kondziolka, MD, NYU Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2014
• Primary Completion (Actual): September 1, 2016
• Study Completion (Actual): September 1, 2017

Study Registration Dates

• First Submitted: September 12, 2014
• First Submitted That Met QC Criteria: September 23, 2014
• First Posted (Estimate): September 25, 2014

Study Record Updates

• Last Update Posted (Actual): December 6, 2017
• Last Update Submitted That Met QC Criteria: December 4, 2017
• Last Verified: December 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: 14-00902