<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/NCT02228460</url>
  </required_header>
  <id_info>
    <org_study_id>ACT13739</org_study_id>
    <secondary_id>2013-005324-41</secondary_id>
    <secondary_id>U1111-1152-1456</secondary_id>
    <nct_id>NCT02228460</nct_id>
  </id_info>
  <brief_title>Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease</brief_title>
  <official_title>A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease</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>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Primary Objective:&#xD;
&#xD;
      To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy&#xD;
      of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants&#xD;
      diagnosed with Fabry disease.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The total duration of study per participant was 7 to 8 months for participants who entered a&#xD;
      planned extension study and approximately 13 to 14 months for participants who did not enter&#xD;
      a planned extension study. A 2-year extension study was planned for eligible participants.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 2014</start_date>
  <completion_date type="Actual">September 2016</completion_date>
  <primary_completion_date type="Actual">September 2016</primary_completion_date>
  <phase>Phase 2</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>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Plasma GL-3 Concentration at Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26</measure>
    <time_frame>Baseline, Week 26</time_frame>
    <description>Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Treatment Emergent Adverse Events (TEAEs)</measure>
    <time_frame>From Baseline up to 212 days</time_frame>
    <description>Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671</measure>
    <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
    <description>Maximum plasma concentration observed for study drug was reported.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671</measure>
    <time_frame>Predose on Days 14, 28, 56, 84, 126, and 182</time_frame>
    <description>Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671</measure>
    <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
    <description>Tmax was defined as time to reach maximum plasma concentration of study drug.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671</measure>
    <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
    <description>Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Terminal Half-life (t1/2z) of GZ/SAR402671</measure>
    <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
    <description>Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)</measure>
    <time_frame>Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182</time_frame>
    <description>Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State</measure>
    <time_frame>Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182</time_frame>
    <description>Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)</measure>
    <time_frame>0-24 hours on Day 182</time_frame>
    <description>Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)</measure>
    <time_frame>0-24 hours on Day 182</time_frame>
    <description>fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours</measure>
    <time_frame>0-24 hours on Day 182</time_frame>
    <description>CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">11</enrollment>
  <condition>Fabry Disease</condition>
  <arm_group>
    <arm_group_label>GZ/SAR402671</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>GZ/SAR402671 15 milligram (mg) once daily orally for 26 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GZ/SAR402671</intervention_name>
    <description>Pharmaceutical form: Capsule; Route of administration: Oral</description>
    <arm_group_label>GZ/SAR402671</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion criteria:&#xD;
&#xD;
          -  The participant was greater than equal to (&gt;=) 18 years of age and less than (&lt;) 50&#xD;
             years of age.&#xD;
&#xD;
          -  The participant was male.&#xD;
&#xD;
          -  The participant had provided a signed informed consent.&#xD;
&#xD;
          -  The participant had a confirmed diagnosis of Fabry disease as documented by leukocyte&#xD;
             α- Galactosidase A (αGAL) activity of &lt;4 nanomole/hour/milligram (nmol/hr/mg)&#xD;
             leukocyte (preferred assay; results from a central laboratory) or plasma αGAL &lt;1.5&#xD;
             nanomole/hour/milliliter (nmol/hr/mL) (results from a central laboratory).&#xD;
&#xD;
          -  The participant had a plasma globotriaosylsphingosine (lyso-GL3) &gt;=65 nanogram per&#xD;
             milliliter (ng/mL).&#xD;
&#xD;
          -  The participant had never been treated with a Fabry disease-specific treatment.&#xD;
&#xD;
          -  If the participant was on renin-angiotensin-aldosterone system (RAAS) blockers and&#xD;
             antidepressants, the dose should be stable (i.e., prescribed dose and frequency) for&#xD;
             at least the immediate 3 months prior to screening.&#xD;
&#xD;
        Exclusion criteria:&#xD;
&#xD;
          -  The participant had an estimated glomerular filtration rate (eGFR) &lt;60 mL/min/1.73 m^2&#xD;
             using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI).&#xD;
&#xD;
          -  The participant had a median urine protein/creatinine ratio (PCR) &gt;=0.5 gram per gram&#xD;
             (g/g) (median of 3 overnight urine collections. Collection of each of the 3 samples&#xD;
             must occur between 4 and 7 days of each other, and all samples must be collected&#xD;
             within a 15 day period). All 3 samples must be collected regardless of the results and&#xD;
             results available prior to Day 1.&#xD;
&#xD;
          -  The participant had undergone a kidney transplant.&#xD;
&#xD;
          -  The participant had either active or a history of clinically significant organic&#xD;
             disease (with the exception of the symptoms related to Fabry disease), including&#xD;
             clinically significant cardiovascular, hepatic, pulmonary, hematologic, neurological&#xD;
             or renal disease, or other medical condition, serious inter-current illness, or&#xD;
             extenuating circumstances that, in the opinion of the Investigator, would preclude&#xD;
             participation in the trial.&#xD;
&#xD;
          -  The participant had abnormal liver function (serum total bilirubin &gt; the upper limit&#xD;
             of normal, or serum alanine aminotransferase ([ALT] and aspartate aminotransferase&#xD;
             [AST] &gt;2.0 times the upper limit of normal).&#xD;
&#xD;
          -  The participant had, according to World Health Organization (WHO) grading a cortical&#xD;
             cataract (COR) &gt; one-quarter of the lens circumference (Grade COR-2) or a posterior&#xD;
             subcapsular cataract (PSC) &gt;2 millimeter (mm) (Grade PSC-2). Participants with nuclear&#xD;
             cataracts were not excluded.&#xD;
&#xD;
          -  The participant was currently receiving potentially cataractogenic medications.&#xD;
&#xD;
          -  The participant had received strong or moderate inducers or inhibitors of Cytochrome&#xD;
             P450 3A4 (CYP3A4) per Food and Drug Administration (FDA) classification within 14 days&#xD;
             prior to enrollment or within 5 times the elimination half-life or PD half-life of the&#xD;
             medication, whichever is longer.&#xD;
&#xD;
          -  The participant was scheduled for in-patient hospitalization, including elective&#xD;
             surgery, during the study.&#xD;
&#xD;
          -  The participant had a positive result on any of the following tests: hepatitis B&#xD;
             surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) antibodies, anti-human&#xD;
             immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 Ab). Participants&#xD;
             with a positive hepatitis B surface antibody (HBsAb) test with a history of prior&#xD;
             hepatitis B immunization were eligible if other criteria met (i.e., negative tests&#xD;
             for: HBsAg, hepatitis B core antibody [HBcAb], and hepatitis C virus antibody&#xD;
             [HCVAb]).&#xD;
&#xD;
          -  The participant had participated in a study involving an investigational drug within&#xD;
             the past 30 days of the start of the trial.&#xD;
&#xD;
          -  The participant was unwilling to comply with the requirements of the protocol.&#xD;
&#xD;
          -  The participant was a sexually active man who was not willing to use 2 forms of birth&#xD;
             control including a barrier method during the study until 6 weeks after the last&#xD;
             treatment with investigational medicinal product (IMP).&#xD;
&#xD;
          -  The participant had a history or ongoing clinically significant cardiac arrhythmia,&#xD;
             defined as either atrial fibrillation, sustained or non-sustained ventricular&#xD;
             tachycardia.&#xD;
&#xD;
          -  The participant had any contraindication to magnetic resonance imaging (MRI).&#xD;
&#xD;
          -  The participant had one of the following central nervous system exclusion criteria:&#xD;
&#xD;
               -  Acute stroke, within 3 months of the screening visit.&#xD;
&#xD;
               -  History of seizures.&#xD;
&#xD;
        The above information is not intended to contain all considerations relevant to a&#xD;
        participant's potential participation in a clinical trial.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>49 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Sciences &amp; Operations</last_name>
    <role>Study Director</role>
    <affiliation>Sanofi</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Investigational Site Number 840002</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840003</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 840001</name>
      <address>
        <city>Fairfax</city>
        <state>Virginia</state>
        <zip>22030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 250001</name>
      <address>
        <city>Garches</city>
        <zip>92380</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 616001</name>
      <address>
        <city>Warszawa</city>
        <zip>04-730</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 643002</name>
      <address>
        <city>Moscow</city>
        <zip>125167</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 826003</name>
      <address>
        <city>Birmingham</city>
        <zip>B15 2TH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 826002</name>
      <address>
        <city>Cambridge</city>
        <zip>CB2 OQQ</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Poland</country>
    <country>Russian Federation</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Czech Republic</country>
    <country>Czechia</country>
  </removed_countries>
  <verification_date>September 2016</verification_date>
  <study_first_submitted>August 27, 2014</study_first_submitted>
  <study_first_submitted_qc>August 27, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 29, 2014</study_first_posted>
  <results_first_submitted>November 26, 2019</results_first_submitted>
  <results_first_submitted_qc>November 26, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">December 17, 2019</results_first_posted>
  <disposition_first_submitted>September 5, 2017</disposition_first_submitted>
  <disposition_first_submitted_qc>September 5, 2017</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">September 11, 2017</disposition_first_posted>
  <last_update_submitted>November 26, 2019</last_update_submitted>
  <last_update_submitted_qc>November 26, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">December 17, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Venglustat</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fabry Disease</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Participants were enrolled in the study at 8 centers in 5 countries between 11 November 2014 and 06 September 2016. A total of 14 participants were screened in the study.</recruitment_details>
      <pre_assignment_details>Out of 14 screened participants, 11 participants were enrolled and treated in the study.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>GZ/SAR402671</title>
          <description>GZ/SAR402671 15 milligram (mg) once daily, orally for 26 weeks.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="11"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="9"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment.</population>
      <group_list>
        <group group_id="B1">
          <title>GZ/SAR402671</title>
          <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="11"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="26.5" spread="7.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="11"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
        <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
          <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
          <population>Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Baseline Score: 1 / Week 26 Score: 1</title>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 1 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 1</title>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>A McNemar test was used to test the treatment effect based on paired pre-treatment and post-treatment (Week 26) frequencies of skin GL-3 score grouped into the categories (0 to &lt;2; 2 to 3).</groups_desc>
            <non_inferiority_type>Other</non_inferiority_type>
            <p_value>0.3173</p_value>
            <p_value_desc>Threshold for significance at 0.05 level.</p_value_desc>
            <method>McNemar Test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium</title>
        <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium</title>
          <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.22" lower_limit="-0.73" upper_limit="0.29"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Wilcoxon signed rank test was used to assess the mean change from Baseline to Week 26 in skin GL-3 score.</groups_desc>
            <non_inferiority_type>Other</non_inferiority_type>
            <p_value>0.625</p_value>
            <p_value_desc>Threshold for significance at 0.05 level.</p_value_desc>
            <method>Wilcoxon signed rank test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Plasma GL-3 Concentration at Week 26</title>
        <description>Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Plasma GL-3 Concentration at Week 26</title>
          <description>Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>mcg/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.62" spread="1.07"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26</title>
        <description>Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26</title>
          <description>Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>ng/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-30.99" spread="22.83"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26</title>
        <description>Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26</title>
          <description>Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>mcg/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.26" spread="1.43"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
        <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
          <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Baseline Score: 1 / Week 26 Score: 1</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 1</title>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
        <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
          <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Baseline Score: 1.5 / Week 26 Score: 1.5</title>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
        <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26</title>
          <description>Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Baseline Score: 1 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Baseline Score: 2 / Week 26 Score: 2</title>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26</title>
        <description>Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.</description>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26</title>
          <description>Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.</description>
          <population>Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.</population>
          <units>mg/mmol Cr</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="8"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.25" spread="0.19"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With Treatment Emergent Adverse Events (TEAEs)</title>
        <description>Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).</description>
        <time_frame>From Baseline up to 212 days</time_frame>
        <population>Analysis was performed on safety population which included all enrolled participants who received at least 1 dose of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment Emergent Adverse Events (TEAEs)</title>
          <description>Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).</description>
          <population>Analysis was performed on safety population which included all enrolled participants who received at least 1 dose of study drug.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671</title>
        <description>Maximum plasma concentration observed for study drug was reported.</description>
        <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
        <population>Analysis was performed on PK population which included all participants for whom the primary PK data were considered sufficient and interpretable. Here, 'number analyzed' = participants with available data at specified time-point.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671</title>
          <description>Maximum plasma concentration observed for study drug was reported.</description>
          <population>Analysis was performed on PK population which included all participants for whom the primary PK data were considered sufficient and interpretable. Here, 'number analyzed' = participants with available data at specified time-point.</population>
          <units>ng/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="11"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="24.7" spread="5.89"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 182</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="192.0" spread="96.4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671</title>
        <description>Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.</description>
        <time_frame>Predose on Days 14, 28, 56, 84, 126, and 182</time_frame>
        <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671</title>
          <description>Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.</description>
          <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
          <units>ng/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 14</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="11"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="152.0" spread="68.9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 28</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="11"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="165.0" spread="66.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 56</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="182.0" spread="90.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 84</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="164.0" spread="124.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 126</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="175.0" spread="94.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 182</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="164.0" spread="89.4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671</title>
        <description>Tmax was defined as time to reach maximum plasma concentration of study drug.</description>
        <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
        <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671</title>
          <description>Tmax was defined as time to reach maximum plasma concentration of study drug.</description>
          <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
          <units>hours</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="11"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8.00" lower_limit="1.07" upper_limit="24.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 182</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.00" lower_limit="0.00" upper_limit="8.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671</title>
        <description>Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.</description>
        <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
        <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671</title>
          <description>Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.</description>
          <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
          <units>ng*hour/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="11"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="476" spread="125"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 182</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4110" spread="2090"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Terminal Half-life (t1/2z) of GZ/SAR402671</title>
        <description>Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.</description>
        <time_frame>Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)</time_frame>
        <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Terminal Half-life (t1/2z) of GZ/SAR402671</title>
          <description>Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.</description>
          <population>Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.</population>
          <units>hours</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="86.8" spread="39.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 182</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="128.0" spread="59.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)</title>
        <description>Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).</description>
        <time_frame>Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182</time_frame>
        <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)</title>
          <description>Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).</description>
          <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
          <units>mL/hour</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7490" spread="10900"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State</title>
        <description>Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.</description>
        <time_frame>Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182</time_frame>
        <population>Since the percent extrapolation of AUC for all participants was &gt;30%, AUC could not be determined and hence, Vss/F could not be calculated.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State</title>
          <description>Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.</description>
          <population>Since the percent extrapolation of AUC for all participants was &gt;30%, AUC could not be determined and hence, Vss/F could not be calculated.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)</title>
        <description>Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.</description>
        <time_frame>0-24 hours on Day 182</time_frame>
        <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)</title>
          <description>Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.</description>
          <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
          <units>mcg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3210.0" spread="1640.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)</title>
        <description>fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.</description>
        <time_frame>0-24 hours on Day 182</time_frame>
        <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)</title>
          <description>fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.</description>
          <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
          <units>percentage of dose</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="21.4" spread="10.9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours</title>
        <description>CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.</description>
        <time_frame>0-24 hours on Day 182</time_frame>
        <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>GZ/SAR402671</title>
            <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours</title>
          <description>CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.</description>
          <population>Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.</population>
          <units>mL/hour</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="925" spread="407"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Baseline up to 212 days</time_frame>
      <desc>Reported AEs are TEAEs that is AEs developed/worsened during &quot;on treatment period&quot; (from first administration through last administration of study drug + 30 days or end of study participation for participant, whichever occurs first). Analysis was performed on safety population that included all enrolled participants who received at least 1 dose of study drug.</desc>
      <group_list>
        <group group_id="E1">
          <title>GZ/SAR402671</title>
          <description>GZ/SAR402671 15 mg once daily, orally for 26 weeks.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 19.0</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Haemolysis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Floppy infant</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Depressed mood</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 19.0</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Atrial fibrillation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Tinnitus</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Blepharitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Chalazion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Eye pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Lacrimation increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Lenticular opacities</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Retinal vascular disorder</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Vision blurred</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Dry mouth</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Oesophageal discomfort</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Toothache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Feeling hot</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Influenza like illness</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Non-cardiac chest pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Ear infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Rhinitis</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Muscle strain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Wound</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Electrocardiogram T wave abnormal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Electrocardiogram abnormal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Nuclear magnetic resonance imaging brain abnormal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Vibration test abnormal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Muscle twitching</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Musculoskeletal chest pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Myalgia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Neck pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Amnesia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Hypoaesthesia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Migraine</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Paraesthesia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Sinus headache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>White matter lesion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Depressed mood</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Acne</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Angiokeratoma</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
              </event>
              <event>
                <sub_title>Hyperhidrosis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Trial Transparency Team</name_or_title>
      <organization>Sanofi</organization>
      <phone>800-633-1610 ext 1#</phone>
      <email>Contact-US@sanofi.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT02228460
org study id: ACT13739
secondary id: 2013-005324-41
secondary id: U1111-1152-1456
nct id: NCT02228460
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
Primary Objective:

      To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy
      of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants
      diagnosed with Fabry disease.
The total duration of study per participant was 7 to 8 months for participants who entered a
      planned extension study and approximately 13 to 14 months for participants who did not enter
      a planned extension study. A 2-year extension study was planned for eligible participants.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
time frame: Baseline, Week 26
description: Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
measure: Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium
time frame: Baseline, Week 26
description: Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.
measure: Change From Baseline in Plasma GL-3 Concentration at Week 26
time frame: Baseline, Week 26
description: Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.
measure: Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26
time frame: Baseline, Week 26
description: Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.
measure: Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26
time frame: Baseline, Week 26
description: Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.
measure: Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
time frame: Baseline, Week 26
description: Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
measure: Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
time frame: Baseline, Week 26
description: Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
measure: Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
time frame: Baseline, Week 26
description: Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
measure: Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26
time frame: Baseline, Week 26
description: Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
time frame: From Baseline up to 212 days
description: Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).
measure: Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671
time frame: Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
description: Maximum plasma concentration observed for study drug was reported.
measure: PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671
time frame: Predose on Days 14, 28, 56, 84, 126, and 182
description: Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.
measure: PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671
time frame: Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
description: Tmax was defined as time to reach maximum plasma concentration of study drug.
measure: PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671
time frame: Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
description: Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.
measure: PK: Terminal Half-life (t1/2z) of GZ/SAR402671
time frame: Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
description: Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.
measure: PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)
time frame: Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182
description: Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).
measure: PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State
time frame: Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182
description: Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.
measure: PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)
time frame: 0-24 hours on Day 182
description: Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.
measure: PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)
time frame: 0-24 hours on Day 182
description: fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.
measure: PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours
time frame: 0-24 hours on Day 182
description: CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.
arm group label: GZ/SAR402671
arm group type: Experimental
description: GZ/SAR402671 15 milligram (mg) once daily orally for 26 weeks.
intervention type: Drug
intervention name: GZ/SAR402671
description: Pharmaceutical form: Capsule; Route of administration: Oral
arm group label: GZ/SAR402671
criteria: 
gender: Male
minimum age: 18 Years
maximum age: 49 Years
healthy volunteers: No
last name: Clinical Sciences & Operations
role: Study Director
affiliation: Sanofi
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Poland
country: Russian Federation
country: United Kingdom
country: United States
country: Czech Republic
country: Czechia
responsible party type: Sponsor
mesh term: Fabry Disease
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 