<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/NCT03788317</url>
  </required_header>
  <id_info>
    <org_study_id>KU GOKAEK 2018/358</org_study_id>
    <nct_id>NCT03788317</nct_id>
  </id_info>
  <brief_title>Improved Sitting Balance: Body Functions to Participation</brief_title>
  <official_title>Integrated Botulinum Toxin for Improved Sitting Balance in Children With Cerebral Palsy: Body Functions to Activity and Participation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Kocaeli University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Kocaeli University</source>
  <oversight_info>
    <has_dmc>Yes</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>
      The aim of this study is to evaluate the changes in activity and participation domains of&#xD;
      International Classification of Functioning, Disability and Health (ICF) in children with&#xD;
      cerebral palsy (CP) who received integrated Botulinum Toxin-A (BoNT-A) injections into the&#xD;
      lower limb(s) with a treatment goal of improved sitting balance.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Postural problems play a substantial role in activities of daily living in children with CP&#xD;
      having Gross Motor Function Classification System (GMFCS) levels IV or V. Improvement in&#xD;
      sitting balance may positively effect the performance and caregiving of vital tasks of daily&#xD;
      life in these children and may furtherly improve the quality of life of both the child and&#xD;
      family. BoNT-A is an important treatment in children with CP because it is safe in young&#xD;
      children and allows combined treatment. In the current literature its efficacy on body&#xD;
      functions and structures by hypertonia management is well known. However there is&#xD;
      inconclusive evidence for improvement in activity and participation domains of ICF with&#xD;
      BoNT-A. CP is a very heterogenous population and in most of the studies the inclusion&#xD;
      criteria do not adress level of underlying motor control and the primary efficacy assessment&#xD;
      methods were usually chosen to cover hypertonia. Besides generally the efficacy endpoints&#xD;
      were so soon to detect changes in activity or participation domains of ICF. Lack of&#xD;
      association of rehabilitation programs is another important concern for the current clinical&#xD;
      studies.&#xD;
&#xD;
      In this present study a well defined subpopulation of CP children having GMFCS levels of IV&#xD;
      or V who each received integrated BoNT-A treatment with a primary goal of improved sitting&#xD;
      balance will be recruited to identify the probable changes in activity and participation&#xD;
      domains of ICF as well as changes in quality of life of the family and the child.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 28, 2018</start_date>
  <completion_date type="Actual">July 1, 2019</completion_date>
  <primary_completion_date type="Actual">June 26, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>The Child and Adolescent Scale of Participation (CASP)</measure>
    <time_frame>20 weeks</time_frame>
    <description>Participation measurement The CASP consists of 20 ordinal-scaled items and four subsections: 1) Home Participation 2) Community Participation 3) School Participation and 4) Home and Community Living Activities The 20 items are rated on a four-point scale: Age Expected (Full participation) to Unable.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>The Caregiver Priorities &amp; Child Health Index of Life with Disabilities (CPCHILD)</measure>
    <time_frame>20 weeks</time_frame>
    <description>Health Related Quality of Life measurement The CPCHILD© Questionnaire measures the caregiver's perspective about the child's health status, comfort, wellbeing, functional abilities and ease of caregiving. It is a useful proxy measure of health related quality of life of children with severe disabilities.&#xD;
Basic Scoring Procedure Scores for each domain and for the total survey are standardized and range from 0 (worse) to 100 (best).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Goal Assessment Scale (GAS)</measure>
    <time_frame>20 weeks</time_frame>
    <description>Functional goal attainment measurement GAS 5-Point Rating Scale Score Predicted Attainment&#xD;
(-2)Less than expected outcome&#xD;
(-1) Expected outcome after intervention&#xD;
(0)Much less than expected outcome&#xD;
(+1) Greater than expected outcome&#xD;
(+2) Much greater than expected outcome</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Gross Motor Function Classification System (GMFCS)</measure>
    <time_frame>20 weeks</time_frame>
    <description>Motor Development The gross motor skills (e.g. sitting and walking) of children and young people with cerebral palsy can be categorised into 5 different levels from I to V using a tool called GMFCS.</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean change from baseline Modified Ashworth Scale (MAS)</measure>
    <time_frame>20 weeks</time_frame>
    <description>Tone measurement Scoring 0= No increase muscle tone; 1= Slight increase ın muscle tone, manifested by a catch and release or by minimal resislance at the end of the range of motion when the affected part (s) is moved in flexion or extension;&#xD;
1+=Slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM; 2 =More marked increase in muscle tone through most of the ROM, but affected part (s) easily moved; 3 =Considerable increase in muscle tone, passive movement difficult; 4 =Affected part (s) rigid in flexion or extension.</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean change from baseline angle of catch (XV3) Tardieu scale</measure>
    <time_frame>20 weeks</time_frame>
    <description>Spasticity measurement Degree of anle of catch is measured baseline and after treatment to define the change from baseline</description>
  </other_outcome>
  <enrollment type="Actual">29</enrollment>
  <condition>Cerebral Palsy</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Botulinum toxin type A</intervention_name>
    <description>Injections to hypertonic muscles</description>
    <other_name>Botox, Dysport</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        All children diagnosis of cerebral palsy with respect to Rosenbaum criteria having received&#xD;
        BoNT-A injections to lower limb(s) as well as an integrated treatment approach&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  All children with a diagnosis of cerebral palsy with respect to Rosenbaum criteria&#xD;
&#xD;
          -  Gross Motor Functional Classification Scale level of IV-V&#xD;
&#xD;
          -  Being scheduled for BoNT-A treatment to lower extremity&#xD;
&#xD;
          -  Primary goal of treatment selected as improved sitting balance&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Severe cognitive or behavioural problems&#xD;
&#xD;
          -  Severe dystonia&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Nigar Dursun</last_name>
    <role>Study Director</role>
    <affiliation>Kocaeli University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Kocaeli University</name>
      <address>
        <city>Kocaeli</city>
        <zip>41050</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Turkey</country>
  </location_countries>
  <reference>
    <citation>Nguyen L, Di Rezze B, Mesterman R, Rosenbaum P, Gorter JW. Effects of Botulinum Toxin Treatment in Nonambulatory Children and Adolescents With Cerebral Palsy: Understanding Parents' Perspectives. J Child Neurol. 2018 Oct;33(11):724-733. doi: 10.1177/0883073818786567. Epub 2018 Jul 24.</citation>
    <PMID>30039731</PMID>
  </reference>
  <reference>
    <citation>Nguyen L, Mesterman R, Gorter JW. Development of an inventory of goals using the International Classification of Functioning, Disability and Health in a population of non-ambulatory children and adolescents with cerebral palsy treated with botulinum toxin A. BMC Pediatr. 2018 Jan 4;18(1):1. doi: 10.1186/s12887-017-0974-x.</citation>
    <PMID>29301539</PMID>
  </reference>
  <reference>
    <citation>Delgado MR, Tilton A, Russman B, Benavides O, Bonikowski M, Carranza J, Dabrowski E, Dursun N, Gormley M, Jozwiak M, Matthews D, Maciag-Tymecka I, Unlu E, Pham E, Tse A, Picaut P. AbobotulinumtoxinA for Equinus Foot Deformity in Cerebral Palsy: A Randomized Controlled Trial. Pediatrics. 2016 Feb;137(2):e20152830. doi: 10.1542/peds.2015-2830. Epub 2016 Jan 26.</citation>
    <PMID>26812925</PMID>
  </reference>
  <reference>
    <citation>Dursun N, Gokbel T, Akarsu M, Dursun E. Randomized Controlled Trial on Effectiveness of Intermittent Serial Casting on Spastic Equinus Foot in Children with Cerebral Palsy After Botulinum Toxin-A Treatment. Am J Phys Med Rehabil. 2017 Apr;96(4):221-225. doi: 10.1097/PHM.0000000000000627.</citation>
    <PMID>27631386</PMID>
  </reference>
  <reference>
    <citation>Reid SM, Carlin JB, Reddihough DS. Using the Gross Motor Function Classification System to describe patterns of motor severity in cerebral palsy. Dev Med Child Neurol. 2011 Nov;53(11):1007-12. doi: 10.1111/j.1469-8749.2011.04044.x.</citation>
    <PMID>22014320</PMID>
  </reference>
  <verification_date>August 2019</verification_date>
  <study_first_submitted>December 25, 2018</study_first_submitted>
  <study_first_submitted_qc>December 25, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 27, 2018</study_first_posted>
  <last_update_submitted>August 20, 2019</last_update_submitted>
  <last_update_submitted_qc>August 20, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Kocaeli University</investigator_affiliation>
    <investigator_full_name>Nigar Dursun</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <keyword>Cerebral Palsy, Botulinum Toxin A, Participation</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cerebral Palsy</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>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT03788317
org study id: KU GOKAEK 2018/358
nct id: NCT03788317
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
The aim of this study is to evaluate the changes in activity and participation domains of
      International Classification of Functioning, Disability and Health (ICF) in children with
      cerebral palsy (CP) who received integrated Botulinum Toxin-A (BoNT-A) injections into the
      lower limb(s) with a treatment goal of improved sitting balance.
Postural problems play a substantial role in activities of daily living in children with CP
      having Gross Motor Function Classification System (GMFCS) levels IV or V. Improvement in
      sitting balance may positively effect the performance and caregiving of vital tasks of daily
      life in these children and may furtherly improve the quality of life of both the child and
      family. BoNT-A is an important treatment in children with CP because it is safe in young
      children and allows combined treatment. In the current literature its efficacy on body
      functions and structures by hypertonia management is well known. However there is
      inconclusive evidence for improvement in activity and participation domains of ICF with
      BoNT-A. CP is a very heterogenous population and in most of the studies the inclusion
      criteria do not adress level of underlying motor control and the primary efficacy assessment
      methods were usually chosen to cover hypertonia. Besides generally the efficacy endpoints
      were so soon to detect changes in activity or participation domains of ICF. Lack of
      association of rehabilitation programs is another important concern for the current clinical
      studies.

      In this present study a well defined subpopulation of CP children having GMFCS levels of IV
      or V who each received integrated BoNT-A treatment with a primary goal of improved sitting
      balance will be recruited to identify the probable changes in activity and participation
      domains of ICF as well as changes in quality of life of the family and the child.
observational model: Case-Only
time perspective: Prospective
measure: The Child and Adolescent Scale of Participation (CASP)
time frame: 20 weeks
description: Participation measurement The CASP consists of 20 ordinal-scaled items and four subsections: 1) Home Participation 2) Community Participation 3) School Participation and 4) Home and Community Living Activities The 20 items are rated on a four-point scale: Age Expected (Full participation) to Unable.
measure: The Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD)
time frame: 20 weeks
description: Health Related Quality of Life measurement The CPCHILD© Questionnaire measures the caregiver's perspective about the child's health status, comfort, wellbeing, functional abilities and ease of caregiving. It is a useful proxy measure of health related quality of life of children with severe disabilities.
Basic Scoring Procedure Scores for each domain and for the total survey are standardized and range from 0 (worse) to 100 (best).
measure: Goal Assessment Scale (GAS)
time frame: 20 weeks
description: Functional goal attainment measurement GAS 5-Point Rating Scale Score Predicted Attainment
(-2)Less than expected outcome
(-1) Expected outcome after intervention
(0)Much less than expected outcome
(+1) Greater than expected outcome
(+2) Much greater than expected outcome
measure: Gross Motor Function Classification System (GMFCS)
time frame: 20 weeks
description: Motor Development The gross motor skills (e.g. sitting and walking) of children and young people with cerebral palsy can be categorised into 5 different levels from I to V using a tool called GMFCS.
measure: Mean change from baseline Modified Ashworth Scale (MAS)
time frame: 20 weeks
description: Tone measurement Scoring 0= No increase muscle tone; 1= Slight increase ın muscle tone, manifested by a catch and release or by minimal resislance at the end of the range of motion when the affected part (s) is moved in flexion or extension;
1+=Slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM; 2 =More marked increase in muscle tone through most of the ROM, but affected part (s) easily moved; 3 =Considerable increase in muscle tone, passive movement difficult; 4 =Affected part (s) rigid in flexion or extension.
measure: Mean change from baseline angle of catch (XV3) Tardieu scale
time frame: 20 weeks
description: Spasticity measurement Degree of anle of catch is measured baseline and after treatment to define the change from baseline
intervention type: Drug
intervention name: Botulinum toxin type A
description: Injections to hypertonic muscles
other name: Botox, Dysport
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: 18 Years
healthy volunteers: No
last name: Nigar Dursun
role: Study Director
affiliation: Kocaeli University
facility: 
country: Turkey
citation: Nguyen L, Di Rezze B, Mesterman R, Rosenbaum P, Gorter JW. Effects of Botulinum Toxin Treatment in Nonambulatory Children and Adolescents With Cerebral Palsy: Understanding Parents' Perspectives. J Child Neurol. 2018 Oct;33(11):724-733. doi: 10.1177/0883073818786567. Epub 2018 Jul 24.
PMID: 30039731
citation: Nguyen L, Mesterman R, Gorter JW. Development of an inventory of goals using the International Classification of Functioning, Disability and Health in a population of non-ambulatory children and adolescents with cerebral palsy treated with botulinum toxin A. BMC Pediatr. 2018 Jan 4;18(1):1. doi: 10.1186/s12887-017-0974-x.
PMID: 29301539
citation: Delgado MR, Tilton A, Russman B, Benavides O, Bonikowski M, Carranza J, Dabrowski E, Dursun N, Gormley M, Jozwiak M, Matthews D, Maciag-Tymecka I, Unlu E, Pham E, Tse A, Picaut P. AbobotulinumtoxinA for Equinus Foot Deformity in Cerebral Palsy: A Randomized Controlled Trial. Pediatrics. 2016 Feb;137(2):e20152830. doi: 10.1542/peds.2015-2830. Epub 2016 Jan 26.
PMID: 26812925
citation: Dursun N, Gokbel T, Akarsu M, Dursun E. Randomized Controlled Trial on Effectiveness of Intermittent Serial Casting on Spastic Equinus Foot in Children with Cerebral Palsy After Botulinum Toxin-A Treatment. Am J Phys Med Rehabil. 2017 Apr;96(4):221-225. doi: 10.1097/PHM.0000000000000627.
PMID: 27631386
citation: Reid SM, Carlin JB, Reddihough DS. Using the Gross Motor Function Classification System to describe patterns of motor severity in cerebral palsy. Dev Med Child Neurol. 2011 Nov;53(11):1007-12. doi: 10.1111/j.1469-8749.2011.04044.x.
PMID: 22014320
responsible party type: Principal Investigator
investigator affiliation: Kocaeli University
investigator full name: Nigar Dursun
investigator title: Professor
mesh term: Cerebral Palsy
mesh term: Botulinum Toxins
mesh term: Botulinum Toxins, Type A
mesh term: abobotulinumtoxinA
sharing ipd: Undecided