<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/NCT03291223</url>
  </required_header>
  <id_info>
    <org_study_id>GOS</org_study_id>
    <nct_id>NCT03291223</nct_id>
  </id_info>
  <brief_title>Gaucher Disease Outcome Survey (GOS)</brief_title>
  <official_title>Gaucher Disease Outcome Survey (GOS)</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 Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center,&#xD;
      long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or&#xD;
      type of treatment received. No experimental intervention is involved. Patients undergo&#xD;
      clinical assessments and receive care as determined by the patients' treating physician.&#xD;
&#xD;
      The objectives of the registry include to evaluate the safety and long-term effectiveness of&#xD;
      velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher&#xD;
      Disease-specific treatments, to gain a better understanding of the natural history of GD and&#xD;
      to serve as a database for evidence-based management of Gaucher Disease over time in&#xD;
      real-life clinical practice.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      20 MAY 2020: The temporary enrollment stop of new patients into this study due to the&#xD;
      COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again&#xD;
      enrolling new patients. However, some countries/sites may still have paused the enrollment of&#xD;
      new patients due to the pandemic.&#xD;
&#xD;
      24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19&#xD;
      situation. The duration of this pause is dependent on the leveling and control of the&#xD;
      COVID-19 pandemic.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">July 27, 2010</start_date>
  <completion_date type="Anticipated">December 15, 2023</completion_date>
  <primary_completion_date type="Anticipated">December 15, 2023</primary_completion_date>
  <study_type>Observational [Patient Registry]</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <target_duration>6 Months</target_duration>
  <primary_outcome>
    <measure>Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Infusion-related Reactions (IRRs)</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Increase of Hemoglobin Concentration</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Hemoglobin concentration will be assessed.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Increase of Platelet Count</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Platelet count will be assessed.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Decrease in Liver Volume</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Liver volume will be assessed by abdominal imaging.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Decrease in Spleen Volume</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Spleen volume will be assessed by abdominal imaging.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Increase in Bone Mineral Density (BMD)</measure>
    <time_frame>Baseline to one year for up to 20 years</time_frame>
    <description>Bone mineral density will be assessed.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">1257</enrollment>
  <condition>Gaucher Disease</condition>
  <arm_group>
    <arm_group_label>GOS Participants</arm_group_label>
    <description>GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        GOS is a disease specific registry irrespective of treatment or treatment status, open to&#xD;
        all patients of any age or sex with Gaucher disease of any type. Patients included may be&#xD;
        those who are untreated, naive to therapy, individuals who are currently or have been&#xD;
        previously treated with velaglucerase alfa (VPRIV), or individuals who have been receiving&#xD;
        or are currently exposed to other treatments for Gaucher disease. There is no predefined&#xD;
        sample size.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of&#xD;
             Gaucher disease&#xD;
&#xD;
          -  Signed and dated written informed consent from the patient or, for patients aged &lt;18&#xD;
             years (&lt;16 years in the United Kingdom [UK]), their parent and/or legally authorized&#xD;
             representatives (LAR), and assent of the minor where applicable. Legally authorized&#xD;
             representatives are also applicable for cognitively impaired patients.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        - Patients currently enrolled in ongoing blinded clinical trials (drugs or devices;&#xD;
        includes all blinded trials)&#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>Shire Study Physician</last_name>
    <role>Study Director</role>
    <affiliation>Shire</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Shire Contact</last_name>
    <phone>+1 866 842 5335</phone>
    <email>ClinicalTransparency@shire.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Central Contact</name>
      <address>
        <city>Lexington</city>
        <state>Massachusetts</state>
        <zip>02421</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Shire Central Contact</last_name>
      <email>ClinicalTransparency@shire.com</email>
    </contact>
    <investigator>
      <last_name>Central Contact</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://clinicaltrials.takeda.com/study-detail/5f6b5fcd4db2bf003ab4662b</url>
    <description>To obtain more information on the study, click here/on this link</description>
  </link>
  <verification_date>March 2023</verification_date>
  <study_first_submitted>August 8, 2017</study_first_submitted>
  <study_first_submitted_qc>September 19, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 25, 2017</study_first_posted>
  <last_update_submitted>March 13, 2023</last_update_submitted>
  <last_update_submitted_qc>March 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 16, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Gaucher Disease</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03291223
org study id: GOS
nct id: NCT03291223
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center,
      long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or
      type of treatment received. No experimental intervention is involved. Patients undergo
      clinical assessments and receive care as determined by the patients' treating physician.

      The objectives of the registry include to evaluate the safety and long-term effectiveness of
      velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher
      Disease-specific treatments, to gain a better understanding of the natural history of GD and
      to serve as a database for evidence-based management of Gaucher Disease over time in
      real-life clinical practice.
20 MAY 2020: The temporary enrollment stop of new patients into this study due to the
      COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again
      enrolling new patients. However, some countries/sites may still have paused the enrollment of
      new patients due to the pandemic.

      24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19
      situation. The duration of this pause is dependent on the leveling and control of the
      COVID-19 pandemic.
observational model: Cohort
time perspective: Prospective
measure: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
time frame: Baseline to one year for up to 20 years
description: Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
measure: Number of Participants With Infusion-related Reactions (IRRs)
time frame: Baseline to one year for up to 20 years
description: An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.
measure: Increase of Hemoglobin Concentration
time frame: Baseline to one year for up to 20 years
description: Hemoglobin concentration will be assessed.
measure: Increase of Platelet Count
time frame: Baseline to one year for up to 20 years
description: Platelet count will be assessed.
measure: Decrease in Liver Volume
time frame: Baseline to one year for up to 20 years
description: Liver volume will be assessed by abdominal imaging.
measure: Decrease in Spleen Volume
time frame: Baseline to one year for up to 20 years
description: Spleen volume will be assessed by abdominal imaging.
measure: Increase in Bone Mineral Density (BMD)
time frame: Baseline to one year for up to 20 years
description: Bone mineral density will be assessed.
arm group label: GOS Participants
description: GOS is a disease specific registry open to all Gaucher patients 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: Shire Study Physician
role: Study Director
affiliation: Shire
last name: Shire Contact
phone: +1 866 842 5335
email: ClinicalTransparency@shire.com
facility: 
status: Recruiting
contact: 
investigator: 
country: United States
url: https://clinicaltrials.takeda.com/study-detail/5f6b5fcd4db2bf003ab4662b
description: To obtain more information on the study, click here/on this link
responsible party type: Sponsor
mesh term: Gaucher Disease