<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/NCT03018730</url>
  </required_header>
  <id_info>
    <org_study_id>PB-102-F30</org_study_id>
    <nct_id>NCT03018730</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)</brief_title>
  <official_title>An Open Label Study of the Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Protalix</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Chiesi Farmaceutici S.p.A.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Protalix</source>
  <oversight_info>
    <has_dmc>No</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>
      This is an open label switch over study to assess the safety and efficacy of PRX-102&#xD;
      (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a&#xD;
      stable dose (&gt;80% labelled dose/kg) for at least 6 months. Patients will be screened and&#xD;
      evaluated over 3 months while continuing on agalsidase alfa. Following the screening period,&#xD;
      the patient will be enrolled and switched from their agalsidase alfa treatment to receive&#xD;
      intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25%&#xD;
      of treated patients will be female.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Dosage and administration details:&#xD;
&#xD;
      pegunigalsidase alfa individual dose for each patient was prepared according to the patient's&#xD;
      weight. Pegunigalsidase alfa administrated at 1 mg/kg, intravenously over 3 hours, every 2&#xD;
      weeks. After the first 2 months of treatment with pegunigalsidase alfa, infusion time may be&#xD;
      reduced gradually to 1.5 hours pending patient tolerability.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">May 17, 2017</start_date>
  <completion_date type="Actual">January 9, 2020</completion_date>
  <primary_completion_date type="Actual">December 17, 2019</primary_completion_date>
  <phase>Phase 3</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>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03</measure>
    <time_frame>12 months</time_frame>
    <description>Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment</description>
  </primary_outcome>
  <other_outcome>
    <measure>eGFR</measure>
    <time_frame>Baseline and Month 12 (week 52)</time_frame>
    <description>eGFR was calculated based on the serum creatinine values that were assessed at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The absolute change in eGFR from baseline measurement at visit 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics.</description>
  </other_outcome>
  <other_outcome>
    <measure>Mean Annualised Change in eGFR (Slope)</measure>
    <time_frame>Pre-switch, Post-switch</time_frame>
    <description>The annualized change in eGFR (slope) per patient was calculated with all available eGFR values using a linear regression.&#xD;
The mean pre-switch slope is the eGFR slope during screening period and pre-infusion visit 1 (while on Replagal®).&#xD;
The mean post-switch slope is the eGFR slope during PRX-102 treatment, calculated based on eGFR vales at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 after visit 1.&#xD;
The mean change in eGFR slope from pre- to post-switch is the mean difference between the two slopes.&#xD;
eGFR was calculated based on the serum creatinine values according to the CKD-EPI formula.</description>
  </other_outcome>
  <other_outcome>
    <measure>Plasma Lyso-Gb3</measure>
    <time_frame>Baseline and month 12 (week 52)</time_frame>
    <description>Plasma Lyso-Gb3 is Fabry disease specific biomarker that can assess treatment outcome which was measured at Baseline and weeks 12, 26, 38, 52. Baseline and Month 12 (week 52) reported.</description>
  </other_outcome>
  <other_outcome>
    <measure>Number of Participants According to Protein/Creatinine Ratio (UPCR)</measure>
    <time_frame>12 months</time_frame>
    <description>Urine Protein to Creatinine Ratio (UPCR), assessed by spot urine test, at Month 12 (Week 52).Number of Participants According to Protein/Creatinine Ratio (UPCR) level</description>
  </other_outcome>
  <other_outcome>
    <measure>Left Ventricular Mass Index (g/m^2) by MRI</measure>
    <time_frame>12 months</time_frame>
    <description>Left ventricular mass was determined based on cardiac MRI data and the LVMI was indexed to patient's body surface area (g/m^2). In male patients the normal range for LVMI was 57-91 g/m^2, in female patients 47-77 g/m^2.</description>
  </other_outcome>
  <other_outcome>
    <measure>Quality of Life EQ VAS</measure>
    <time_frame>12 months</time_frame>
    <description>The EQ VAS, of the EQ 5D 5L questionnaire, records the subject's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score &quot;100&quot;) and 'Worst imaginable health state' (score &quot;0&quot;).</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">22</enrollment>
  <condition>Fabry Disease</condition>
  <arm_group>
    <arm_group_label>PRX-102</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>PRX-102 infusion every 2 weeks</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>PRX-102 (pegunigalsidase alfa)</intervention_name>
    <description>PRX-102 1 mg/kg every 2 weeks</description>
    <arm_group_label>PRX-102</arm_group_label>
    <other_name>pegunigalsidase alfa</other_name>
    <other_name>Recombinant human alpha galactosidase-A</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Age: 18-60 years&#xD;
&#xD;
          2. A documented diagnosis of Fabry disease&#xD;
&#xD;
          3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less&#xD;
             than lower limit of normal according to laboratory range and one or more of the&#xD;
             characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata&#xD;
             iii. Clustered angiokeratoma&#xD;
&#xD;
          4. Females: historical genetic test results consistent with Fabry mutations, or in the&#xD;
             case of novel mutations a first degree male relative with Fabry disease, and one or&#xD;
             more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea&#xD;
             verticillata iii. Clustered angiokeratoma&#xD;
&#xD;
          5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (&gt;80%&#xD;
             labelled dose/kg) for at least 6 months&#xD;
&#xD;
          6. eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation&#xD;
&#xD;
          7. Availability of at least 2 historical serum creatinine evaluations since starting&#xD;
             agalsidase alfa treatment and not more than 2 years&#xD;
&#xD;
          8. Female patients and male patients whose co-partners are of child-bearing potential&#xD;
             agree to use a medically acceptable method of contraception, not including the rhythm&#xD;
             method&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa&#xD;
&#xD;
          2. History of renal dialysis or transplantation&#xD;
&#xD;
          3. History of acute kidney injury in the 12 months prior to screening, including specific&#xD;
             kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic&#xD;
             renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as&#xD;
             extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive&#xD;
             nephropathy)&#xD;
&#xD;
          4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)&#xD;
             therapy initiated or dose changed in the 4 weeks prior to screening&#xD;
&#xD;
          5. Urine protein to creatinine ratio (UPCR) &gt; 0.5 g/g and not treated with an ACE&#xD;
             inhibitor or ARB&#xD;
&#xD;
          6. Known history of hypersensitivity to Gadolinium contrast agent that was not managed by&#xD;
             the use of premedication;&#xD;
&#xD;
          7. Females who are pregnant, planning to become pregnant during the study, or are breast&#xD;
             feeding&#xD;
&#xD;
          8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period&#xD;
             before screening&#xD;
&#xD;
          9. Congestive heart failure NYHA Class IV&#xD;
&#xD;
         10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before&#xD;
             screening&#xD;
&#xD;
         11. Presence of any medical, emotional, behavioral or psychological condition that, in the&#xD;
             judgment of the Investigator and/or Medical Monitor would interfere with the patient's&#xD;
             compliance with the requirements of the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Royal Melbourne Hospital</name>
      <address>
        <city>Parkville</city>
        <state>Victoria</state>
        <zip>3050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Capital Health</name>
      <address>
        <city>Halifax</city>
        <state>Nova Scotia</state>
        <zip>B3H 1V8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vseobecna fakultni nemocnice v Praze</name>
      <address>
        <city>Prague</city>
        <country>Czechia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Academisch Medisch Centrum</name>
      <address>
        <city>Amsterdam</city>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Helse Bergen HF Haukeland Universitetssykehus</name>
      <address>
        <city>Bergen</city>
        <country>Norway</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>General Hospital Slovenj Gradec</name>
      <address>
        <city>Slovenj Gradec</city>
        <zip>SI-2380</zip>
        <country>Slovenia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queen Elizabeth Hospital, Department of Neurology,</name>
      <address>
        <city>Edgbaston</city>
        <state>Birmingham</state>
        <zip>B15 2TH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Royal Free Hospital</name>
      <address>
        <city>London</city>
        <zip>NW3 2QG</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Salford Royal NHS Foundation Trust</name>
      <address>
        <city>Salford</city>
        <zip>M6 8HD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Czechia</country>
    <country>Netherlands</country>
    <country>Norway</country>
    <country>Slovenia</country>
    <country>United Kingdom</country>
  </location_countries>
  <removed_countries>
    <country>Czech Republic</country>
    <country>Germany</country>
    <country>Spain</country>
  </removed_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>January 9, 2017</study_first_submitted>
  <study_first_submitted_qc>January 10, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 12, 2017</study_first_posted>
  <results_first_submitted>June 2, 2022</results_first_submitted>
  <results_first_submitted_qc>June 2, 2022</results_first_submitted_qc>
  <results_first_posted type="Actual">June 29, 2022</results_first_posted>
  <last_update_submitted>September 11, 2023</last_update_submitted>
  <last_update_submitted_qc>September 11, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 13, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Fabry disease</keyword>
  <keyword>Enzyme-Replacement Therapy</keyword>
  <keyword>pegunigalsidase alfa</keyword>
  <keyword>PRX-102</keyword>
  <keyword>alpha galactosidase-A</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fabry Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>June 6, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/30/NCT03018730/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>April 28, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/30/NCT03018730/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>A total of 22 adult patients were planned to be included in the study; no more than 25% were planned to be female patients. In practice, 15 males and 7 females (32%) received treatment in this study; 20 patients (13 males and 7 females) completed the 12 month treatment duration.</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>PRX-102</title>
          <description>PRX-102 (pegunigalsidase alfa): PRX-102 infusion 1 mg/kg every 2 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="22"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="20"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>PRX-102</title>
          <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="22"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="22"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <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="44.0" spread="11.0"/>
                  </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="7"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Race</title>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="22"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Canada</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Netherlands</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Czechia</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Norway</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Slovenia</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Australia</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03</title>
        <description>Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment</description>
        <time_frame>12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03</title>
          <description>Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment</description>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="22"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>At least 1 TEAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 mild or moderate TEAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="19"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 severe TEAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 SAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 TEAE unrelated or unlikely related</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="19"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 TEAE related to study treatment</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 SAE related to study treatment</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 TEAE leading to discontinuation</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>At least 1 TEAE leading to death</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>eGFR</title>
        <description>eGFR was calculated based on the serum creatinine values that were assessed at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The absolute change in eGFR from baseline measurement at visit 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics.</description>
        <time_frame>Baseline and Month 12 (week 52)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>eGFR</title>
          <description>eGFR was calculated based on the serum creatinine values that were assessed at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The absolute change in eGFR from baseline measurement at visit 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics.</description>
          <units>mL/min/1.73m^2</units>
          <param>Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline eGFR</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="79.46" spread="4.92"/>
                    <measurement group_id="O2" value="75.87" spread="6.62"/>
                    <measurement group_id="O3" value="86.14" spread="6.72"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Month 12 (week 52) eGFR</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="76.91" spread="5.22"/>
                    <measurement group_id="O2" value="74.27" spread="7.15"/>
                    <measurement group_id="O3" value="81.80" spread="7.09"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change in eGFR from Baseline to Month 12 (week 52)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-2.56" spread="2.14"/>
                    <measurement group_id="O2" value="-1.60" spread="2.75"/>
                    <measurement group_id="O3" value="-4.34" spread="3.54"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Mean Annualised Change in eGFR (Slope)</title>
        <description>The annualized change in eGFR (slope) per patient was calculated with all available eGFR values using a linear regression.&#xD;
The mean pre-switch slope is the eGFR slope during screening period and pre-infusion visit 1 (while on Replagal®).&#xD;
The mean post-switch slope is the eGFR slope during PRX-102 treatment, calculated based on eGFR vales at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 after visit 1.&#xD;
The mean change in eGFR slope from pre- to post-switch is the mean difference between the two slopes.&#xD;
eGFR was calculated based on the serum creatinine values according to the CKD-EPI formula.</description>
        <time_frame>Pre-switch, Post-switch</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Mean Annualised Change in eGFR (Slope)</title>
          <description>The annualized change in eGFR (slope) per patient was calculated with all available eGFR values using a linear regression.&#xD;
The mean pre-switch slope is the eGFR slope during screening period and pre-infusion visit 1 (while on Replagal®).&#xD;
The mean post-switch slope is the eGFR slope during PRX-102 treatment, calculated based on eGFR vales at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 after visit 1.&#xD;
The mean change in eGFR slope from pre- to post-switch is the mean difference between the two slopes.&#xD;
eGFR was calculated based on the serum creatinine values according to the CKD-EPI formula.</description>
          <units>mL/min/1.73m^2/year</units>
          <param>Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>pre-switch</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-5.9" spread="1.34"/>
                    <measurement group_id="O2" value="-6.36" spread="1.89"/>
                    <measurement group_id="O3" value="-5.03" spread="1.65"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>post-switch</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.19" spread="1.77"/>
                    <measurement group_id="O2" value="-1.73" spread="2.64"/>
                    <measurement group_id="O3" value="-0.21" spread="1.47"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change in eGFR slope from pre- to post-switch</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.70" spread="2.26"/>
                    <measurement group_id="O2" value="4.63" spread="3.48"/>
                    <measurement group_id="O3" value="4.83" spread="1.09"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Plasma Lyso-Gb3</title>
        <description>Plasma Lyso-Gb3 is Fabry disease specific biomarker that can assess treatment outcome which was measured at Baseline and weeks 12, 26, 38, 52. Baseline and Month 12 (week 52) reported.</description>
        <time_frame>Baseline and month 12 (week 52)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Plasma Lyso-Gb3</title>
          <description>Plasma Lyso-Gb3 is Fabry disease specific biomarker that can assess treatment outcome which was measured at Baseline and weeks 12, 26, 38, 52. Baseline and Month 12 (week 52) reported.</description>
          <units>nM</units>
          <param>Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="38.51" spread="9.68"/>
                    <measurement group_id="O2" value="51.81" spread="13.60"/>
                    <measurement group_id="O3" value="13.81" spread="2.31"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Month 12 (Week 52)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="24.20" spread="5.10"/>
                    <measurement group_id="O2" value="32.25" spread="6.89"/>
                    <measurement group_id="O3" value="9.24" spread="1.08"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-14.31" spread="5.13"/>
                    <measurement group_id="O2" value="-19.55" spread="7.55"/>
                    <measurement group_id="O3" value="-4.57" spread="1.42"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Number of Participants According to Protein/Creatinine Ratio (UPCR)</title>
        <description>Urine Protein to Creatinine Ratio (UPCR), assessed by spot urine test, at Month 12 (Week 52).Number of Participants According to Protein/Creatinine Ratio (UPCR) level</description>
        <time_frame>12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants According to Protein/Creatinine Ratio (UPCR)</title>
          <description>Urine Protein to Creatinine Ratio (UPCR), assessed by spot urine test, at Month 12 (Week 52).Number of Participants According to Protein/Creatinine Ratio (UPCR) level</description>
          <units>Subjects</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Protein Undetectable</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                    <measurement group_id="O2" value="7"/>
                    <measurement group_id="O3" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Normal to Mildly increased</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                    <measurement group_id="O2" value="2"/>
                    <measurement group_id="O3" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Moderately increased</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Severely increased</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="4"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Left Ventricular Mass Index (g/m^2) by MRI</title>
        <description>Left ventricular mass was determined based on cardiac MRI data and the LVMI was indexed to patient's body surface area (g/m^2). In male patients the normal range for LVMI was 57-91 g/m^2, in female patients 47-77 g/m^2.</description>
        <time_frame>12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Left Ventricular Mass Index (g/m^2) by MRI</title>
          <description>Left ventricular mass was determined based on cardiac MRI data and the LVMI was indexed to patient's body surface area (g/m^2). In male patients the normal range for LVMI was 57-91 g/m^2, in female patients 47-77 g/m^2.</description>
          <units>g/m^2</units>
          <param>Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="86.9" spread="6.9"/>
                    <measurement group_id="O2" value="97.6" spread="8.9"/>
                    <measurement group_id="O3" value="66.9" spread="5.8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Month 12 (Week 52)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="89.4" spread="6.1"/>
                    <measurement group_id="O2" value="98.3" spread="7.8"/>
                    <measurement group_id="O3" value="74.1" spread="7.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.1" spread="2.8"/>
                    <measurement group_id="O2" value="2.4" spread="3.4"/>
                    <measurement group_id="O3" value="7.1" spread="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Quality of Life EQ VAS</title>
        <description>The EQ VAS, of the EQ 5D 5L questionnaire, records the subject's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score &quot;100&quot;) and 'Worst imaginable health state' (score &quot;0&quot;).</description>
        <time_frame>12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>PRX-102 Efficacy Population</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O2">
            <title>PRX-102 Male</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
          <group group_id="O3">
            <title>PRX-102 Female</title>
            <description>PRX-102 infusion 1 mg/kg every 2 weeks</description>
          </group>
        </group_list>
        <measure>
          <title>Quality of Life EQ VAS</title>
          <description>The EQ VAS, of the EQ 5D 5L questionnaire, records the subject's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score &quot;100&quot;) and 'Worst imaginable health state' (score &quot;0&quot;).</description>
          <units>Score</units>
          <param>Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="20"/>
                <count group_id="O2" value="13"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="71.8" spread="4.3"/>
                    <measurement group_id="O2" value="66.8" spread="5.3"/>
                    <measurement group_id="O3" value="81.6" spread="6.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Month 12</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="76.9" spread="4.5"/>
                    <measurement group_id="O2" value="71.5" spread="5.4"/>
                    <measurement group_id="O3" value="86.7" spread="7.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.1" spread="3.3"/>
                    <measurement group_id="O2" value="4.8" spread="4.9"/>
                    <measurement group_id="O3" value="5.6" spread="3.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>12 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>All Patients</title>
          <description>PRX-102 infusion 1 mg/kg every 2 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="22"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>Type I hypersensitivity</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Infectious mononucleosis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</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="21" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Palpitations</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Gastroesophageal reflux disease</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Toothache</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>Type 1 hypersensitivity</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="9" subjects_affected="7" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Lower respiratory tract infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Infusion related reaction</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="5" subjects_affected="5" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" events="8" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="22"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash pruritic</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Erythema</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </event>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="22"/>
              </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 clinical trial agreement that is generally provided to the sites and Investigators contains a Publication paragraph that indicates the following: shall not, without the Sponsor's prior written consent, independently publish, present or otherwise disclose any results of or information pertaining to the trial until a multi-center publication is published, subject to certain limitations regarding timing and the confidentiality of unpublished data.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Sari Alon</name_or_title>
      <organization>Protalix Ltd.</organization>
      <phone>+972-4-9028100 ext 215</phone>
      <email>sari@protalix.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/NCT03018730
org study id: PB-102-F30
nct id: NCT03018730
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
This is an open label switch over study to assess the safety and efficacy of PRX-102
      (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a
      stable dose (>80% labelled dose/kg) for at least 6 months. Patients will be screened and
      evaluated over 3 months while continuing on agalsidase alfa. Following the screening period,
      the patient will be enrolled and switched from their agalsidase alfa treatment to receive
      intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25%
      of treated patients will be female.
Dosage and administration details:

      pegunigalsidase alfa individual dose for each patient was prepared according to the patient's
      weight. Pegunigalsidase alfa administrated at 1 mg/kg, intravenously over 3 hours, every 2
      weeks. After the first 2 months of treatment with pegunigalsidase alfa, infusion time may be
      reduced gradually to 1.5 hours pending patient tolerability.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
time frame: 12 months
description: Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment
measure: eGFR
time frame: Baseline and Month 12 (week 52)
description: eGFR was calculated based on the serum creatinine values that were assessed at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The absolute change in eGFR from baseline measurement at visit 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics.
measure: Mean Annualised Change in eGFR (Slope)
time frame: Pre-switch, Post-switch
description: The annualized change in eGFR (slope) per patient was calculated with all available eGFR values using a linear regression.
The mean pre-switch slope is the eGFR slope during screening period and pre-infusion visit 1 (while on Replagal®).
The mean post-switch slope is the eGFR slope during PRX-102 treatment, calculated based on eGFR vales at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 after visit 1.
The mean change in eGFR slope from pre- to post-switch is the mean difference between the two slopes.
eGFR was calculated based on the serum creatinine values according to the CKD-EPI formula.
measure: Plasma Lyso-Gb3
time frame: Baseline and month 12 (week 52)
description: Plasma Lyso-Gb3 is Fabry disease specific biomarker that can assess treatment outcome which was measured at Baseline and weeks 12, 26, 38, 52. Baseline and Month 12 (week 52) reported.
measure: Number of Participants According to Protein/Creatinine Ratio (UPCR)
time frame: 12 months
description: Urine Protein to Creatinine Ratio (UPCR), assessed by spot urine test, at Month 12 (Week 52).Number of Participants According to Protein/Creatinine Ratio (UPCR) level
measure: Left Ventricular Mass Index (g/m^2) by MRI
time frame: 12 months
description: Left ventricular mass was determined based on cardiac MRI data and the LVMI was indexed to patient's body surface area (g/m^2). In male patients the normal range for LVMI was 57-91 g/m^2, in female patients 47-77 g/m^2.
measure: Quality of Life EQ VAS
time frame: 12 months
description: The EQ VAS, of the EQ 5D 5L questionnaire, records the subject's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score "100") and 'Worst imaginable health state' (score "0").
arm group label: PRX-102
arm group type: Experimental
description: PRX-102 infusion every 2 weeks
intervention type: Biological
intervention name: PRX-102 (pegunigalsidase alfa)
description: PRX-102 1 mg/kg every 2 weeks
arm group label: PRX-102
other name: pegunigalsidase alfa
other name: Recombinant human alpha galactosidase-A
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Australia
country: Canada
country: Czechia
country: Netherlands
country: Norway
country: Slovenia
country: United Kingdom
country: Czech Republic
country: Germany
country: Spain
responsible party type: Sponsor
mesh term: Fabry Disease
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 