<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/NCT02227823</url>
  </required_header>
  <id_info>
    <org_study_id>1376</org_study_id>
    <nct_id>NCT02227823</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3</brief_title>
  <acronym>EMOTAS</acronym>
  <official_title>Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Centre Hospitalier Régional de la Citadelle</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Centre Hospitalier Régional de la Citadelle</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy&#xD;
      on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a&#xD;
      genetically transmitted disease inducing muscular weakness predominating on shoulders and&#xD;
      hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is&#xD;
      due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a&#xD;
      neuromuscular junction (NMJ) involvement, according to recent studies.&#xD;
&#xD;
      EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance&#xD;
      and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by&#xD;
      non-invasive therapy quality of life of patients.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>July 2014</start_date>
  <completion_date type="Anticipated">July 2017</completion_date>
  <primary_completion_date type="Anticipated">July 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Factorial Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change from Baseline in the distance walked at 6-minute walk test at 6 months</measure>
    <time_frame>6 months</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of decrement at 6 months</measure>
    <time_frame>6 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of MFM-D1</measure>
    <time_frame>6 months</time_frame>
    <description>Comparison of treated and control group values will be made</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of Moviplate values at 6 months</measure>
    <time_frame>6 months</time_frame>
    <description>Comparison between treated and control group value will be made</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of the ratio at 6 minutes walk test at 6 months</measure>
    <time_frame>6 months</time_frame>
    <description>It's the ratio between the number of meters during the last minute of the 6-minute walk test and the first minute of the 6-minute walk test.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>Spinal Muscular Atrophy Type 3</condition>
  <arm_group>
    <arm_group_label>significant decrement</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg</description>
  </arm_group>
  <arm_group>
    <arm_group_label>no decrement</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>Patient without significant decrement will not receive any treatment and will be the control group</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Pyridostigmine Bromide</intervention_name>
    <arm_group_label>significant decrement</arm_group_label>
    <other_name>Mestinon</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Spinal muscular atrophy type 3, genetically confirmed&#xD;
&#xD;
               -  Age higher than 6 years old&#xD;
&#xD;
               -  Ambulatory patient&#xD;
&#xD;
               -  Informed consent signed&#xD;
&#xD;
               -  More than 100 meters of walking at 6-minute walk test at screening&#xD;
&#xD;
               -  Value at screening and baseline in a range of 20% of the highest value at&#xD;
                  6-minute walk test&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patient who had surgical intervention or suffer from a recent traumatism (less than 6&#xD;
             months)&#xD;
&#xD;
               -  Associated pathology such as endocrinopathy, infectious disease, allergy,&#xD;
                  myopathy, chronic or acute inflammatory pathology, during 3 weeks preceding the&#xD;
                  inclusion.&#xD;
&#xD;
               -  Other therapeutics than food supplements or those frequently prescribed in spinal&#xD;
                  muscular atrophy or its complications&#xD;
&#xD;
               -  Non tolerance of electromyography&#xD;
&#xD;
               -  Limited collaboration due to trouble in information comprehension&#xD;
&#xD;
               -  Pathology inducing contra-indication for pyridostigmine treatment (allergy at&#xD;
                  molecule, asthma, Parkinson disease, mechanic obstruction of urinary or digestive&#xD;
                  tracts)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Stephanie Delstanche</last_name>
    <role>Principal Investigator</role>
    <affiliation>Centre de référence des maladies neuromusculaire de Liège</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle</name>
      <address>
        <city>Liège</city>
        <zip>4000</zip>
        <country>Belgium</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Stephanie Delstanche</last_name>
      <email>stephanie.delstanche@chrcitadelle.be</email>
    </contact>
    <contact_backup>
      <last_name>Severine Denis</last_name>
      <email>severine.denis@chrcitadelle.be</email>
    </contact_backup>
    <investigator>
      <last_name>Stephanie Delstanche</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Belgium</country>
  </location_countries>
  <verification_date>August 2014</verification_date>
  <study_first_submitted>August 20, 2014</study_first_submitted>
  <study_first_submitted_qc>August 26, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 28, 2014</study_first_posted>
  <last_update_submitted>August 26, 2014</last_update_submitted>
  <last_update_submitted_qc>August 26, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">August 28, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Centre Hospitalier Régional de la Citadelle</investigator_affiliation>
    <investigator_full_name>Dr. Stéphanie Delstanche</investigator_full_name>
    <investigator_title>Neurologist</investigator_title>
  </responsible_party>
  <keyword>spinal muscular atrophy type 3</keyword>
  <keyword>electromyography</keyword>
  <keyword>fatigability</keyword>
  <keyword>decrement</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Atrophy</mesh_term>
    <mesh_term>Muscular Atrophy, Spinal</mesh_term>
    <mesh_term>Spinal Muscular Atrophies of Childhood</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Bromides</mesh_term>
    <mesh_term>Pyridostigmine Bromide</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/NCT02227823
org study id: 1376
nct id: NCT02227823
lead sponsor: 
has dmc: No
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy
      on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a
      genetically transmitted disease inducing muscular weakness predominating on shoulders and
      hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is
      due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a
      neuromuscular junction (NMJ) involvement, according to recent studies.

      EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance
      and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by
      non-invasive therapy quality of life of patients.
allocation: Non-Randomized
intervention model: Factorial Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change from Baseline in the distance walked at 6-minute walk test at 6 months
time frame: 6 months
measure: Change from baseline of decrement at 6 months
time frame: 6 months
measure: Change from baseline of MFM-D1
time frame: 6 months
description: Comparison of treated and control group values will be made
measure: Change from baseline of Moviplate values at 6 months
time frame: 6 months
description: Comparison between treated and control group value will be made
measure: Change from baseline of the ratio at 6 minutes walk test at 6 months
time frame: 6 months
description: It's the ratio between the number of meters during the last minute of the 6-minute walk test and the first minute of the 6-minute walk test.
arm group label: significant decrement
arm group type: Experimental
description: Patients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg
arm group label: no decrement
arm group type: No Intervention
description: Patient without significant decrement will not receive any treatment and will be the control group
intervention type: Drug
intervention name: Pyridostigmine Bromide
arm group label: significant decrement
other name: Mestinon
criteria: 
gender: All
minimum age: 6 Years
maximum age: N/A
healthy volunteers: No
last name: Stephanie Delstanche
role: Principal Investigator
affiliation: Centre de référence des maladies neuromusculaire de Liège
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
country: Belgium
responsible party type: Principal Investigator
investigator affiliation: Centre Hospitalier Régional de la Citadelle
investigator full name: Dr. Stéphanie Delstanche
investigator title: Neurologist
mesh term: Muscular Atrophy
mesh term: Muscular Atrophy, Spinal
mesh term: Spinal Muscular Atrophies of Childhood
mesh term: Atrophy
mesh term: Bromides
mesh term: Pyridostigmine Bromide