<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/NCT04429984</url>
  </required_header>
  <id_info>
    <org_study_id>SHP669-406</org_study_id>
    <nct_id>NCT04429984</nct_id>
  </id_info>
  <brief_title>Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India</brief_title>
  <official_title>A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India</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 main aim of this study is to measure the safety and to find out the effects of VPRIV in&#xD;
      participants with Gaucher disease using both retrospective and prospective data when used in&#xD;
      the post-marketing setting and to collect genetic mutation data from participants with&#xD;
      Gaucher disease.&#xD;
&#xD;
      This study is about collecting data available in the participant's medical record as well as&#xD;
      data from each participant's ongoing treatment. No study medicines will be provided to&#xD;
      participants in this study.&#xD;
&#xD;
      When the participants start the study, they will visit the study clinic close to&#xD;
      approximately 12 months.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 28, 2021</start_date>
  <completion_date type="Actual">April 22, 2023</completion_date>
  <primary_completion_date type="Actual">April 22, 2023</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Other</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Adverse Events (AEs)</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Adverse Drug Reactions (ADRs)</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Hemoglobin concentration will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Platelet count will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Spleen size will be assessed using ultrasound or Magnetic Resonance Image (MRI) in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Liver Size Using Both Retrospective and Prospective Data</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Liver size will be assessed using ultrasound or MRI in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment History Based on Previous VPRIV Treatment</measure>
    <time_frame>At Baseline</time_frame>
    <description>Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Platelet Count Based on Previous VPRIV Treatment</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Spleen Size Based on Previous VPRIV Treatment</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Liver Size Based on Previous VPRIV Treatment</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment</measure>
    <time_frame>Baseline up to approximately 12 months</time_frame>
    <description>An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A SAE is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious AEs will be collected using retrospective data of the previous VPRIV treatment. Baseline is defined as measurement from first time participants was dosed on CAP. Data will be assessed retrospectively based on participant records.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">21</enrollment>
  <condition>Gaucher Disease</condition>
  <arm_group>
    <arm_group_label>VPRIV: All Participants</arm_group_label>
    <description>Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Participants with confirmed type 1 Gaucher disease irrespective of any age or gender who&#xD;
        have been prescribed VPRIV therapy according to the investigator's judgment will be&#xD;
        included in this study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participants with type 1 Gaucher disease prescribed VPRIV according to the&#xD;
             investigator's judgment and current Indian Prescribing information (PI) are eligible&#xD;
             for this study.&#xD;
&#xD;
          -  Participants or legally authorized representative must provide written informed&#xD;
             consent to participate.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        - Participants will be excluded from this study if the participant met any of the&#xD;
        contraindications included in the current Indian PI for VPRIV.&#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>Amrita Institute of Medical Sciences &amp; Research Centre (AIMS)</name>
      <address>
        <city>Kochi</city>
        <state>Kerala</state>
        <zip>682041</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>All India Institute of Medical Sciences</name>
      <address>
        <city>New Delhi</city>
        <zip>110029</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>India</country>
  </location_countries>
  <link>
    <url>https://clinicaltrials.takeda.com/study-detail/5facd21917465c002989a198</url>
    <description>To obtain more information on the study, click here/on this link</description>
  </link>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>June 9, 2020</study_first_submitted>
  <study_first_submitted_qc>June 10, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 12, 2020</study_first_posted>
  <last_update_submitted>June 6, 2023</last_update_submitted>
  <last_update_submitted_qc>June 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 7, 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>
  <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/NCT04429984
org study id: SHP669-406
nct id: NCT04429984
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The main aim of this study is to measure the safety and to find out the effects of VPRIV in
      participants with Gaucher disease using both retrospective and prospective data when used in
      the post-marketing setting and to collect genetic mutation data from participants with
      Gaucher disease.

      This study is about collecting data available in the participant's medical record as well as
      data from each participant's ongoing treatment. No study medicines will be provided to
      participants in this study.

      When the participants start the study, they will visit the study clinic close to
      approximately 12 months.
observational model: Cohort
time perspective: Other
measure: Number of Participants With Adverse Events (AEs)
time frame: Baseline up to approximately 12 months
description: An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP).
measure: Number of Participants With Adverse Drug Reactions (ADRs)
time frame: Baseline up to approximately 12 months
description: An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
measure: Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data
time frame: Baseline up to approximately 12 months
description: Hemoglobin concentration will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
measure: Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data
time frame: Baseline up to approximately 12 months
description: Platelet count will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
measure: Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data
time frame: Baseline up to approximately 12 months
description: Spleen size will be assessed using ultrasound or Magnetic Resonance Image (MRI) in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
measure: Change From Baseline in Liver Size Using Both Retrospective and Prospective Data
time frame: Baseline up to approximately 12 months
description: Liver size will be assessed using ultrasound or MRI in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is first time participants were dosed on CAP.
measure: Treatment History Based on Previous VPRIV Treatment
time frame: At Baseline
description: Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
measure: Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment
time frame: Baseline up to approximately 12 months
description: Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
measure: Change From Baseline in Platelet Count Based on Previous VPRIV Treatment
time frame: Baseline up to approximately 12 months
description: Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
measure: Change From Baseline in Spleen Size Based on Previous VPRIV Treatment
time frame: Baseline up to approximately 12 months
description: Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
measure: Change From Baseline in Liver Size Based on Previous VPRIV Treatment
time frame: Baseline up to approximately 12 months
description: Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
measure: Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment
time frame: Baseline up to approximately 12 months
description: An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A SAE is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious AEs will be collected using retrospective data of the previous VPRIV treatment. Baseline is defined as measurement from first time participants was dosed on CAP. Data will be assessed retrospectively based on participant records.
arm group label: VPRIV: All Participants
description: Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study.
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: 
facility: 
country: India
url: https://clinicaltrials.takeda.com/study-detail/5facd21917465c002989a198
description: To obtain more information on the study, click here/on this link
responsible party type: Sponsor
mesh term: Gaucher 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/