<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/NCT01674036</url>
  </required_header>
  <id_info>
    <org_study_id>GZFD00111/GZFD00211</org_study_id>
    <secondary_id>TDU12766/FED12767</secondary_id>
    <nct_id>NCT01674036</nct_id>
  </id_info>
  <brief_title>Safety, Tolerability and Pharmacokinetics of Genz-682452 in Healthy Men</brief_title>
  <official_title>A Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics of Ascending Single Oral Doses of Genz-682452 With a Pilot Investigation of Food Effect in Healthy Adult Male 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>
      The objectives of this study are to assess the tolerability, safety and pharmacokinetic&#xD;
      parameters of Genz-682452 in healthy adult males, in two parts.&#xD;
&#xD;
      Part 1 (GZFD00111TDU12766): double-blind, randomized, placebo-controlled, sequential&#xD;
      ascending single dose study;&#xD;
&#xD;
      Part 2 (GZFD00211FED12767): open-label, randomized, 2-sequence, 2-period, 2-treatment&#xD;
      crossover study with a minimum wash-out period; to obtain preliminary information on the&#xD;
      pharmacokinetics, tolerability and safety of Genz-682452 after single oral doses in fed and&#xD;
      fasted conditions.&#xD;
&#xD;
      Subjects are not allowed to participate in more than 1 part of the study.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>August 2012</start_date>
  <completion_date type="Actual">January 2013</completion_date>
  <primary_completion_date type="Actual">January 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>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of participants with adverse events in Part 1</measure>
    <time_frame>Up to 4 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants with adverse events in Part 2</measure>
    <time_frame>Up to 5 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics Plasma Parameters for Part 1 as measured by Cmax, tmax, AUC last, AUC, t1/2z, Vss/F, CL/F</measure>
    <time_frame>Day 1 through Day 5</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics Urine Parameters for Part 1 as measured by assessment of Genz-682452 urinary excretion, Ae0-t, fe0-t</measure>
    <time_frame>Day 1 through Day 5</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics Plasma Parameters for Part 2 as measured by Cmax, tmax, tlag, AUC last, AUC, t1/2z</measure>
    <time_frame>Day 1 to Day 3</time_frame>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">63</enrollment>
  <condition>Healthy Volunteer</condition>
  <arm_group>
    <arm_group_label>Part 1 (GZFD00111/TDU12766): Genz-682452</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive a single oral dose of Genz-682452. Six ascending single doses and an optional seventh dose under fasted conditions will be used.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 1 (GZFD00111/TDU12766): Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants will receive a single oral dose of placebo.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 2 (GZFD00211/FED12767): Genz-682452</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive two single doses of Genz-682452 separated by a 7-day wash-out period, one dose given under fed (standardized high-fat breakfast) and one under fasted conditions. The dose will be based on the blind review of the safety/tolerability/pharmacokinetic data of single dose level cohorts in Part 1.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Genz-682452</intervention_name>
    <description>Capsules for oral administration.</description>
    <arm_group_label>Part 1 (GZFD00111/TDU12766): Genz-682452</arm_group_label>
    <arm_group_label>Part 2 (GZFD00211/FED12767): Genz-682452</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo to Genz-682452</description>
    <arm_group_label>Part 1 (GZFD00111/TDU12766): Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Body weight between 50.0 and 100.0 kg, inclusive, body mass index between 18.0 and&#xD;
             32.0 kg/m^2, inclusive.&#xD;
&#xD;
          -  Certified as healthy by a comprehensive clinical assessment (detailed medical history&#xD;
             and complete physical examination).&#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, or infectious disease, or&#xD;
             signs of acute illness.&#xD;
&#xD;
          -  Frequent headaches and/or migraine, recurrent nausea and/or vomiting.&#xD;
&#xD;
          -  Blood donation, greater than 100 mL, within 2 months before inclusion.&#xD;
&#xD;
          -  Presence or history of drug hypersensitivity, or allergic disease diagnosed and&#xD;
             treated by a physician.&#xD;
&#xD;
          -  History or presence of drug or alcohol abuse.&#xD;
&#xD;
          -  Unable to stop smoking cigarettes during the study.&#xD;
&#xD;
          -  Excessive consumption of beverages containing xanthine bases.&#xD;
      </textblock>
    </criteria>
    <gender>Male</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>August 24, 2012</study_first_submitted>
  <study_first_submitted_qc>August 24, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 28, 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/NCT01674036
org study id: GZFD00111/GZFD00211
secondary id: TDU12766/FED12767
nct id: NCT01674036
lead sponsor: 
has dmc: No
The objectives of this study are to assess the tolerability, safety and pharmacokinetic
      parameters of Genz-682452 in healthy adult males, in two parts.

      Part 1 (GZFD00111TDU12766): double-blind, randomized, placebo-controlled, sequential
      ascending single dose study;

      Part 2 (GZFD00211FED12767): open-label, randomized, 2-sequence, 2-period, 2-treatment
      crossover study with a minimum wash-out period; to obtain preliminary information on the
      pharmacokinetics, tolerability and safety of Genz-682452 after single oral doses in fed and
      fasted conditions.

      Subjects are not allowed to participate in more than 1 part of the study.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Number of participants with adverse events in Part 1
time frame: Up to 4 weeks
measure: Number of participants with adverse events in Part 2
time frame: Up to 5 weeks
measure: Pharmacokinetics Plasma Parameters for Part 1 as measured by Cmax, tmax, AUC last, AUC, t1/2z, Vss/F, CL/F
time frame: Day 1 through Day 5
measure: Pharmacokinetics Urine Parameters for Part 1 as measured by assessment of Genz-682452 urinary excretion, Ae0-t, fe0-t
time frame: Day 1 through Day 5
measure: Pharmacokinetics Plasma Parameters for Part 2 as measured by Cmax, tmax, tlag, AUC last, AUC, t1/2z
time frame: Day 1 to Day 3
arm group label: Part 1 (GZFD00111/TDU12766): Genz-682452
arm group type: Experimental
description: Participants will receive a single oral dose of Genz-682452. Six ascending single doses and an optional seventh dose under fasted conditions will be used.
arm group label: Part 1 (GZFD00111/TDU12766): Placebo
arm group type: Placebo Comparator
description: Participants will receive a single oral dose of placebo.
arm group label: Part 2 (GZFD00211/FED12767): Genz-682452
arm group type: Experimental
description: Participants will receive two single doses of Genz-682452 separated by a 7-day wash-out period, one dose given under fed (standardized high-fat breakfast) and one under fasted conditions. The dose will be based on the blind review of the safety/tolerability/pharmacokinetic data of single dose level cohorts in Part 1.
intervention type: Drug
intervention name: Genz-682452
description: Capsules for oral administration.
arm group label: Part 1 (GZFD00111/TDU12766): Genz-682452
arm group label: Part 2 (GZFD00211/FED12767): Genz-682452
intervention type: Drug
intervention name: Placebo
description: Placebo to Genz-682452
arm group label: Part 1 (GZFD00111/TDU12766): Placebo
criteria: 
gender: Male
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