<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/NCT03789786</url>
  </required_header>
  <id_info>
    <org_study_id>STUDY00002353</org_study_id>
    <nct_id>NCT03789786</nct_id>
  </id_info>
  <brief_title>Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy</brief_title>
  <official_title>Long-term Outcomes of Selective Dorsal Rhizotomy Among Individuals With Cerebral Palsy Compared to a Matched Control Group</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Gillette Children's Specialty Healthcare</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Shriners Hospitals for Children</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Gillette Children's Specialty Healthcare</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal&#xD;
      rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity&#xD;
      in order to prevent further morbidities. Better understanding of the long-term outcomes of&#xD;
      SDR is essential for clinicians and families. The results of this study will have direct&#xD;
      clinical impact by equipping providers with the necessary information to counsel families&#xD;
      during medical decision making.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Excessive&#xD;
      spasticity is thought to be uncomfortable, reduce function, cause gait deviations (e.g.&#xD;
      equinus), and contribute toward musculoskeletal deformity and an elevated energy cost while&#xD;
      walking. As such, SDR is a surgical method used by some hospitals to permanently reduce&#xD;
      spasticity in order to prevent the aforementioned morbidities.&#xD;
&#xD;
      Treatment philosophies differ widely in regards to how aggressively to manage spasticity.&#xD;
      Some centers (e.g. Gillette) aggressively treat spasticity early in life through a variety of&#xD;
      measures such as SDR, intrathecal baclofen pumps, and botulinum toxin injections. Other&#xD;
      centers (e.g. Shriners Hospitals for Children - Salt Lake City and Spokane) offer little in&#xD;
      the way of spasticity reduction treatments.&#xD;
&#xD;
      There are several compelling reasons to conduct the proposed research study. First, emerging&#xD;
      evidence suggests that the elimination of spasticity during childhood via SDR does not&#xD;
      prevent contractures and only partially explains poor gross motor function, both previously&#xD;
      thought to be clear outcomes of the surgery. Additionally, many of the longitudinal cohort&#xD;
      studies that examined SDR outcomes have shown many outcome measures peak 1-3 years after&#xD;
      surgery, and then decline toward baseline (i.e. pre-SDR) levels. Lastly, the quality of the&#xD;
      SDR outcome literature is poor. Rarely are outcomes looked at in context of a proper control&#xD;
      group. Either a control group is absent or comprised of typically developing children. This&#xD;
      limits our ability to understand how patients with cerebral palsy may age without undergoing&#xD;
      an SDR.&#xD;
&#xD;
      Better understanding of the long-term outcomes of SDR is essential for clinicians and&#xD;
      families. The surgery, in general, is costly to families (time, expense, risk, etc.) and&#xD;
      clinicians should have every confidence in the intended outcomes for any intervention.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 1, 2018</start_date>
  <completion_date type="Actual">January 31, 2020</completion_date>
  <primary_completion_date type="Actual">January 31, 2020</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Other</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Three-dimensional gait and motion analysis</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare three-dimensional gait kinematics and kinetics across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Energy expenditure</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare energy expenditure across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Spasticity</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare spasticity, measured by Modified Ashworth Score (0 no increase in tone - 4 rigid in flexion or extension), across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Passive range of motion</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare passive range of motion, measured by lower extremity physical exam, across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Selective motor control</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare selective motor control (0 patterned movement - 2 complete isolated movement) across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Lower extremity strength</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare lower extremity strength, measured by the manual muscle test, across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Gross Motor Function Measure (GMFM-66)</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess function using portions of the GMFM-66 (0 low function - 100 high function) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Gillette Functional Assessment Questionnaire (Gillette FAQ)</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess function and activity using the Gillette FAQ (self-reported survey, 0 low function - 10 high function) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Functional Mobility Scale (FMS)</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess function and activity using the FMS (self-reported survey, 1 uses wheelchair - 6 independent) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Participation Enfranchisement survey</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess participation using the Participation Enfranchisement survey (self-reported survey, true/false) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Diener Satisfaction with Life Scale</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess satisfaction using the Diener Satisfaction with Life Scale (self-reported survey, 5 dissatisfied- 35 satisfied) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>World Health Organization (WHO) Quality of Life Scale</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess satisfaction using the WHO Quality of Life Scale (self-reported survey, 0 low quality of life - 100 high quality of life) and compare across groups</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Multiple Sclerosis Spasticity Scale (MSSS-88)</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Assess pain using portions of the MSSS-88 (self-reported survey, 21 not at all bothered - 84 extremely bothered) and compare across groups</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in gait and motion analysis</measure>
    <time_frame>Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Compare change in gait kinematics and kinetics within groups and if the change is similar between groups</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of prior surgery and anti-spastic treatments</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Incidence of prior surgery and anti-spastic treatments</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cost of prior surgery and anti-spastic treatments</measure>
    <time_frame>Time of long-term follow-up research visit (on average 10 years post-baseline)</time_frame>
    <description>Cost of prior surgery and anti-spastic treatments</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">78</enrollment>
  <condition>Cerebral Palsy</condition>
  <arm_group>
    <arm_group_label>Cases (+SDR)</arm_group_label>
    <description>Patients with cerebral palsy that underwent an SDR</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Controls (-SDR)</arm_group_label>
    <description>Matched patients with cerebral palsy but did not undergo an SDR</description>
  </arm_group>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Gait and Motion Analysis</intervention_name>
    <description>Gait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.)</description>
    <arm_group_label>Cases (+SDR)</arm_group_label>
    <arm_group_label>Controls (-SDR)</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Gross Motor Function Measure (GMFM)</intervention_name>
    <description>Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping</description>
    <arm_group_label>Cases (+SDR)</arm_group_label>
    <arm_group_label>Controls (-SDR)</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Behavioral</intervention_type>
    <intervention_name>Surveys</intervention_name>
    <description>Six surveys are used to assess function, activity, participation, pain, quality of life, and treatment history</description>
    <arm_group_label>Cases (+SDR)</arm_group_label>
    <arm_group_label>Controls (-SDR)</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Former/current patients at Gillette Children's Specialty Healthcare, Shriners Hospitals for&#xD;
        Children - Salt Lake City, or Shriners Hospitals for Children - Spokane&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria for Controls (-SDR) and Cases (+SDR):&#xD;
&#xD;
          -  Able to speak and read English&#xD;
&#xD;
          -  Diagnosed with bilateral cerebral palsy (i.e. no hemiplegics)&#xD;
&#xD;
          -  Minimum age of 21 years presently&#xD;
&#xD;
          -  Had a baseline gait and motion analysis&#xD;
&#xD;
        Controls (-SDR):&#xD;
&#xD;
          -  No SDR&#xD;
&#xD;
          -  No history of intrathecal baclofen (ITB) pump implantation for &gt; 1 year&#xD;
&#xD;
          -  No ITB pump at time of long-term follow-up (explant &gt; or = 6 months)&#xD;
&#xD;
          -  No history of oral baclofen for &gt; 1 year&#xD;
&#xD;
          -  No oral baclofen use at time of long-term follow-up&#xD;
&#xD;
          -  No more than 10 sessions on botulinum toxin, phenol, or alcohol injection&#xD;
&#xD;
        Cases (+SDR):&#xD;
&#xD;
          -  History of SDR &gt; 5 years ago&#xD;
&#xD;
          -  Had a baseline gait analysis &lt; or = 18 months before SDR&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  none&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>21 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Michael Schwartz, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Gillette Children's Specialty Healthcare</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Gillette Children's Specialty Healthcare</name>
      <address>
        <city>Saint Paul</city>
        <state>Minnesota</state>
        <zip>55101</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Children - Salt Lake City</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84103</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Childrens - Spokane</name>
      <address>
        <city>Spokane</city>
        <state>Washington</state>
        <zip>99204</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2021</verification_date>
  <study_first_submitted>November 20, 2018</study_first_submitted>
  <study_first_submitted_qc>December 26, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 31, 2018</study_first_posted>
  <last_update_submitted>February 4, 2021</last_update_submitted>
  <last_update_submitted_qc>February 4, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">February 5, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Gillette Children's Specialty Healthcare</investigator_affiliation>
    <investigator_full_name>Michael H Schwartz, PhD</investigator_full_name>
    <investigator_title>Director of Bioengineering Research, Gillette Children's Specialty Healthcare; Associate Professor of Orthopaedic Surgery, University of Minnesota - Twin Cities</investigator_title>
  </responsible_party>
  <keyword>Selective Dorsal Rhizotomy</keyword>
  <keyword>Outcomes</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cerebral Palsy</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03789786
org study id: STUDY00002353
nct id: NCT03789786
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Selective dorsal
      rhizotomy (SDR) is a surgical method used by some hospitals to permanently reduce spasticity
      in order to prevent further morbidities. Better understanding of the long-term outcomes of
      SDR is essential for clinicians and families. The results of this study will have direct
      clinical impact by equipping providers with the necessary information to counsel families
      during medical decision making.
Spasticity affects up to 80% of individuals diagnosed with cerebral palsy. Excessive
      spasticity is thought to be uncomfortable, reduce function, cause gait deviations (e.g.
      equinus), and contribute toward musculoskeletal deformity and an elevated energy cost while
      walking. As such, SDR is a surgical method used by some hospitals to permanently reduce
      spasticity in order to prevent the aforementioned morbidities.

      Treatment philosophies differ widely in regards to how aggressively to manage spasticity.
      Some centers (e.g. Gillette) aggressively treat spasticity early in life through a variety of
      measures such as SDR, intrathecal baclofen pumps, and botulinum toxin injections. Other
      centers (e.g. Shriners Hospitals for Children - Salt Lake City and Spokane) offer little in
      the way of spasticity reduction treatments.

      There are several compelling reasons to conduct the proposed research study. First, emerging
      evidence suggests that the elimination of spasticity during childhood via SDR does not
      prevent contractures and only partially explains poor gross motor function, both previously
      thought to be clear outcomes of the surgery. Additionally, many of the longitudinal cohort
      studies that examined SDR outcomes have shown many outcome measures peak 1-3 years after
      surgery, and then decline toward baseline (i.e. pre-SDR) levels. Lastly, the quality of the
      SDR outcome literature is poor. Rarely are outcomes looked at in context of a proper control
      group. Either a control group is absent or comprised of typically developing children. This
      limits our ability to understand how patients with cerebral palsy may age without undergoing
      an SDR.

      Better understanding of the long-term outcomes of SDR is essential for clinicians and
      families. The surgery, in general, is costly to families (time, expense, risk, etc.) and
      clinicians should have every confidence in the intended outcomes for any intervention.
observational model: Case-Control
time perspective: Other
measure: Three-dimensional gait and motion analysis
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare three-dimensional gait kinematics and kinetics across groups
measure: Energy expenditure
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare energy expenditure across groups
measure: Spasticity
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare spasticity, measured by Modified Ashworth Score (0 no increase in tone - 4 rigid in flexion or extension), across groups
measure: Passive range of motion
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare passive range of motion, measured by lower extremity physical exam, across groups
measure: Selective motor control
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare selective motor control (0 patterned movement - 2 complete isolated movement) across groups
measure: Lower extremity strength
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Compare lower extremity strength, measured by the manual muscle test, across groups
measure: Gross Motor Function Measure (GMFM-66)
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess function using portions of the GMFM-66 (0 low function - 100 high function) and compare across groups
measure: Gillette Functional Assessment Questionnaire (Gillette FAQ)
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess function and activity using the Gillette FAQ (self-reported survey, 0 low function - 10 high function) and compare across groups
measure: Functional Mobility Scale (FMS)
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess function and activity using the FMS (self-reported survey, 1 uses wheelchair - 6 independent) and compare across groups
measure: Participation Enfranchisement survey
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess participation using the Participation Enfranchisement survey (self-reported survey, true/false) and compare across groups
measure: Diener Satisfaction with Life Scale
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess satisfaction using the Diener Satisfaction with Life Scale (self-reported survey, 5 dissatisfied- 35 satisfied) and compare across groups
measure: World Health Organization (WHO) Quality of Life Scale
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess satisfaction using the WHO Quality of Life Scale (self-reported survey, 0 low quality of life - 100 high quality of life) and compare across groups
measure: Multiple Sclerosis Spasticity Scale (MSSS-88)
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Assess pain using portions of the MSSS-88 (self-reported survey, 21 not at all bothered - 84 extremely bothered) and compare across groups
measure: Change in gait and motion analysis
time frame: Baseline (qualifying exam for cases and controls) compared to long-term follow-up research visit (on average 10 years post-baseline)
description: Compare change in gait kinematics and kinetics within groups and if the change is similar between groups
measure: Incidence of prior surgery and anti-spastic treatments
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Incidence of prior surgery and anti-spastic treatments
measure: Cost of prior surgery and anti-spastic treatments
time frame: Time of long-term follow-up research visit (on average 10 years post-baseline)
description: Cost of prior surgery and anti-spastic treatments
arm group label: Cases (+SDR)
description: Patients with cerebral palsy that underwent an SDR
arm group label: Controls (-SDR)
description: Matched patients with cerebral palsy but did not undergo an SDR
intervention type: Diagnostic Test
intervention name: Gait and Motion Analysis
description: Gait and motion analysis is comprised of 3-dimensional kinematics and kinetics, electromyography, energy expenditure, and physical exam (range of motion, strength, spasticity, etc.)
arm group label: Cases (+SDR)
arm group label: Controls (-SDR)
intervention type: Diagnostic Test
intervention name: Gross Motor Function Measure (GMFM)
description: Gross Motor Function Measure (GMFM) is an assessment used to evaluate gross motor function over time in individuals with cerebral palsy. The assessment is comprised of movement activities like standing, walking, running, and jumping
arm group label: Cases (+SDR)
arm group label: Controls (-SDR)
intervention type: Behavioral
intervention name: Surveys
description: Six surveys are used to assess function, activity, participation, pain, quality of life, and treatment history
arm group label: Cases (+SDR)
arm group label: Controls (-SDR)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 21 Years
maximum age: N/A
healthy volunteers: No
last name: Michael Schwartz, PhD
role: Principal Investigator
affiliation: Gillette Children's Specialty Healthcare
facility: 
facility: 
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Gillette Children's Specialty Healthcare
investigator full name: Michael H Schwartz, PhD
investigator title: Director of Bioengineering Research, Gillette Children's Specialty Healthcare; Associate Professor of Orthopaedic Surgery, University of Minnesota - Twin Cities
mesh term: Cerebral Palsy
sharing ipd: No