<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/NCT02235090</url>
  </required_header>
  <id_info>
    <org_study_id>CSMA-INPN-1</org_study_id>
    <nct_id>NCT02235090</nct_id>
  </id_info>
  <brief_title>Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting</brief_title>
  <official_title>Assessment of Feasibility and Statistical Reliability of Functional Outcomes Measurement in Neuromuscular Patients Without Trial Site Visiting by Standard Functional Scales and by Special Autonomic Device in Double-blind, Placebo Controlled Study of Cervical Spinal Cord Transdermal Direct Current Stimulation in Patients With Spinal Muscular Atrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Charitable Foundation Children with Spinal Muscular Atrophy</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Charitable Foundation Children with Spinal Muscular Atrophy</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Clinical trials organization in several neuromuscular disorders (NMD) has some specific&#xD;
      issues. Nonambulant status and difficulties with transportation are among them. Moreover a&#xD;
      lot of patients with NMD have so poor condition that even short transportation is able to&#xD;
      worse it. Such situation forces researchers to limit a region of recruitment for clinical&#xD;
      trials and to exclude from trials more severe subgroup of patients, which cause additional&#xD;
      issues especially for rare diseases.&#xD;
&#xD;
      The purpose of this study is to prove hypothesis about possibility to reliably monitor&#xD;
      patient condition remotely, without trial site visiting. Visit-free study design is&#xD;
      potentially able to widen eligible patient population and to decrease patient dropout rate as&#xD;
      well as burden of numerous assessments. Meanwhile assessment frequency could be increased&#xD;
      enabling monitoring of short fluctuations in patients' condition.&#xD;
&#xD;
      Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above&#xD;
      issues are completely applicable. Direct current stimulation (DCS) of neural structures is&#xD;
      well studied and safe intervention, however, its effects on SMA patients' strength and&#xD;
      durability has not been reported for today. The investigators suppose that investigation of&#xD;
      DCS action in SMA patient population is an adequate model for visit-free design feasibility,&#xD;
      reliability and sensitivity evaluation.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Trial was not started due to lack of funding and nobody received an intervention.&#xD;
  </why_stopped>
  <start_date type="Actual">October 2016</start_date>
  <completion_date type="Actual">October 30, 2017</completion_date>
  <primary_completion_date type="Actual">September 30, 2017</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity</measure>
    <time_frame>Before and 0, 15, 30 minutes after spinal cord direct current stimulation</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Short time fluctuations of Hammersmith Functional Motor Scale indexes</measure>
    <time_frame>Three times, three days consecutive measurement, every two months, assessed up to 6 months</time_frame>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Spinal Muscular Atrophy</condition>
  <condition>Neuromuscular Disorders</condition>
  <arm_group>
    <arm_group_label>Zero-strength of direct current stimulation</arm_group_label>
    <arm_group_type>Sham Comparator</arm_group_type>
    <description>Sham transdermal direct current stimulation of cervical spinal cord</description>
  </arm_group>
  <arm_group>
    <arm_group_label>100 microamperes direct current stimulation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Transdermal direct current stimulation of cervical spinal cord</description>
  </arm_group>
  <arm_group>
    <arm_group_label>1 milliampere direct current stimulation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Transdermal direct current stimulation of cervical spinal cord</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Direct current stimulation of cervical spinal cord</intervention_name>
    <description>10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord</description>
    <arm_group_label>1 milliampere direct current stimulation</arm_group_label>
    <arm_group_label>100 microamperes direct current stimulation</arm_group_label>
    <arm_group_label>Zero-strength of direct current stimulation</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Signed informed consent&#xD;
&#xD;
          -  5q SMA confirmed by molecular testing&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Need for ventilation&#xD;
&#xD;
          -  Hypersensitivity (pain or allergic reaction) to current stimulation&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Andriy V. Shatillo, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>State Institution &quot;Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine&quot;</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>SI &quot;Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine&quot;</name>
      <address>
        <city>Kharkiv</city>
        <zip>61068</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Ukraine</country>
  </location_countries>
  <verification_date>February 2019</verification_date>
  <study_first_submitted>September 3, 2014</study_first_submitted>
  <study_first_submitted_qc>September 5, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 9, 2014</study_first_posted>
  <last_update_submitted>February 12, 2019</last_update_submitted>
  <last_update_submitted_qc>February 12, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">February 15, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Spinal muscular atrophy</keyword>
  <keyword>Neuromuscular disorders</keyword>
  <keyword>Outcome measures</keyword>
  <keyword>Visit-free clinical trial design</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>Neuromuscular Diseases</mesh_term>
    <mesh_term>Atrophy</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/NCT02235090
org study id: CSMA-INPN-1
nct id: NCT02235090
lead sponsor: 
has dmc: Yes
Clinical trials organization in several neuromuscular disorders (NMD) has some specific
      issues. Nonambulant status and difficulties with transportation are among them. Moreover a
      lot of patients with NMD have so poor condition that even short transportation is able to
      worse it. Such situation forces researchers to limit a region of recruitment for clinical
      trials and to exclude from trials more severe subgroup of patients, which cause additional
      issues especially for rare diseases.

      The purpose of this study is to prove hypothesis about possibility to reliably monitor
      patient condition remotely, without trial site visiting. Visit-free study design is
      potentially able to widen eligible patient population and to decrease patient dropout rate as
      well as burden of numerous assessments. Meanwhile assessment frequency could be increased
      enabling monitoring of short fluctuations in patients' condition.

      Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above
      issues are completely applicable. Direct current stimulation (DCS) of neural structures is
      well studied and safe intervention, however, its effects on SMA patients' strength and
      durability has not been reported for today. The investigators suppose that investigation of
      DCS action in SMA patient population is an adequate model for visit-free design feasibility,
      reliability and sensitivity evaluation.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Other
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity
time frame: Before and 0, 15, 30 minutes after spinal cord direct current stimulation
measure: Short time fluctuations of Hammersmith Functional Motor Scale indexes
time frame: Three times, three days consecutive measurement, every two months, assessed up to 6 months
arm group label: Zero-strength of direct current stimulation
arm group type: Sham Comparator
description: Sham transdermal direct current stimulation of cervical spinal cord
arm group label: 100 microamperes direct current stimulation
arm group type: Experimental
description: Transdermal direct current stimulation of cervical spinal cord
arm group label: 1 milliampere direct current stimulation
arm group type: Experimental
description: Transdermal direct current stimulation of cervical spinal cord
intervention type: Other
intervention name: Direct current stimulation of cervical spinal cord
description: 10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord
arm group label: 1 milliampere direct current stimulation
arm group label: 100 microamperes direct current stimulation
arm group label: Zero-strength of direct current stimulation
criteria: 
gender: All
minimum age: 5 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Andriy V. Shatillo, MD, PhD
role: Principal Investigator
affiliation: State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
facility: 
country: Ukraine
responsible party type: Sponsor
mesh term: Muscular Atrophy
mesh term: Muscular Atrophy, Spinal
mesh term: Neuromuscular Diseases
mesh term: Atrophy