<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/NCT03034317</url>
  </required_header>
  <id_info>
    <org_study_id>20-1000-51</org_study_id>
    <nct_id>NCT03034317</nct_id>
  </id_info>
  <brief_title>NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS)</brief_title>
  <acronym>PARADIGM</acronym>
  <official_title>Multi-Center, Concurrent-Controlled, On-Label Study of the NeuRx Diaphragm Pacing System (DPS) in Participants With Amyotrophic Lateral Sclerosis (ALS)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Synapse Biomedical</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Synapse Biomedical</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>Yes</is_fda_regulated_device>
    <is_us_export>Yes</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this research study is to collect more information about the use, safety, and&#xD;
      effectiveness of the NeuRx DPS® in ALS patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Characterize the safety and benefit of on-label Humanitarian Device Exemption (HDE) use of&#xD;
      DPS in patients with ALS who initiated noninvasive ventilation (NIV).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    No participants enrolled&#xD;
  </why_stopped>
  <start_date type="Actual">February 2, 2017</start_date>
  <completion_date type="Actual">February 12, 2018</completion_date>
  <primary_completion_date type="Actual">February 8, 2018</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
    <masking_description>Open label</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Survival</measure>
    <time_frame>24 months</time_frame>
    <description>Compare the survival of patients who initiated noninvasive ventilation (NIV).and who receive DPS to controls without DPS: (1) concurrent controls; (2) historical controls (e.g., Lechtzin and PRO-ACT databases); and (3) virtual controls (Origent).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Relationship between survival time and clinical features of ALS</measure>
    <time_frame>24 months</time_frame>
    <description>Determine whether there is a relationship between survival time and ALS onset type.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relationship between survival time and ALS assessment scores</measure>
    <time_frame>24 months</time_frame>
    <description>Determine whether there is a relationship between survival time and Upper Motor Neuron (UMN)/ Lower Motor Neuron (LMN) assessment scores.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relationship between survival time and onset of weakness from ALS to treatment</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relationship between survival time and ALS treatment interventions</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relationship between survival time and intraoperative strength of contraction</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Characterize change in overall and respiratory function</measure>
    <time_frame>24 months</time_frame>
    <description>Characterize longitudinal change in overall function (ALSFRS-R) before and after treatment with DPS compared to controls.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Characterize change in respiratory function</measure>
    <time_frame>24 months</time_frame>
    <description>Characterize longitudinal change in respiratory function (FVC, MIP) before and after treatment with DPS compared to controls.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Types device and procedure-related adverse events</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Frequency of device and procedure-related adverse events</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Types of respiratory serious adverse events</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Frequency of respiratory adverse events</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>NeuRx DPS</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS).</description>
  </arm_group>
  <arm_group>
    <arm_group_label>No DPS</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>Subjects who have initiated noninvasive ventilation (NIV) and do not receive the DPS device.</description>
  </arm_group>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>NeuRx DPS</intervention_name>
    <description>Patients who are diagnosed with ALS, have initiated NIV and choose to have the NeuRx DPS implanted.</description>
    <arm_group_label>NeuRx DPS</arm_group_label>
    <other_name>Diaphragm Pacing System</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age 21 or older&#xD;
&#xD;
          -  Familial or sporadic ALS diagnosed as possible, laboratory-supported probable,&#xD;
             probable, or definite according to the World Federation of Neurology El Escorial&#xD;
             criteria&#xD;
&#xD;
          -  Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral&#xD;
             diaphragm movement with fluoroscopic sniff test or with phrenic nerve conduction&#xD;
             studies&#xD;
&#xD;
          -  Chronic hypoventilation was documented by at least one of the following:&#xD;
&#xD;
               -  MIP (respiratory muscle strength) less than 60 cmH2O, or&#xD;
&#xD;
               -  Forced Vital Capacity (FVC) less than 50% predicted, or&#xD;
&#xD;
               -  PaCO2 greater than or equal to 45 mmHg, or&#xD;
&#xD;
               -  Nocturnal SaO2 (arterial oxygen saturation) less than or equal to 88% for at&#xD;
                  least five continuous minutes&#xD;
&#xD;
          -  Initiation of non-invasive ventilation at or before time of enrollment&#xD;
&#xD;
          -  Suitable surgical candidate to receive diaphragm pacing stimulation&#xD;
&#xD;
          -  Negative pregnancy test in female participants of childbearing potential (treatment&#xD;
             group)&#xD;
&#xD;
          -  Informed consent from patient or designated representative&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  FVC less than 45% predicted within 10 days prior to surgery.&#xD;
&#xD;
          -  Participation in simultaneous therapeutic clinical trial which could affect the safety&#xD;
             or benefit outcomes of this study.&#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>Robert Miller, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>California Pacific Medical Center</affiliation>
  </overall_official>
  <verification_date>March 2020</verification_date>
  <study_first_submitted>January 13, 2017</study_first_submitted>
  <study_first_submitted_qc>January 25, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 27, 2017</study_first_posted>
  <last_update_submitted>March 24, 2020</last_update_submitted>
  <last_update_submitted_qc>March 24, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">March 26, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Diaphragm Pacing</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Motor Neuron Disease</mesh_term>
    <mesh_term>Amyotrophic Lateral Sclerosis</mesh_term>
    <mesh_term>Sclerosis</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/NCT03034317
org study id: 20-1000-51
nct id: NCT03034317
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: Yes
is us export: Yes
The purpose of this research study is to collect more information about the use, safety, and
      effectiveness of the NeuRx DPS® in ALS patients.
Characterize the safety and benefit of on-label Humanitarian Device Exemption (HDE) use of
      DPS in patients with ALS who initiated noninvasive ventilation (NIV).
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
masking description: Open label
measure: Survival
time frame: 24 months
description: Compare the survival of patients who initiated noninvasive ventilation (NIV).and who receive DPS to controls without DPS: (1) concurrent controls; (2) historical controls (e.g., Lechtzin and PRO-ACT databases); and (3) virtual controls (Origent).
measure: Relationship between survival time and clinical features of ALS
time frame: 24 months
description: Determine whether there is a relationship between survival time and ALS onset type.
measure: Relationship between survival time and ALS assessment scores
time frame: 24 months
description: Determine whether there is a relationship between survival time and Upper Motor Neuron (UMN)/ Lower Motor Neuron (LMN) assessment scores.
measure: Relationship between survival time and onset of weakness from ALS to treatment
time frame: 24 months
measure: Relationship between survival time and ALS treatment interventions
time frame: 24 months
measure: Relationship between survival time and intraoperative strength of contraction
time frame: 24 months
measure: Characterize change in overall and respiratory function
time frame: 24 months
description: Characterize longitudinal change in overall function (ALSFRS-R) before and after treatment with DPS compared to controls.
measure: Characterize change in respiratory function
time frame: 24 months
description: Characterize longitudinal change in respiratory function (FVC, MIP) before and after treatment with DPS compared to controls.
measure: Types device and procedure-related adverse events
time frame: 24 months
measure: Frequency of device and procedure-related adverse events
time frame: 24 months
measure: Types of respiratory serious adverse events
time frame: 24 months
measure: Frequency of respiratory adverse events
time frame: 24 months
arm group label: NeuRx DPS
arm group type: Active Comparator
description: Subjects who have initiated noninvasive ventilation (NIV) and receive the NeuRx diaphragm pacing system (DPS).
arm group label: No DPS
arm group type: No Intervention
description: Subjects who have initiated noninvasive ventilation (NIV) and do not receive the DPS device.
intervention type: Device
intervention name: NeuRx DPS
description: Patients who are diagnosed with ALS, have initiated NIV and choose to have the NeuRx DPS implanted.
arm group label: NeuRx DPS
other name: Diaphragm Pacing System
criteria: 
gender: All
minimum age: 21 Years
maximum age: N/A
healthy volunteers: No
last name: Robert Miller, MD
role: Principal Investigator
affiliation: California Pacific Medical Center
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
sharing ipd: No