<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/NCT04488705</url>
  </required_header>
  <id_info>
    <org_study_id>ET-TMEM-01</org_study_id>
    <secondary_id>2020-001080-92</secondary_id>
    <nct_id>NCT04488705</nct_id>
  </id_info>
  <brief_title>A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD002 in Healthy Subjects</brief_title>
  <official_title>A First in Human, Randomised, Double Blind, Placebo-controlled, Three-part Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses (SAD &amp; MAD) of Inhaled ETD002 in Healthy Male and Female Subjects</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Enterprise Therapeutics Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Enterprise Therapeutics Ltd</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a first in human study of ETD002 a new drug being developed for the treatment of&#xD;
      cystic fibrosis.The study is a randomised, double-blind, placebo-controlled, interventional&#xD;
      study to assess the safety and tolerability of ascending single and repeat doses of inhaled&#xD;
      ETD002 in healthy male and female subjects.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 13, 2020</start_date>
  <completion_date type="Actual">May 19, 2021</completion_date>
  <primary_completion_date type="Actual">May 19, 2021</primary_completion_date>
  <phase>Phase 1</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>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of participants reporting one or more treatment emergent adverse event (TEAE)</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants who discontinue due to an adverse event (AE)</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants who meet the markedly abnormal criteria for 12-lead ECG assessment at least once post dose</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants who meet the markedly abnormal criteria for safety laboratory assessments at least once post dose</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose</measure>
    <time_frame>Baseline to Week 8</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Plasma concentrations of ETD002</measure>
    <time_frame>Day 1 pre-dose and at multiple time points (up to 14 days) post final dose</time_frame>
    <description>Blood levels of ETD002 measured after dosing</description>
  </secondary_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">80</enrollment>
  <condition>Healthy</condition>
  <arm_group>
    <arm_group_label>Single ascending dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Repeat dose - 7 days</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Repeat dose - 14 days</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Repeat dose - 7 days with SABA</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ETD002 - single dose</intervention_name>
    <description>Single ascending doses of ETD002</description>
    <arm_group_label>Single ascending dose</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo - single dose</intervention_name>
    <description>Single doses of placebo</description>
    <arm_group_label>Single ascending dose</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ETD002 - 7 day repeat dose</intervention_name>
    <description>Twice daily doses of ETD002 for 7 days</description>
    <arm_group_label>Repeat dose - 7 days</arm_group_label>
    <arm_group_label>Repeat dose - 7 days with SABA</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo - 7 day repeat dose</intervention_name>
    <description>Twice daily doses of Placebo for 7 days</description>
    <arm_group_label>Repeat dose - 7 days</arm_group_label>
    <arm_group_label>Repeat dose - 7 days with SABA</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ETD002 - 14 day repeat dose</intervention_name>
    <description>Twice daily doses of ETD002 for 14 days</description>
    <arm_group_label>Repeat dose - 14 days</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo - 14 day repeat dose</intervention_name>
    <description>Twice daily doses of Placebo for 14 days</description>
    <arm_group_label>Repeat dose - 14 days</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Salbutamol</intervention_name>
    <description>Twice daily doses of salbutamol for 3 days (Days 5, 6 &amp; 7)</description>
    <arm_group_label>Repeat dose - 7 days with SABA</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Males &amp; females using suitable methods of contraception or females of non-childbearing&#xD;
             potential&#xD;
&#xD;
          -  Consent to study participation&#xD;
&#xD;
          -  Body weight ≥ 50 kg and body mass index within the range 19 - 30 kg/m2&#xD;
&#xD;
          -  Vital signs assessments within normal ranges&#xD;
&#xD;
          -  Healthy as determined following physical examination at screening visit&#xD;
&#xD;
          -  Spirometry readings (FEV1 and FVC) to be ≥ 80% of predicted value&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Acute or chronic illness detected at screening visit&#xD;
&#xD;
          -  Respiratory tract infection within 4 weeks of the screening visit&#xD;
&#xD;
          -  Use of prescription or OTC medication within 14 days of the screening visit&#xD;
&#xD;
          -  History of regular alcohol consumption over recommended limits within 6 months of the&#xD;
             study, or history/evidence of alcohol or drug abuse&#xD;
&#xD;
          -  Smoking or use of tobacco products within 6 months of screening&#xD;
&#xD;
          -  Abnormal blood/urine laboratory screening test results&#xD;
&#xD;
          -  Current, or history of, allergy that may be contraindicated&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>50 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>David Morris, MD</last_name>
    <role>Study Director</role>
    <affiliation>Enterprise Therapeutics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hammersmith Medicines Research</name>
      <address>
        <city>London</city>
        <zip>NW10 7EW</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>May 2021</verification_date>
  <study_first_submitted>July 23, 2020</study_first_submitted>
  <study_first_submitted_qc>July 23, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 28, 2020</study_first_posted>
  <last_update_submitted>May 20, 2021</last_update_submitted>
  <last_update_submitted_qc>May 20, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">May 21, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Cystic fibrosis</keyword>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Albuterol</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/NCT04488705
org study id: ET-TMEM-01
secondary id: 2020-001080-92
nct id: NCT04488705
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a first in human study of ETD002 a new drug being developed for the treatment of
      cystic fibrosis.The study is a randomised, double-blind, placebo-controlled, interventional
      study to assess the safety and tolerability of ascending single and repeat doses of inhaled
      ETD002 in healthy male and female subjects.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Number of participants reporting one or more treatment emergent adverse event (TEAE)
time frame: Baseline to Week 8
measure: Number of participants who discontinue due to an adverse event (AE)
time frame: Baseline to Week 8
measure: Number of participants who meet the markedly abnormal criteria for 12-lead ECG assessment at least once post dose
time frame: Baseline to Week 8
measure: Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose
time frame: Baseline to Week 8
measure: Number of participants who meet the markedly abnormal criteria for safety laboratory assessments at least once post dose
time frame: Baseline to Week 8
measure: Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose
time frame: Baseline to Week 8
measure: Plasma concentrations of ETD002
time frame: Day 1 pre-dose and at multiple time points (up to 14 days) post final dose
description: Blood levels of ETD002 measured after dosing
arm group label: Single ascending dose
arm group type: Experimental
arm group label: Repeat dose - 7 days
arm group type: Experimental
arm group label: Repeat dose - 14 days
arm group type: Experimental
arm group label: Repeat dose - 7 days with SABA
arm group type: Experimental
intervention type: Drug
intervention name: ETD002 - single dose
description: Single ascending doses of ETD002
arm group label: Single ascending dose
intervention type: Drug
intervention name: Placebo - single dose
description: Single doses of placebo
arm group label: Single ascending dose
intervention type: Drug
intervention name: ETD002 - 7 day repeat dose
description: Twice daily doses of ETD002 for 7 days
arm group label: Repeat dose - 7 days
arm group label: Repeat dose - 7 days with SABA
intervention type: Drug
intervention name: Placebo - 7 day repeat dose
description: Twice daily doses of Placebo for 7 days
arm group label: Repeat dose - 7 days
arm group label: Repeat dose - 7 days with SABA
intervention type: Drug
intervention name: ETD002 - 14 day repeat dose
description: Twice daily doses of ETD002 for 14 days
arm group label: Repeat dose - 14 days
intervention type: Drug
intervention name: Placebo - 14 day repeat dose
description: Twice daily doses of Placebo for 14 days
arm group label: Repeat dose - 14 days
intervention type: Drug
intervention name: Salbutamol
description: Twice daily doses of salbutamol for 3 days (Days 5, 6 & 7)
arm group label: Repeat dose - 7 days with SABA
criteria: 
gender: All
minimum age: 18 Years
maximum age: 50 Years
healthy volunteers: Accepts Healthy Volunteers
last name: David Morris, MD
role: Study Director
affiliation: Enterprise Therapeutics
facility: 
country: United Kingdom
responsible party type: Sponsor
mesh term: Albuterol