<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/NCT03016897</url>
  </required_header>
  <id_info>
    <org_study_id>BNI_ALS_001</org_study_id>
    <nct_id>NCT03016897</nct_id>
  </id_info>
  <brief_title>ALS Testing Through Home-based Outcome Measures</brief_title>
  <acronym>ALS AT HOME</acronym>
  <official_title>ALS Testing Through Home-based Outcome Measures (ALS AT HOME)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Jeremy Shefner</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Beth Israel Deaconess Medical Center</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Barrow Neurological Institute</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      ALS AT HOME is a single-center study of up to 150 participants being done to determine the&#xD;
      extent to which frequent sampling can improve the qualities of outcome measures collected at&#xD;
      home by study participants.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      ALS AT HOME is a single-center study of up to 150 participants being done to determine the&#xD;
      extent to which frequent sampling can improve the qualities of outcome measures collected at&#xD;
      home by study participants.&#xD;
&#xD;
      The objectives of this study are to&#xD;
&#xD;
        1. assess the extent to which frequent sampling can reduce variability for the following&#xD;
           outcome measures in ALS patients: ALSFRS-R, quantitative hand grip, pulmonary function,&#xD;
           EIM in 4 extremities, and actigraphy and voice/speech tracking;&#xD;
&#xD;
        2. assess the compliance of ALS patients in obtained outcome measures at home over the&#xD;
           course of 9 months;&#xD;
&#xD;
        3. directly compare outcome measures collected by patients with measurements obtained at&#xD;
           study sites at less frequent intervals by co-enrolling 50 patients participating in the&#xD;
           Answer ALS study; and&#xD;
&#xD;
        4. provide information to Answer ALS to more fully characterize patients in that study.&#xD;
&#xD;
      The study proposed here will assess the extent to which reducing variability of measurement&#xD;
      through frequent sampling can reduce both sample size and duration of clinical trials,&#xD;
      provide a first-of-its-kind detailed assessment of disease progression, and evaluate an&#xD;
      entirely new paradigm for patient participation in such trials without the need to be&#xD;
      geographically yoked to a clinical study center. An important component of this proposal is&#xD;
      to jointly recruit 50 patients who also participate in the Answer ALS study that intends to&#xD;
      generate 1000 IP cell lines from patients who receive ongoing assessment at 5 clinical sites.&#xD;
      The 100 participants that will be co-enrolled in Answer ALS and ALS AT HOME will perform&#xD;
      outcome measures at home in addition to having outcome measures performed at clinic sites&#xD;
      through Answer ALS participation. Therefore, the investigators will be able to directly&#xD;
      assess how the measures obtained by patients at home relate to those obtained by a trained&#xD;
      evaluator at a study center using standard metrics. The investigators will also provide all&#xD;
      data generated at home to the Answer ALS investigators, further increasing the phenotypic&#xD;
      data available for that project.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 22, 2017</start_date>
  <completion_date type="Actual">September 30, 2019</completion_date>
  <primary_completion_date type="Actual">June 28, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Pulmonary Function</measure>
    <time_frame>Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly</time_frame>
    <description>Respirometer</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Quantitative Hand Grip</measure>
    <time_frame>Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly</time_frame>
    <description>Digital Handgrip Meter</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Electrical Impedance Myography (EIM) Measurements</measure>
    <time_frame>Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly</time_frame>
    <description>Skulpt Chisel</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Actigraphy</measure>
    <time_frame>Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly</time_frame>
    <description>Activity Band</description>
  </primary_outcome>
  <primary_outcome>
    <measure>ALSFRS-R</measure>
    <time_frame>Weekly up to 9 months</time_frame>
    <description>Questionnaire</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Patient-reported Experience measures (PREMs)</measure>
    <time_frame>At Week 1, and then at 3, 6, and 9 months</time_frame>
    <description>Questionnaire</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Adverse Events</measure>
    <time_frame>Monthly up to 9 months</time_frame>
    <description>Questionnaire</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Voice/Speech Tracking</measure>
    <time_frame>Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly</time_frame>
    <description>Smartphone application</description>
  </primary_outcome>
  <number_of_groups>3</number_of_groups>
  <enrollment type="Actual">144</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Group 1</arm_group_label>
    <description>Participants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Group 2</arm_group_label>
    <description>Current participants of the Answer ALS study Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Group 3</arm_group_label>
    <description>Participants without neurological disease (controls) Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter</intervention_name>
    <description>Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter</description>
    <arm_group_label>Group 1</arm_group_label>
    <arm_group_label>Group 2</arm_group_label>
    <arm_group_label>Group 3</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        ALS patient volunteers will be invited to participate in this study as well as healthy&#xD;
        subjects for the control group.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  male or female, age 18 to 85 years old,&#xD;
&#xD;
          -  ownership of a Smart Device (phone, tablet, etc) with Bluetooth capabilities,&#xD;
&#xD;
          -  continuous internet access at home,&#xD;
&#xD;
          -  willing and able to provide informed consent in compliance with the regulatory&#xD;
             requirements through a web based interface,&#xD;
&#xD;
          -  definite, Probable, or Possible ALS by modified El Escorial criteria, as documented by&#xD;
             medical records, with duration from diagnosis 60 months or less, and&#xD;
&#xD;
          -  for the 50 participants in Group 2, participation in the Answer ALS study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  diagnosed and actively undergoing treatment for cancer, heart failure, end stage renal&#xD;
             disease, or another significant medical condition deemed by the PI to likely affect&#xD;
             the participant's ability to comply with the protocol,&#xD;
&#xD;
          -  unwilling or unable to comply with the requirements of this protocol, including the&#xD;
             presence of any condition (physical, mental, or social) that is likely to affect the&#xD;
             participant's ability to comply with the protocol,&#xD;
&#xD;
          -  any other reasons that, in the opinion of the PI, the candidate is determined to be&#xD;
             unsuitable for entry into the study.&#xD;
&#xD;
          -  Healthy volunteers who have family members with ALS&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>85 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jeremy Shefner, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Barrow Neurological Institute</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Barrow Neurological Institute</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85013</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>December 22, 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>February 6, 2023</last_update_submitted>
  <last_update_submitted_qc>February 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 8, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Barrow Neurological Institute</investigator_affiliation>
    <investigator_full_name>Jeremy Shefner</investigator_full_name>
    <investigator_title>Senior Vice President</investigator_title>
  </responsible_party>
  <keyword>ALS</keyword>
  <keyword>neurodegenerative</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>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.</ipd_description>
  </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/NCT03016897
org study id: BNI_ALS_001
nct id: NCT03016897
lead sponsor: 
collaborator: 
has dmc: No
ALS AT HOME is a single-center study of up to 150 participants being done to determine the
      extent to which frequent sampling can improve the qualities of outcome measures collected at
      home by study participants.
ALS AT HOME is a single-center study of up to 150 participants being done to determine the
      extent to which frequent sampling can improve the qualities of outcome measures collected at
      home by study participants.

      The objectives of this study are to

        1. assess the extent to which frequent sampling can reduce variability for the following
           outcome measures in ALS patients: ALSFRS-R, quantitative hand grip, pulmonary function,
           EIM in 4 extremities, and actigraphy and voice/speech tracking;

        2. assess the compliance of ALS patients in obtained outcome measures at home over the
           course of 9 months;

        3. directly compare outcome measures collected by patients with measurements obtained at
           study sites at less frequent intervals by co-enrolling 50 patients participating in the
           Answer ALS study; and

        4. provide information to Answer ALS to more fully characterize patients in that study.

      The study proposed here will assess the extent to which reducing variability of measurement
      through frequent sampling can reduce both sample size and duration of clinical trials,
      provide a first-of-its-kind detailed assessment of disease progression, and evaluate an
      entirely new paradigm for patient participation in such trials without the need to be
      geographically yoked to a clinical study center. An important component of this proposal is
      to jointly recruit 50 patients who also participate in the Answer ALS study that intends to
      generate 1000 IP cell lines from patients who receive ongoing assessment at 5 clinical sites.
      The 100 participants that will be co-enrolled in Answer ALS and ALS AT HOME will perform
      outcome measures at home in addition to having outcome measures performed at clinic sites
      through Answer ALS participation. Therefore, the investigators will be able to directly
      assess how the measures obtained by patients at home relate to those obtained by a trained
      evaluator at a study center using standard metrics. The investigators will also provide all
      data generated at home to the Answer ALS investigators, further increasing the phenotypic
      data available for that project.
observational model: Case-Control
time perspective: Prospective
measure: Change in Pulmonary Function
time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
description: Respirometer
measure: Change in Quantitative Hand Grip
time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
description: Digital Handgrip Meter
measure: Change in Electrical Impedance Myography (EIM) Measurements
time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
description: Skulpt Chisel
measure: Change in Actigraphy
time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
description: Activity Band
measure: ALSFRS-R
time frame: Weekly up to 9 months
description: Questionnaire
measure: Change in Patient-reported Experience measures (PREMs)
time frame: At Week 1, and then at 3, 6, and 9 months
description: Questionnaire
measure: Adverse Events
time frame: Monthly up to 9 months
description: Questionnaire
measure: Change in Voice/Speech Tracking
time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
description: Smartphone application
arm group label: Group 1
description: Participants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
arm group label: Group 2
description: Current participants of the Answer ALS study Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
arm group label: Group 3
description: Participants without neurological disease (controls) Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
intervention type: Other
intervention name: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
description: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
arm group label: Group 1
arm group label: Group 2
arm group label: Group 3
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: 85 Years
healthy volunteers: Accepts Healthy Volunteers
last name: Jeremy Shefner, MD
role: Principal Investigator
affiliation: Barrow Neurological Institute
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Barrow Neurological Institute
investigator full name: Jeremy Shefner
investigator title: Senior Vice President
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
sharing ipd: Yes
ipd description: IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.