<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/NCT01710826</url>
  </required_header>
  <id_info>
    <org_study_id>GZFD00311</org_study_id>
    <secondary_id>TDR12768</secondary_id>
    <nct_id>NCT01710826</nct_id>
  </id_info>
  <brief_title>A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Genz-682452 in Healthy Volunteers</brief_title>
  <official_title>A Single-center, Double-blind, Randomized, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ascending 14-day Repeated Oral Doses of Genz-682452 in Healthy Male and Female Subjects</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Genzyme, a Sanofi Company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sanofi</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      To assess in healthy adult subjects:&#xD;
&#xD;
        -  The tolerability and safety of ascending repeated oral doses of Genz-682452.&#xD;
&#xD;
        -  The pharmacokinetic parameters of Genz-682452 after ascending repeated oral doses.&#xD;
&#xD;
        -  The pharmacodynamics of Genz-682452 after ascending repeated oral doses.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>October 2012</start_date>
  <completion_date type="Actual">February 2013</completion_date>
  <primary_completion_date type="Actual">February 2013</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>Basic Science</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Participants with Treatment-Emergent Adverse Events (TEAEs)</measure>
    <time_frame>Day 1 through Day 14</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics as measure by plasma parameters</measure>
    <time_frame>Day 1 through Day 14</time_frame>
    <description>Maximum plasma concentration (Cmax), Plasma Concentration Observed Just Before Treatment Administration During Repeated Dosing (Ctrough), First time to reach Cmax (Tmax), Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval 0-23 hours (AUC0-24), Terminal Half-Life (t1/2z), Apparent Total Body Clearance of Genz-682452 at steady state (CLss/F), plasma 4 beta-hydroxycholesterol (4β-HC)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3)</measure>
    <time_frame>Day 1, through Day 14</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory)</measure>
    <time_frame>Day 1 through Day 14</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">36</enrollment>
  <condition>Healthy</condition>
  <arm_group>
    <arm_group_label>Genz-682452</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>This study will include three cohorts for doses of Genz-682452: Dose 1, Dose 2, Dose 3. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo comparator taken by participants randomized to the placebo arm in each of the three cohorts. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Genz-682452</intervention_name>
    <description>Capsules for oral administration.</description>
    <arm_group_label>Genz-682452</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo capsules matching the Genz-682452 capsules.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Body weight between 50.0 and 95.0 kg, inclusive, if male and between 40.0 and 85.0 kg&#xD;
             inclusive, if female, body mass index between 18.0 and 30.0 kg/m^2, inclusive.&#xD;
&#xD;
          -  Certified as healthy by a comprehensive clinical assessment.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any history or presence of clinically relevant cardiovascular, pulmonary,&#xD;
             gastrointestinal, hepatic, renal, metabolic, hematological, neurological,&#xD;
             osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or&#xD;
             infectious disease, or signs of acute illness.&#xD;
&#xD;
          -  Frequent headaches and/or migraine, recurrent nausea and/or vomiting.&#xD;
&#xD;
          -  Blood donation, any volume, within 2 months before inclusion.&#xD;
&#xD;
          -  Symptomatic postural hypotension.&#xD;
&#xD;
          -  History or presence of drug or alcohol abuse.&#xD;
&#xD;
          -  Positive result on any of the following tests: hepatitis B surface (HBs Ag) antigen,&#xD;
             antihepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2&#xD;
             antibodies (anti-HIV1 and anti HIV2 Ab).&#xD;
&#xD;
          -  Positive result on urine drug screen.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>45 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Monitor</last_name>
    <role>Study Director</role>
    <affiliation>Genzyme, a Sanofi Company</affiliation>
  </overall_official>
  <location>
    <facility>
      <address>
        <city>Austin</city>
        <state>Texas</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2014</verification_date>
  <study_first_submitted>October 17, 2012</study_first_submitted>
  <study_first_submitted_qc>October 17, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 19, 2012</study_first_posted>
  <last_update_submitted>March 17, 2014</last_update_submitted>
  <last_update_submitted_qc>March 17, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">March 19, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Venglustat</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/NCT01710826
org study id: GZFD00311
secondary id: TDR12768
nct id: NCT01710826
lead sponsor: 
has dmc: No
To assess in healthy adult subjects:

        -  The tolerability and safety of ascending repeated oral doses of Genz-682452.

        -  The pharmacokinetic parameters of Genz-682452 after ascending repeated oral doses.

        -  The pharmacodynamics of Genz-682452 after ascending repeated oral doses.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Basic Science
masking: Double (Participant, Investigator)
measure: Participants with Treatment-Emergent Adverse Events (TEAEs)
time frame: Day 1 through Day 14
measure: Pharmacokinetics as measure by plasma parameters
time frame: Day 1 through Day 14
description: Maximum plasma concentration (Cmax), Plasma Concentration Observed Just Before Treatment Administration During Repeated Dosing (Ctrough), First time to reach Cmax (Tmax), Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval 0-23 hours (AUC0-24), Terminal Half-Life (t1/2z), Apparent Total Body Clearance of Genz-682452 at steady state (CLss/F), plasma 4 beta-hydroxycholesterol (4β-HC)
measure: Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3)
time frame: Day 1, through Day 14
measure: Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory)
time frame: Day 1 through Day 14
arm group label: Genz-682452
arm group type: Experimental
description: This study will include three cohorts for doses of Genz-682452: Dose 1, Dose 2, Dose 3. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo comparator taken by participants randomized to the placebo arm in each of the three cohorts. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.
intervention type: Biological
intervention name: Genz-682452
description: Capsules for oral administration.
arm group label: Genz-682452
intervention type: Biological
intervention name: Placebo
description: Placebo capsules matching the Genz-682452 capsules.
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 45 Years
healthy volunteers: Accepts Healthy Volunteers
last name: Medical Monitor
role: Study Director
affiliation: Genzyme, a Sanofi Company
facility: 
country: United States
responsible party type: Sponsor
mesh term: Venglustat