<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/NCT02988297</url>
  </required_header>
  <id_info>
    <org_study_id>06.2.1.H6</org_study_id>
    <nct_id>NCT02988297</nct_id>
  </id_info>
  <brief_title>Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis</brief_title>
  <official_title>Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Revalesio Corporation</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Revalesio Corporation</source>
  <oversight_info>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine whether nebulized RNS60 is effective in the&#xD;
      treatment of amyotrophic lateral sclerosis (ALS).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Not yet recruiting</overall_status>
  <start_date type="Anticipated">May 2024</start_date>
  <completion_date type="Anticipated">June 2026</completion_date>
  <primary_completion_date type="Anticipated">June 2026</primary_completion_date>
  <phase>Phase 2</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>Treatment</primary_purpose>
    <masking>Triple (Participant, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>ALS functional rating scale-revised (ALSFRS-R) score</measure>
    <time_frame>24 weeks</time_frame>
    <description>The mean change of the ALSFRS-R total score</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Deaths or tracheostomies</measure>
    <time_frame>28 weeks</time_frame>
    <description>The cumulative proportion of deaths or tracheostomies</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of regulatory T cells (Treg)</measure>
    <time_frame>24 weeks</time_frame>
    <description>The mean change in the proportion of Tregs</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Slow vital capacity (SVC)</measure>
    <time_frame>24 weeks</time_frame>
    <description>The mean change of the SVC score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>ALS assessment questionnaire (ALSAQ-40) score</measure>
    <time_frame>24 weeks</time_frame>
    <description>The mean change of the ALS assessment questionnaire (ALSAQ-40) score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Adverse events (AEs)</measure>
    <time_frame>28 weeks</time_frame>
    <description>The mean number of AEs</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">140</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>RNS60</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Nebulized RNS60 will be administered by daily inhalation for 24 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Nebulized Placebo will be administered by daily inhalation for 24 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>RNS60</intervention_name>
    <description>Nebulized RNS60 will be administered by daily inhalation for 24 weeks.</description>
    <arm_group_label>RNS60</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Nebulized Placebo will be administered by daily inhalation for 24 weeks.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Probable, probable-laboratory supported, or definite ALS, either sporadic or familial&#xD;
             according to modified El Escorial criteria&#xD;
&#xD;
          -  Disease duration &lt; 3 years&#xD;
&#xD;
          -  Age 18 to 80&#xD;
&#xD;
          -  Able to provide informed consent and to comply with study procedures&#xD;
&#xD;
          -  Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of&#xD;
             riluzole for at least 30 days, prior to screening (riluzole-naïve participants are&#xD;
             permitted in the study)&#xD;
&#xD;
          -  Women must not be lactating or able to become pregnant (e.g. post menopausal,&#xD;
             surgically sterile, or using adequate birth control) for the duration of the study,&#xD;
             and for 3 months after study completion&#xD;
&#xD;
          -  Men should practice contraception for the duration of the study and for 3 months after&#xD;
             completion&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of a motor neuron disease other than ALS (e.g., primary lateral sclerosis,&#xD;
             progressive muscular atrophy, progressive bulbar palsy)&#xD;
&#xD;
          -  Clinically significant unstable medical condition (other than ALS) that would pose a&#xD;
             risk to the participant if they were to participate in the study or that would impact&#xD;
             survival or functional disability in the next 12 months&#xD;
&#xD;
          -  Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine&#xD;
             aminotransferase (ALT) &gt; 3 times the upper limit of the normal&#xD;
&#xD;
          -  Renal insufficiency (Glomerular Filtration Rate &lt; 60)&#xD;
&#xD;
          -  Active pulmonary disease&#xD;
&#xD;
          -  Prior poor compliance with an inhalation device&#xD;
&#xD;
          -  The presence of unstable psychiatric disease, cognitive impairment, or dementia that&#xD;
             would impair ability of the participant to provide informed consent, according to&#xD;
             judgment by the principal investigator.&#xD;
&#xD;
          -  History of human immunodeficiency virus (HIV) infection, clinically significant&#xD;
             chronic hepatitis, or other active infection.&#xD;
&#xD;
          -  Active participation in another ALS clinical trial within 30 days of the Screening&#xD;
             Visit&#xD;
&#xD;
          -  Other experimental treatments or anti-inflammatory/immunomodulatory medications used&#xD;
             in the preceding 3 months&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>December 7, 2016</study_first_submitted>
  <study_first_submitted_qc>December 7, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 9, 2016</study_first_posted>
  <last_update_submitted>September 14, 2023</last_update_submitted>
  <last_update_submitted_qc>September 14, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 15, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <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>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>RNS60</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/NCT02988297
org study id: 06.2.1.H6
nct id: NCT02988297
lead sponsor: 
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to determine whether nebulized RNS60 is effective in the
      treatment of amyotrophic lateral sclerosis (ALS).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Investigator, Outcomes Assessor)
measure: ALS functional rating scale-revised (ALSFRS-R) score
time frame: 24 weeks
description: The mean change of the ALSFRS-R total score
measure: Deaths or tracheostomies
time frame: 28 weeks
description: The cumulative proportion of deaths or tracheostomies
measure: Proportion of regulatory T cells (Treg)
time frame: 24 weeks
description: The mean change in the proportion of Tregs
measure: Slow vital capacity (SVC)
time frame: 24 weeks
description: The mean change of the SVC score
measure: ALS assessment questionnaire (ALSAQ-40) score
time frame: 24 weeks
description: The mean change of the ALS assessment questionnaire (ALSAQ-40) score
measure: Adverse events (AEs)
time frame: 28 weeks
description: The mean number of AEs
arm group label: RNS60
arm group type: Experimental
description: Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
arm group label: Placebo
arm group type: Placebo Comparator
description: Nebulized Placebo will be administered by daily inhalation for 24 weeks.
intervention type: Drug
intervention name: RNS60
description: Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
arm group label: RNS60
intervention type: Drug
intervention name: Placebo
description: Nebulized Placebo will be administered by daily inhalation for 24 weeks.
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
mesh term: RNS60