<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/NCT03769454</url>
  </required_header>
  <id_info>
    <org_study_id>PP-001-1101</org_study_id>
    <nct_id>NCT03769454</nct_id>
  </id_info>
  <brief_title>A Phase I Study of PP-001 Eye Drops in Healthy Adult Volunteers</brief_title>
  <official_title>A Phase I Safety and Tolerability Study of PP-001 Eye Drops in Healthy Adult Volunteers</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Panoptes Pharma GmbH</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Panoptes Pharma GmbH</source>
  <oversight_info>
    <has_dmc>Yes</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>
      In this study, PP-001 eye drops are assessed for safety and tolerability in healthy, adult&#xD;
      volunteers. PP-001 is a novel, anti-inflammatory small molecule that inhibits a specific&#xD;
      enzyme (Dihydroorotate Dehydrogenase).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      In this prospective, single-centre, double blind (within each cohort), placebo controlled,&#xD;
      randomised study, safety and tolerability of PP-001 eye drops is assessed in healthy, adult&#xD;
      volunteers. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase&#xD;
      (DHODH) with anti-inflammatory properties. PP-001 eye drops will be administered up to 4&#xD;
      times per day over a period of 13 days in ascending doses. Participants will be monitored for&#xD;
      safety and tolerability evaluation of the study drug.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">November 15, 2018</start_date>
  <completion_date type="Anticipated">January 2021</completion_date>
  <primary_completion_date type="Anticipated">December 2020</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>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Evaluation of safety and tolerability by determining treatment emergent adverse events</measure>
    <time_frame>28 days</time_frame>
    <description>To assess safety and tolerability of ascending, multiple daily doses of PP-001 eye drops in healthy, adult volunteers</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood</measure>
    <time_frame>1 and 12 days</time_frame>
    <description>To evaluate the pharmacokinetics (Cmax) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood</measure>
    <time_frame>1 and 12 days</time_frame>
    <description>To evaluate the pharmacokinetics (AUC) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation</description>
  </secondary_outcome>
  <number_of_arms>6</number_of_arms>
  <enrollment type="Anticipated">24</enrollment>
  <condition>Keratoconjunctivitis</condition>
  <arm_group>
    <arm_group_label>PP-001 low dose group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo low dose group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>PP-001 mid dose group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo mid dose group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>PP-001 high dose group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo high dose group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>PP-001</intervention_name>
    <description>PP-001 eye drops</description>
    <arm_group_label>PP-001 high dose group</arm_group_label>
    <arm_group_label>PP-001 low dose group</arm_group_label>
    <arm_group_label>PP-001 mid dose group</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo eye drops</description>
    <arm_group_label>Placebo high dose group</arm_group_label>
    <arm_group_label>Placebo low dose group</arm_group_label>
    <arm_group_label>Placebo mid dose group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  male or female healthy volunteers 18 - 64 years of age&#xD;
&#xD;
          -  good general state of health and participants must not have a diagnosis of any eye&#xD;
             disease that could effect the pharmacokinetics of PP-001&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  participation in other clinical trials within 30 days prior to dosing start (ocular&#xD;
             and non-ocular clinical trials)&#xD;
&#xD;
          -  pregnant or nursing patients&#xD;
&#xD;
          -  regular use of any ocular agents within 60 days prior to start dosing&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>64 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University Hospital Vienna</name>
      <address>
        <city>Vienna</city>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
  </location_countries>
  <verification_date>March 2020</verification_date>
  <study_first_submitted>December 6, 2018</study_first_submitted>
  <study_first_submitted_qc>December 6, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 7, 2018</study_first_posted>
  <last_update_submitted>March 10, 2020</last_update_submitted>
  <last_update_submitted_qc>March 10, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">March 11, 2020</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>Keratoconjunctivitis</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/NCT03769454
org study id: PP-001-1101
nct id: NCT03769454
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
In this study, PP-001 eye drops are assessed for safety and tolerability in healthy, adult
      volunteers. PP-001 is a novel, anti-inflammatory small molecule that inhibits a specific
      enzyme (Dihydroorotate Dehydrogenase).
In this prospective, single-centre, double blind (within each cohort), placebo controlled,
      randomised study, safety and tolerability of PP-001 eye drops is assessed in healthy, adult
      volunteers. PP-001 is a novel small molecule inhibitor of Dihydroorotate Dehydrogenase
      (DHODH) with anti-inflammatory properties. PP-001 eye drops will be administered up to 4
      times per day over a period of 13 days in ascending doses. Participants will be monitored for
      safety and tolerability evaluation of the study drug.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Evaluation of safety and tolerability by determining treatment emergent adverse events
time frame: 28 days
description: To assess safety and tolerability of ascending, multiple daily doses of PP-001 eye drops in healthy, adult volunteers
measure: Evaluation of Peak Plasma Concentration (Cmax) in peripheral blood
time frame: 1 and 12 days
description: To evaluate the pharmacokinetics (Cmax) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation
measure: Evaluation of area under the plasma concentration versus time curve (AUC) in peripheral blood
time frame: 1 and 12 days
description: To evaluate the pharmacokinetics (AUC) of PP-001 in healthy, adult volunteers following single and multiple daily ocular instillation
arm group label: PP-001 low dose group
arm group type: Experimental
arm group label: Placebo low dose group
arm group type: Placebo Comparator
arm group label: PP-001 mid dose group
arm group type: Experimental
arm group label: Placebo mid dose group
arm group type: Placebo Comparator
arm group label: PP-001 high dose group
arm group type: Experimental
arm group label: Placebo high dose group
arm group type: Placebo Comparator
intervention type: Drug
intervention name: PP-001
description: PP-001 eye drops
arm group label: PP-001 high dose group
arm group label: PP-001 low dose group
arm group label: PP-001 mid dose group
intervention type: Other
intervention name: Placebo
description: Placebo eye drops
arm group label: Placebo high dose group
arm group label: Placebo low dose group
arm group label: Placebo mid dose group
criteria: 
gender: All
minimum age: 18 Years
maximum age: 64 Years
healthy volunteers: Accepts Healthy Volunteers
facility: 
country: Austria
responsible party type: Sponsor
mesh term: Keratoconjunctivitis