<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT01734122</url>
  </required_header>
  <id_info>
    <org_study_id>120632</org_study_id>
    <nct_id>NCT01734122</nct_id>
  </id_info>
  <brief_title>Stereotactic Radiosurgery for Essential Tremor and Parkinsonian Tremor</brief_title>
  <official_title>Stereotactic Radiosurgery for Essential Tremor and Parkinsonian Tremor</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vanderbilt University Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Vanderbilt University Medical Center</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine the changes in quality of life and degree of tremor&#xD;
      for patients with essential tremor or Parkinsonian tremor who are treated by stereotactic&#xD;
      radiosurgery (SRS). This is a questionnaire-based study. Please see Detailed Description&#xD;
      below for more information.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Tremor is the most common of all movement disorders. Patients with essential tremor (ET) or&#xD;
      Parkinsonian tremor (PT) may be severely disabled by their tremor. When the tremor is&#xD;
      inadequately controlled by medications, surgical options are often offered. However, some&#xD;
      patients may prefer a noninvasive treatment approach and some patients are not surgical&#xD;
      candidates for medical reasons.&#xD;
&#xD;
      Stereotactic radiosurgery (SRS) is a treatment that uses high-energy highly-focused radiation&#xD;
      (X-rays) to destroy a tiny region in the brain that causes tremor to occur. This region is&#xD;
      known as the ventral intermedius nucleus of the thalamus and is the same area targeted by&#xD;
      surgical deep brain stimulator (DBS) treatment or neurosurgical operation (thalamotomy).&#xD;
      Multiple case series publications using Gamma Knife stereotactic radiation have been reported&#xD;
      that show stereotactic radiosurgery is safe and effective treatment for ET and PT, making it&#xD;
      a standard treatment for inoperable and inadequately controlled tremor patients. However,&#xD;
      radiation treatment by linear accelerator machines are much more common than Gamma Knife&#xD;
      treatment machines in the United States, and there are no reports of large series of ET and&#xD;
      PT patients treated using linear accelerator-based SRS using a noninvasive mask-based&#xD;
      targeting system.&#xD;
&#xD;
      This clinical trial asks the question of whether linear accelerator-based SRS is as safe and&#xD;
      effective as that reported for Gamma Knife procedures, and whether it is a valid alternative&#xD;
      to surgical thalamotomy or implantation of a deep brain stimulator (DBS) device. The primary&#xD;
      measurements are quality of life related to tremor before and after stereotactic radiosurgery&#xD;
      treatment, and secondary measurements are degree of tremor severity and usage of&#xD;
      tremor-related medications.&#xD;
&#xD;
      Stereotactic Radiosurgery (SRS) is a standard-of-care treatment for patients with severe&#xD;
      tremor. This clinical trial is a questionnaire-based trial that only involves completion of 2&#xD;
      questionnaires at the consultation appointments with neurology and radiation oncology. The&#xD;
      questionnaires only take a few minutes and will provide us with valuable information about&#xD;
      how severe the patient's tremor is before and after SRS treatment at 3-month intervals for 1&#xD;
      year. Currently we are only performing SRS treatment on one side of the brain (effects one&#xD;
      side of the body) so the side with the worst tremor is treated.&#xD;
&#xD;
      Financial: All associated steps listed below are considered the Standard of Care to receive&#xD;
      SRS treatment for tremor and are billed to the patient's insurance per the usual routine.&#xD;
      Patients can receive this treatment without participating in the clinical trial&#xD;
      questionnaires. There is no financial support, no compensation, and no travel stipend&#xD;
      available to patients who participate in this trial.&#xD;
&#xD;
      What to expect:&#xD;
&#xD;
        1. All patients interested in SRS treatment for tremor must be evaluated by a movement&#xD;
           disorder neurologist. The neurologist will assess the severity of the tremor. Your&#xD;
           primary physician or outside neurologist must make a referral to the Vanderbilt Movement&#xD;
           Disorder Clinic for initial assessment. For clinical trial participation, a&#xD;
           questionnaire will be completed as a baseline measurement (this takes a few minutes). If&#xD;
           the patient is an appropriate candidate for SRS and there may be additional evaluations&#xD;
           and discussion with the entire Movement Disorder group at monthly meetings.&#xD;
&#xD;
           ** Please note: for Parkinson's disease patients, please come to the neurology&#xD;
           evaluation &quot;functionally off medication&quot;, which means do NOT take the morning dose of&#xD;
           Parkinson's related medications.&#xD;
&#xD;
        2. The patient will undergo a neuropsychological evaluation prior to SRS treatment, which&#xD;
           is the standard of care for patients considering DBS or SRS treatment.&#xD;
&#xD;
        3. The patient will meet with a neurosurgeon who will be involved in the SRS planning and&#xD;
           procedure.&#xD;
&#xD;
        4. Patients referred for SRS treatment will be evaluated by a radiation oncologist, Dr.&#xD;
           Austin Kirschner or Dr. Anthony Cmelak. At this appointment, discussion will include&#xD;
           descriptions of the procedure in detail and review of the risks and benefits of&#xD;
           treatment. For clinical trial participation, a questionnaire will be completed as a&#xD;
           baseline assessment of the patient's tremor-related quality of life (this takes a few&#xD;
           minutes).&#xD;
&#xD;
        5. If the patient chooses to proceed with SRS, in the department of radiation oncology a&#xD;
           tight-fitting thermal plastic mask will be custom made to fit around the patient's head&#xD;
           and a CT scan of the head will be performed in radiation oncology. This step takes about&#xD;
           45 minutes.&#xD;
&#xD;
        6. A specialized MRI of the brain will be obtained for treatment planning. This step takes&#xD;
           about 1 hour.&#xD;
&#xD;
        7. Approximately 2 weeks after completing all the consultation appointments and obtaining&#xD;
           the imaging for treatment planning, the single stereotactic radiation treatment will be&#xD;
           performed. The SRS procedure takes approximately 2.5 hours. It is entirely outpatient&#xD;
           and does not require anesthesia. The procedure is noninvasive and painless (X-ray&#xD;
           treatment). During the entire treatment, the head is held tightly in the plastic mask.&#xD;
           Oral anti-anxiety medication may be given to relax the patient.&#xD;
&#xD;
           Follow-up:&#xD;
&#xD;
        8. At 3 month intervals for 1 year, the patient will have follow-up appointments with&#xD;
           radiation oncology and neurology. A brain MRI may be performed at some of these&#xD;
           intervals to document the treatment effect on the brain tissue. For participation in the&#xD;
           clinical trial, at these follow-up appointments the quality of life questionnaire will&#xD;
           be completed at the radiation oncology visit and the severity of tremor questionnaire&#xD;
           will be completed at the neurology visit .&#xD;
&#xD;
        9. At 6 months after SRS treatment, a neuropsychological evaluation will be performed.&#xD;
&#xD;
      All patients interested in stereotactic radiosurgery (SRS) or participating in this&#xD;
      questionnaire-based clinical trial should feel free to call the contact phone numbers listed&#xD;
      below or have their physician refer the patient for consultation with the Movement Disorder&#xD;
      Clinic in the neurology department at Vanderbilt.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 21, 2013</start_date>
  <completion_date type="Anticipated">February 2028</completion_date>
  <primary_completion_date type="Anticipated">February 2027</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Tremor-related Quality of Life</measure>
    <time_frame>1 year</time_frame>
    <description>The tremor-related quality of life for each patient will be assessed by self-assessment questionnaire at 3 month intervals for 1 year after treatment by stereotactic radiation.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Severity of Tremor</measure>
    <time_frame>1 year</time_frame>
    <description>The severity of tremor will be assessed by objective measure (Fahn-Tolosa-Marin tremor rating scale) at 3 month intervals for 1 year following the stereotactic radiation treatment.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Neuropsychological Assessment</measure>
    <time_frame>Baseline and 6 months post-treatment</time_frame>
    <description>All patients will undergo a neuropsychological assessment (1-2 hr examination) at baseline and at 6 months post-treatment. This will assess any unanticipated changes in neuropsychological brain function.</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Anticipated">60</enrollment>
  <condition>Tremor</condition>
  <condition>Essential Tremor</condition>
  <condition>Parkinson Disease</condition>
  <condition>Radiosurgery</condition>
  <condition>Quality of Life</condition>
  <arm_group>
    <arm_group_label>Essential Tremor</arm_group_label>
    <description>Patients with severe, medication-refractory Essential Tremor</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Parkinsonian Tremor</arm_group_label>
    <description>Patients with severe, medication-refractory, tremor-dominant Parkinsons</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Questionnaires / Observational Assessments</intervention_name>
    <description>Includes QUEST, PDQ-39, FTM, and Neuropsych evaluation</description>
    <arm_group_label>Essential Tremor</arm_group_label>
    <arm_group_label>Parkinsonian Tremor</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        This trial enrolls persons with essential tremor or Parkinsonian tremor that is&#xD;
        inadequately controlled by medications.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Must be certified by a neurologist as having essential tremor or Parkinsonian tremor&#xD;
             that is insufficiently controlled by medication alone.&#xD;
&#xD;
          -  Preference to receive radiation treatment for tremor treatment, rather than surgical&#xD;
             DBS implantation or surgical thalamotomy&#xD;
&#xD;
          -  At least 18 years old&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Contraindications to cranial radiation (such as prior radiation to the thalamus)&#xD;
&#xD;
          -  Inability to have a MRI of the brain&#xD;
&#xD;
          -  Prior surgical thalamotomy treatment (but contralateral deep brain stimulator [DBS] is&#xD;
             permitted)&#xD;
&#xD;
          -  Estimated life expectancy less than 1 year&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Vanderbilt University Medical Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Send Patient Referrals to Movement Disorder Coordinators</last_name>
      <phone>615-322-0141</phone>
      <phone_ext>Fax 6153436847</phone_ext>
    </contact>
    <contact_backup>
      <last_name>Vanderbilt University Radiation Oncology (nursing staff)</last_name>
      <phone>615-343-2624</phone>
    </contact_backup>
    <investigator>
      <last_name>Austin N. Kirschner, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Anthony J. Cmelak, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Fenna T. Phibbs, MD, MPH</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Elise Bradley, PhD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.vicc.org/ct/</url>
    <description>Find a Clinical Trial</description>
  </link>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>November 20, 2012</study_first_submitted>
  <study_first_submitted_qc>November 20, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 27, 2012</study_first_posted>
  <last_update_submitted>August 20, 2023</last_update_submitted>
  <last_update_submitted_qc>August 20, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 22, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Vanderbilt University Medical Center</investigator_affiliation>
    <investigator_full_name>Austin Kirschner</investigator_full_name>
    <investigator_title>Assistant Professor</investigator_title>
  </responsible_party>
  <keyword>Stereotactic Radiosurgery</keyword>
  <keyword>Essential Tremor</keyword>
  <keyword>Parkinsonian Tremor</keyword>
  <keyword>Quality of Life</keyword>
  <keyword>Severity of Tremor</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Parkinson Disease</mesh_term>
    <mesh_term>Tremor</mesh_term>
    <mesh_term>Essential Tremor</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01734122
org study id: 120632
nct id: NCT01734122
lead sponsor: 
has dmc: No
The purpose of this study is to determine the changes in quality of life and degree of tremor
      for patients with essential tremor or Parkinsonian tremor who are treated by stereotactic
      radiosurgery (SRS). This is a questionnaire-based study. Please see Detailed Description
      below for more information.
Tremor is the most common of all movement disorders. Patients with essential tremor (ET) or
      Parkinsonian tremor (PT) may be severely disabled by their tremor. When the tremor is
      inadequately controlled by medications, surgical options are often offered. However, some
      patients may prefer a noninvasive treatment approach and some patients are not surgical
      candidates for medical reasons.

      Stereotactic radiosurgery (SRS) is a treatment that uses high-energy highly-focused radiation
      (X-rays) to destroy a tiny region in the brain that causes tremor to occur. This region is
      known as the ventral intermedius nucleus of the thalamus and is the same area targeted by
      surgical deep brain stimulator (DBS) treatment or neurosurgical operation (thalamotomy).
      Multiple case series publications using Gamma Knife stereotactic radiation have been reported
      that show stereotactic radiosurgery is safe and effective treatment for ET and PT, making it
      a standard treatment for inoperable and inadequately controlled tremor patients. However,
      radiation treatment by linear accelerator machines are much more common than Gamma Knife
      treatment machines in the United States, and there are no reports of large series of ET and
      PT patients treated using linear accelerator-based SRS using a noninvasive mask-based
      targeting system.

      This clinical trial asks the question of whether linear accelerator-based SRS is as safe and
      effective as that reported for Gamma Knife procedures, and whether it is a valid alternative
      to surgical thalamotomy or implantation of a deep brain stimulator (DBS) device. The primary
      measurements are quality of life related to tremor before and after stereotactic radiosurgery
      treatment, and secondary measurements are degree of tremor severity and usage of
      tremor-related medications.

      Stereotactic Radiosurgery (SRS) is a standard-of-care treatment for patients with severe
      tremor. This clinical trial is a questionnaire-based trial that only involves completion of 2
      questionnaires at the consultation appointments with neurology and radiation oncology. The
      questionnaires only take a few minutes and will provide us with valuable information about
      how severe the patient's tremor is before and after SRS treatment at 3-month intervals for 1
      year. Currently we are only performing SRS treatment on one side of the brain (effects one
      side of the body) so the side with the worst tremor is treated.

      Financial: All associated steps listed below are considered the Standard of Care to receive
      SRS treatment for tremor and are billed to the patient's insurance per the usual routine.
      Patients can receive this treatment without participating in the clinical trial
      questionnaires. There is no financial support, no compensation, and no travel stipend
      available to patients who participate in this trial.

      What to expect:

        1. All patients interested in SRS treatment for tremor must be evaluated by a movement
           disorder neurologist. The neurologist will assess the severity of the tremor. Your
           primary physician or outside neurologist must make a referral to the Vanderbilt Movement
           Disorder Clinic for initial assessment. For clinical trial participation, a
           questionnaire will be completed as a baseline measurement (this takes a few minutes). If
           the patient is an appropriate candidate for SRS and there may be additional evaluations
           and discussion with the entire Movement Disorder group at monthly meetings.

           ** Please note: for Parkinson's disease patients, please come to the neurology
           evaluation "functionally off medication", which means do NOT take the morning dose of
           Parkinson's related medications.

        2. The patient will undergo a neuropsychological evaluation prior to SRS treatment, which
           is the standard of care for patients considering DBS or SRS treatment.

        3. The patient will meet with a neurosurgeon who will be involved in the SRS planning and
           procedure.

        4. Patients referred for SRS treatment will be evaluated by a radiation oncologist, Dr.
           Austin Kirschner or Dr. Anthony Cmelak. At this appointment, discussion will include
           descriptions of the procedure in detail and review of the risks and benefits of
           treatment. For clinical trial participation, a questionnaire will be completed as a
           baseline assessment of the patient's tremor-related quality of life (this takes a few
           minutes).

        5. If the patient chooses to proceed with SRS, in the department of radiation oncology a
           tight-fitting thermal plastic mask will be custom made to fit around the patient's head
           and a CT scan of the head will be performed in radiation oncology. This step takes about
           45 minutes.

        6. A specialized MRI of the brain will be obtained for treatment planning. This step takes
           about 1 hour.

        7. Approximately 2 weeks after completing all the consultation appointments and obtaining
           the imaging for treatment planning, the single stereotactic radiation treatment will be
           performed. The SRS procedure takes approximately 2.5 hours. It is entirely outpatient
           and does not require anesthesia. The procedure is noninvasive and painless (X-ray
           treatment). During the entire treatment, the head is held tightly in the plastic mask.
           Oral anti-anxiety medication may be given to relax the patient.

           Follow-up:

        8. At 3 month intervals for 1 year, the patient will have follow-up appointments with
           radiation oncology and neurology. A brain MRI may be performed at some of these
           intervals to document the treatment effect on the brain tissue. For participation in the
           clinical trial, at these follow-up appointments the quality of life questionnaire will
           be completed at the radiation oncology visit and the severity of tremor questionnaire
           will be completed at the neurology visit .

        9. At 6 months after SRS treatment, a neuropsychological evaluation will be performed.

      All patients interested in stereotactic radiosurgery (SRS) or participating in this
      questionnaire-based clinical trial should feel free to call the contact phone numbers listed
      below or have their physician refer the patient for consultation with the Movement Disorder
      Clinic in the neurology department at Vanderbilt.
observational model: Cohort
time perspective: Prospective
measure: Tremor-related Quality of Life
time frame: 1 year
description: The tremor-related quality of life for each patient will be assessed by self-assessment questionnaire at 3 month intervals for 1 year after treatment by stereotactic radiation.
measure: Severity of Tremor
time frame: 1 year
description: The severity of tremor will be assessed by objective measure (Fahn-Tolosa-Marin tremor rating scale) at 3 month intervals for 1 year following the stereotactic radiation treatment.
measure: Neuropsychological Assessment
time frame: Baseline and 6 months post-treatment
description: All patients will undergo a neuropsychological assessment (1-2 hr examination) at baseline and at 6 months post-treatment. This will assess any unanticipated changes in neuropsychological brain function.
arm group label: Essential Tremor
description: Patients with severe, medication-refractory Essential Tremor
arm group label: Parkinsonian Tremor
description: Patients with severe, medication-refractory, tremor-dominant Parkinsons
intervention type: Other
intervention name: Questionnaires / Observational Assessments
description: Includes QUEST, PDQ-39, FTM, and Neuropsych evaluation
arm group label: Essential Tremor
arm group label: Parkinsonian Tremor
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
investigator: 
investigator: 
investigator: 
country: United States
url: http://www.vicc.org/ct/
description: Find a Clinical Trial
responsible party type: Principal Investigator
investigator affiliation: Vanderbilt University Medical Center
investigator full name: Austin Kirschner
investigator title: Assistant Professor
mesh term: Parkinson Disease
mesh term: Tremor
mesh term: Essential Tremor