<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/NCT02230228</url>
  </required_header>
  <id_info>
    <org_study_id>ALK001-P1001</org_study_id>
    <nct_id>NCT02230228</nct_id>
  </id_info>
  <brief_title>Phase 1 Safety Study of ALK-001 in Healthy Volunteers</brief_title>
  <official_title>A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Alkeus Pharmaceuticals, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Alkeus Pharmaceuticals, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of&#xD;
      ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>April 2014</start_date>
  <completion_date type="Actual">February 2015</completion_date>
  <primary_completion_date type="Actual">September 2014</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Basic Science</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Safety of 4-week daily dosing of ALK-001 in healthy adults.</measure>
    <time_frame>4 weeks</time_frame>
    <description>Safety evaluations combine: adverse events (AE), laboratory testing (hematology and biochemistry panels), 12-lead electrocardiograms (ECGs), vital signs, physical examination, and visual function (visual acuity and a self-reported questionnaire).</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">40</enrollment>
  <condition>Stargardt Disease</condition>
  <condition>Age-related Macular Degeneration</condition>
  <condition>Other Retinal Dystrophies</condition>
  <arm_group>
    <arm_group_label>ALK-001 capsules</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ALK-001 (No generic name)</intervention_name>
    <description>Daily, oral administration of ALK-001 capsules.</description>
    <arm_group_label>ALK-001 capsules</arm_group_label>
    <other_name>C20-D3-Retinyl Acetate</other_name>
    <other_name>C20 Deuterated vitamin A</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Main Inclusion Criteria&#xD;
&#xD;
          -  Adult between 21 and 70 years old (inclusive)&#xD;
&#xD;
          -  Healthy subject, as judged by investigator&#xD;
&#xD;
          -  Subject is able and willing to comply with study requirements (study medication&#xD;
             compliance, schedule of follow-up visits)&#xD;
&#xD;
          -  Subject has provided informed consent to participate&#xD;
&#xD;
          -  If female, subject uses a medically accepted birth control method and agrees to use&#xD;
             such a method for entire clinical trial period&#xD;
&#xD;
        Main Exclusion Criteria:&#xD;
&#xD;
          -  Subject has taken disallowed items during the past 30 days&#xD;
&#xD;
          -  Female with a positive urine pregnancy test at screening&#xD;
&#xD;
          -  Lactating woman&#xD;
&#xD;
          -  Subject has participated in any clinical study involving an investigational drug,&#xD;
             biologic or device, during the past 30 days&#xD;
&#xD;
          -  History or current evidence of gastrointestinal malabsorption&#xD;
&#xD;
          -  Subject has any other medical condition, which in the opinion of the investigator, is&#xD;
             likely to prevent compliance with protocol and/or interfere with successful collection&#xD;
             of study&#xD;
&#xD;
          -  Subject has, in the opinion of investigator, clinically significant laboratory&#xD;
             result(s), positive drug or alcohol screening, or ECG, which makes subject unsuitable&#xD;
             for participation.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>21 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Leonide Saad, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Alkeus Pharmaceuticals, Inc.</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ilyas Washington, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Columbia University</affiliation>
  </overall_official>
  <verification_date>March 2015</verification_date>
  <study_first_submitted>August 27, 2014</study_first_submitted>
  <study_first_submitted_qc>September 2, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 3, 2014</study_first_posted>
  <last_update_submitted>March 21, 2015</last_update_submitted>
  <last_update_submitted_qc>March 21, 2015</last_update_submitted_qc>
  <last_update_posted type="Estimate">March 24, 2015</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>Macular Degeneration</mesh_term>
    <mesh_term>Retinal Dystrophies</mesh_term>
    <mesh_term>Stargardt Disease</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Vitamin A</mesh_term>
    <mesh_term>Retinol acetate</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/NCT02230228
org study id: ALK001-P1001
nct id: NCT02230228
lead sponsor: 
has dmc: No
This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of
      ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Basic Science
masking: None (Open Label)
measure: Safety of 4-week daily dosing of ALK-001 in healthy adults.
time frame: 4 weeks
description: Safety evaluations combine: adverse events (AE), laboratory testing (hematology and biochemistry panels), 12-lead electrocardiograms (ECGs), vital signs, physical examination, and visual function (visual acuity and a self-reported questionnaire).
arm group label: ALK-001 capsules
arm group type: Experimental
intervention type: Drug
intervention name: ALK-001 (No generic name)
description: Daily, oral administration of ALK-001 capsules.
arm group label: ALK-001 capsules
other name: C20-D3-Retinyl Acetate
other name: C20 Deuterated vitamin A
criteria: 
gender: All
minimum age: 21 Years
maximum age: 70 Years
healthy volunteers: Accepts Healthy Volunteers
last name: Leonide Saad, PhD
role: Study Director
affiliation: Alkeus Pharmaceuticals, Inc.
last name: Ilyas Washington, PhD
role: Study Chair
affiliation: Columbia University
responsible party type: Sponsor
mesh term: Macular Degeneration
mesh term: Retinal Dystrophies
mesh term: Stargardt Disease
mesh term: Vitamin A
mesh term: Retinol acetate