<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/NCT03289065</url>
  </required_header>
  <id_info>
    <org_study_id>FOS</org_study_id>
    <nct_id>NCT03289065</nct_id>
  </id_info>
  <brief_title>Fabry Outcome Survey (FOS)</brief_title>
  <acronym>FOS</acronym>
  <official_title>Fabry Outcome Survey (FOS)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Shire</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Takeda</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to collect data that will increase understanding of Fabry&#xD;
      disease history and progression, in treated and untreated patients with Fabry disease. The&#xD;
      data from FOS may provide guidance to healthcare professionals about disease treatment&#xD;
      options.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 1, 2001</start_date>
  <completion_date type="Actual">September 30, 2021</completion_date>
  <primary_completion_date type="Actual">September 30, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Other</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>An adverse event (AE) is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or ADR that meets one or more of the following criteria/outcomes is classified as SAE whether considered to be related to the pharmaceutical product or not: death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Infusion-related Reactions (IRRs)</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>An infusion-related reaction (IRR) is defined as an AE that has been assessed as at least possibly related to treatment with Replagal and occurs during an infusion or up to 24 hours post Replagal infusion.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Renal Function by Estimated Glomerular Filtration Rate (eGFR)</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>Renal function will be measured by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for adults and by Counahan-Barratt for children (&lt;18 years).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Left Ventricular Mass Index (LVMI)</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>Left ventricular mass index (LVMI) will be assessed from baseline or from birth (age at event) to evaluate the course of Fabry disease in participants who are currently untreated or are being treated with an approved Fabry treatment.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Age at Mortality Event (survival)</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>Age at mortality event (survival) will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in Fabry Outcome Survey (FOS)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to First Morbidity Event</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>Time to first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Age at First Morbidity Event</measure>
    <time_frame>Baseline to year 20</time_frame>
    <description>Age at first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">4000</enrollment>
  <condition>Fabry Disease</condition>
  <arm_group>
    <arm_group_label>FOS Participant</arm_group_label>
    <description>FOS is a disease registry open to all Fabry participants irrespective of treatment status or type of treatment.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        The FOS registry is open to participants with Fabry disease irrespective of their&#xD;
        treatment. Participants may be untreated, currently treated or have been previously treated&#xD;
        with Replagal, or any other approved treatment for Fabry disease. There is no predetermined&#xD;
        sample size.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Participants must have a documented diagnosis of Fabry disease&#xD;
&#xD;
               -  This may include a genetic mutation analysis. The collection of the genetic&#xD;
                  mutation analysis result is optional and dependent on the participant providing&#xD;
                  their consent for this data to be used in the FOS registry.&#xD;
&#xD;
               -  Participants can be untreated, currently or previously treated with Replagal, or&#xD;
                  any other approved treatment for Fabry disease.&#xD;
&#xD;
          2. Signed and dated written informed consent from the participant&#xD;
&#xD;
               -  For participants aged less than (&lt;) 18 years (or as per local regulation), parent&#xD;
                  and/or participant's legally authorized representative (LAR), and assent of the&#xD;
                  minor, where applicable, is necessary.&#xD;
&#xD;
               -  If a participant is unable to read or if a legally acceptable representative is&#xD;
                  unable to read, an impartial witness should be present during the informed&#xD;
                  consent discussion and should sign and personally date the informed consent.&#xD;
&#xD;
               -  Informed consent must be obtained from LARs for cognitively impaired participants&#xD;
                  when applicable.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        1. Participants currently enrolled in ongoing blinded clinical trials (drugs or devices;&#xD;
        includes all blinded trials) will be excluded from the Registry.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Study Director</last_name>
    <role>Study Director</role>
    <affiliation>Shire</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Shire</name>
      <address>
        <city>Lexington</city>
        <state>Massachusetts</state>
        <zip>02421</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>November 2021</verification_date>
  <study_first_submitted>August 17, 2017</study_first_submitted>
  <study_first_submitted_qc>September 18, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 20, 2017</study_first_posted>
  <last_update_submitted>November 10, 2021</last_update_submitted>
  <last_update_submitted_qc>November 10, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 16, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Genetic</keyword>
  <keyword>Fabry Disease</keyword>
  <keyword>Glycolipid</keyword>
  <keyword>lysosomal</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>Yes</sharing_ipd>
    <ipd_description>Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_info_type>Clinical Study Report (CSR)</ipd_info_type>
    <ipd_access_criteria>IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.</ipd_access_criteria>
    <ipd_url>https://vivli.org/ourmember/takeda/</ipd_url>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03289065
org study id: FOS
nct id: NCT03289065
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to collect data that will increase understanding of Fabry
      disease history and progression, in treated and untreated patients with Fabry disease. The
      data from FOS may provide guidance to healthcare professionals about disease treatment
      options.
observational model: Other
time perspective: Prospective
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
time frame: Baseline to year 20
description: An adverse event (AE) is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or ADR that meets one or more of the following criteria/outcomes is classified as SAE whether considered to be related to the pharmaceutical product or not: death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.
measure: Number of Participants With Infusion-related Reactions (IRRs)
time frame: Baseline to year 20
description: An infusion-related reaction (IRR) is defined as an AE that has been assessed as at least possibly related to treatment with Replagal and occurs during an infusion or up to 24 hours post Replagal infusion.
measure: Renal Function by Estimated Glomerular Filtration Rate (eGFR)
time frame: Baseline to year 20
description: Renal function will be measured by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for adults and by Counahan-Barratt for children (<18 years).
measure: Left Ventricular Mass Index (LVMI)
time frame: Baseline to year 20
description: Left ventricular mass index (LVMI) will be assessed from baseline or from birth (age at event) to evaluate the course of Fabry disease in participants who are currently untreated or are being treated with an approved Fabry treatment.
measure: Age at Mortality Event (survival)
time frame: Baseline to year 20
description: Age at mortality event (survival) will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in Fabry Outcome Survey (FOS)
measure: Time to First Morbidity Event
time frame: Baseline to year 20
description: Time to first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.
measure: Age at First Morbidity Event
time frame: Baseline to year 20
description: Age at first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.
arm group label: FOS Participant
description: FOS is a disease registry open to all Fabry participants irrespective of treatment status or type of treatment.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Study Director
role: Study Director
affiliation: Shire
facility: 
country: United States
responsible party type: Sponsor
mesh term: Fabry Disease
sharing ipd: Yes
ipd description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd info type: Clinical Study Report (CSR)
ipd access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
ipd url: https://vivli.org/ourmember/takeda/