<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/NCT03017729</url>
  </required_header>
  <id_info>
    <org_study_id>A-US-52120-330</org_study_id>
    <nct_id>NCT03017729</nct_id>
  </id_info>
  <brief_title>Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity</brief_title>
  <official_title>A Phase IV, Prospective, Observational, Multicentre Study Evaluating the Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ipsen</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ipsen</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to assess the longitudinal attainment of subject centred and&#xD;
      functional related goals (cumulated Goal Attainment Scale Total (GAS T) score) after&#xD;
      abobotulinumtoxinA injection (including following repeated injection cycles where they occur)&#xD;
      alongside spasticity management used in real life settings over a period of 18 months (and a&#xD;
      maximum of six injection cycles).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 10, 2017</start_date>
  <completion_date type="Actual">November 30, 2020</completion_date>
  <primary_completion_date type="Actual">November 30, 2020</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>Cumulated GAS T score</measure>
    <time_frame>From day 1 up to 30 months</time_frame>
    <description>Defined as the mean of the individual GAS T scores across all cycles will be used to measure progress towards individual therapy goals. If all goals are achieved as expected, the GAS T score is 50.0.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>AbobotulinumtoxinA dose</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time intervals between injections</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of injection points</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Muscle(s) injected</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Sedation used</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Type of injection guidance utilised</measure>
    <time_frame>Day 1, then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Concomitant drug therapies</measure>
    <time_frame>From day 1 up to 30 months</time_frame>
    <description>Listed and tabulated by frequency</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Non-drug therapies</measure>
    <time_frame>From day 1 up to 30 months</time_frame>
    <description>Listed and tabulated by frequency</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Modified Ashworth scale (as applicable) in the injected muscle groups (gastrocnemius, soleus and others) at baseline and per the investigators' decision/routine practice during the course of the study.</measure>
    <time_frame>From day 1 up to 30 months (per investigator's decision/routine practice)</time_frame>
    <description>This evaluation will not be mandatory during this study. Response over time will be presented using descriptive statistics.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Average GAS T score</measure>
    <time_frame>Day 1 then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>Per injection cycle</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage achievement of primary treatment goal</measure>
    <time_frame>Day 1 then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>During each injection cycle and overall</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections</measure>
    <time_frame>Day 1 then every 3.5 months (approximately) up to 30 months</time_frame>
    <description>Per injection cycle</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of adverse events and special situations collected</measure>
    <time_frame>From day 1 up to 30 months</time_frame>
    <description>Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Direct and indirect health care costs</measure>
    <time_frame>From day 1 up to 30 months</time_frame>
    <description>Derived from the collected data, including concomitant treatments.</description>
  </secondary_outcome>
  <enrollment type="Actual">242</enrollment>
  <condition>Lower Limb Spasticity</condition>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Botulinum toxin type A</intervention_name>
    <description>This is an observational study designed to reflect real world clinical practice in the use of abobotulinumtoxinA. AbobotulinumtoxinA injected doses, frequency of injections, number of injection sites and volume injected per site are in accordance with the current USPI and physician's clinical practice.</description>
    <other_name>AbobotulinumtoxinA (Dysport®)</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Pediatric patients with lower limb spasticity&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Female or male subjects aged 2 to 17 years inclusive&#xD;
&#xD;
          -  Decision to prescribe abobotulinumtoxinA, to be made prior to and independently from&#xD;
             the decision to enroll in the study&#xD;
&#xD;
          -  Primary diagnosis of paediatric lower limb (PLL) spasticity and either: Previously&#xD;
             untreated with BoNT (naïve to BoNT), or previously treated with a BoNT (i.e. non naïve&#xD;
             to BoNT), and for those who were previously treated with BoNT-A, they should have&#xD;
             responded to BoNT-A treatment according to the investigator's criteria&#xD;
&#xD;
          -  For non naïve BoNT subjects, a minimum interval of 12 weeks since the last BoNT&#xD;
             injection and in the presence of spasticity&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Known resistance to any BoNT or experienced serious safety issues with previous use of&#xD;
             BoNT&#xD;
&#xD;
          -  Concomitant treatment with other BoNT&#xD;
&#xD;
          -  Known hypersensitivity to abobotulinumtoxinA or related compounds, or any component in&#xD;
             the study drug formulation&#xD;
&#xD;
          -  Subjects with any clinical (or subclinical) evidence of marked defective neuromuscular&#xD;
             transmission (e.g. Lambert Eaton syndrome or myasthenia gravis) or persistent&#xD;
             clinically significant neuromuscular disorders&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Ipsen Medical Director</last_name>
    <role>Study Director</role>
    <affiliation>Ipsen</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's National Medical Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nicklaus Children's Hospital</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33155</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Laszlo J. Mate, M.D.</name>
      <address>
        <city>North Palm Beach</city>
        <state>Florida</state>
        <zip>33408</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinical Integrative Research Central Atlanta</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30328</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mt. Washington Pediatric Hospital</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21209</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>William Beaumont Hospital Pediatric Research</name>
      <address>
        <city>Royal Oak</city>
        <state>Michigan</state>
        <zip>48073</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <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>University of Missouri</name>
      <address>
        <city>Columbia</city>
        <state>Missouri</state>
        <zip>65212</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University School Of Medicine</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Valley Health System</name>
      <address>
        <city>Ridgewood</city>
        <state>New Jersey</state>
        <zip>07450</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Montefiore Medical Center</name>
      <address>
        <city>Bronx</city>
        <state>New York</state>
        <zip>10467</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Akron Children's Hospital</name>
      <address>
        <city>Akron</city>
        <state>Ohio</state>
        <zip>44308</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cleveland Clinic</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dayton Children's Hospital</name>
      <address>
        <city>Dayton</city>
        <state>Ohio</state>
        <zip>45404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Center</name>
      <address>
        <city>Bethany</city>
        <state>Oklahoma</state>
        <zip>73008</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Children</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Good Shepherd Rehabilitation Network</name>
      <address>
        <city>Allentown</city>
        <state>Pennsylvania</state>
        <zip>18103</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Children</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19140</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Children</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29605</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital at Erlanger</name>
      <address>
        <city>Chattanooga</city>
        <state>Tennessee</state>
        <zip>37403</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Scottish Rite Hospital for Children</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75219</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shriners Hospitals for Children</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's</name>
      <address>
        <city>Plano</city>
        <state>Texas</state>
        <zip>75024</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Hospital of San Antonio</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Utah Neuro Rehabilitation</name>
      <address>
        <city>Murray</city>
        <state>Utah</state>
        <zip>84124</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Wisconsin</name>
      <address>
        <city>Milwaukee</city>
        <state>Wisconsin</state>
        <zip>53226</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2020</verification_date>
  <study_first_submitted>December 30, 2016</study_first_submitted>
  <study_first_submitted_qc>January 9, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 11, 2017</study_first_posted>
  <last_update_submitted>December 17, 2020</last_update_submitted>
  <last_update_submitted_qc>December 17, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 19, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscle Spasticity</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Botulinum Toxins</mesh_term>
    <mesh_term>Botulinum Toxins, Type A</mesh_term>
    <mesh_term>abobotulinumtoxinA</mesh_term>
  </intervention_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/NCT03017729
org study id: A-US-52120-330
nct id: NCT03017729
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to assess the longitudinal attainment of subject centred and
      functional related goals (cumulated Goal Attainment Scale Total (GAS T) score) after
      abobotulinumtoxinA injection (including following repeated injection cycles where they occur)
      alongside spasticity management used in real life settings over a period of 18 months (and a
      maximum of six injection cycles).
observational model: Cohort
time perspective: Prospective
measure: Cumulated GAS T score
time frame: From day 1 up to 30 months
description: Defined as the mean of the individual GAS T scores across all cycles will be used to measure progress towards individual therapy goals. If all goals are achieved as expected, the GAS T score is 50.0.
measure: AbobotulinumtoxinA dose
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Time intervals between injections
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Number of injection points
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Muscle(s) injected
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Sedation used
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Type of injection guidance utilised
time frame: Day 1, then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Concomitant drug therapies
time frame: From day 1 up to 30 months
description: Listed and tabulated by frequency
measure: Non-drug therapies
time frame: From day 1 up to 30 months
description: Listed and tabulated by frequency
measure: Modified Ashworth scale (as applicable) in the injected muscle groups (gastrocnemius, soleus and others) at baseline and per the investigators' decision/routine practice during the course of the study.
time frame: From day 1 up to 30 months (per investigator's decision/routine practice)
description: This evaluation will not be mandatory during this study. Response over time will be presented using descriptive statistics.
measure: Average GAS T score
time frame: Day 1 then every 3.5 months (approximately) up to 30 months
description: Per injection cycle
measure: Percentage achievement of primary treatment goal
time frame: Day 1 then every 3.5 months (approximately) up to 30 months
description: During each injection cycle and overall
measure: Percentage achievement of primary treatment goal(s) per goal area(s) after repeated abobotulinumtoxinA injections
time frame: Day 1 then every 3.5 months (approximately) up to 30 months
description: Per injection cycle
measure: Incidence of adverse events and special situations collected
time frame: From day 1 up to 30 months
description: Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA).
measure: Direct and indirect health care costs
time frame: From day 1 up to 30 months
description: Derived from the collected data, including concomitant treatments.
intervention type: Biological
intervention name: Botulinum toxin type A
description: This is an observational study designed to reflect real world clinical practice in the use of abobotulinumtoxinA. AbobotulinumtoxinA injected doses, frequency of injections, number of injection sites and volume injected per site are in accordance with the current USPI and physician's clinical practice.
other name: AbobotulinumtoxinA (Dysport®)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: 17 Years
healthy volunteers: No
last name: Ipsen Medical Director
role: Study Director
affiliation: Ipsen
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country: United States
responsible party type: Sponsor
mesh term: Muscle Spasticity
mesh term: Botulinum Toxins
mesh term: Botulinum Toxins, Type A
mesh term: abobotulinumtoxinA