<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/NCT03749980</url>
  </required_header>
  <id_info>
    <org_study_id>0001</org_study_id>
    <nct_id>NCT03749980</nct_id>
  </id_info>
  <brief_title>MyVHL: Patient Natural History Study</brief_title>
  <acronym>MyVHL</acronym>
  <official_title>MyVHL: Patient Natural History Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Joshua Mann, MPH</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Organization for Rare Disorders</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>VHL Alliance</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>
      MyVHL is a multi-patient database which helps researchers identify patterns across VHL&#xD;
      patients. MyVHL provides you -and researchers -with more complete information about VHL, like&#xD;
      how your lifestyle, medications, and other factors impact the disease and quality of life.&#xD;
      These insights help you better understand the condition and help researchers know where to&#xD;
      focus their efforts.&#xD;
&#xD;
      Due to its rarity, there is less understanding of VHL and the factors that may have an&#xD;
      impact. The data individuals provide in MyVHL helps researchers identify and uncover factors&#xD;
      that may increase risk, inhibit or slow tumor growth, or lead to an effective cure.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 2012</start_date>
  <completion_date type="Anticipated">December 2024</completion_date>
  <primary_completion_date type="Anticipated">December 2024</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>Other</time_perspective>
  </study_design_info>
  <target_duration>1 Year</target_duration>
  <primary_outcome>
    <measure>Number of patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.</measure>
    <time_frame>Through study completion, an average of 1 year.</time_frame>
    <description>Data regarding changes in number of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Size of tumors in patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.</measure>
    <time_frame>Through study completion, an average of 1 year.</time_frame>
    <description>Data regarding changes in size of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.</description>
  </primary_outcome>
  <enrollment type="Anticipated">10000</enrollment>
  <condition>Von Hippel-Lindau Disease</condition>
  <condition>Hereditary Leiomyomatosis and Renal Cell Cancer</condition>
  <condition>Birt-Hogg-Dube Syndrome</condition>
  <condition>SDHB Gene Mutation</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with von Hippel-Lindau Disease (VHL)&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  All patients with von Hippel-Lindau Disease (VHL)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        -&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Joshua Mann, MPH</last_name>
    <role>Principal Investigator</role>
    <affiliation>VHL Alliance</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Joshua Mann, MPH</last_name>
    <phone>161727756674</phone>
    <phone_ext>4</phone_ext>
    <email>josh.mann@vhl.org</email>
  </overall_contact>
  <location>
    <facility>
      <name>VHL Alliance</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02132</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Joshua Mann, MPH</last_name>
      <phone>617-277-5667</phone>
      <phone_ext>4</phone_ext>
      <email>josh.mann@vhl.org</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2022</verification_date>
  <study_first_submitted>May 30, 2018</study_first_submitted>
  <study_first_submitted_qc>November 20, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 21, 2018</study_first_posted>
  <last_update_submitted>October 13, 2022</last_update_submitted>
  <last_update_submitted_qc>October 13, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">October 14, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>VHL Alliance</investigator_affiliation>
    <investigator_full_name>Joshua Mann, MPH</investigator_full_name>
    <investigator_title>Director of Health</investigator_title>
  </responsible_party>
  <keyword>VHL</keyword>
  <keyword>BHD</keyword>
  <keyword>HLRCC</keyword>
  <keyword>SDHB</keyword>
  <keyword>MyVHL</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Carcinoma, Renal Cell</mesh_term>
    <mesh_term>Leiomyomatosis</mesh_term>
    <mesh_term>Birt-Hogg-Dube Syndrome</mesh_term>
    <mesh_term>Von Hippel-Lindau Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>When participant information is stored, the investigators are careful to protect any patient identifying information from discovery by others. Strict security safeguards are in place to reduce the chance of misuse or unplanned release of information.&#xD;
Researchers will only use participant information in a de-identified manner. De-identified means that the researchers will use participant information without knowing their identity. In some cases, they may use some identifying information about the participant for research purposes, subject to an approval process through the VHL Alliance MyVHL Research Committee. At times, the researchers will use participant information with a code, instead of their name; the code would allow results of the research to be linked back to the participant.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
  </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/NCT03749980
org study id: 0001
nct id: NCT03749980
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
MyVHL is a multi-patient database which helps researchers identify patterns across VHL
      patients. MyVHL provides you -and researchers -with more complete information about VHL, like
      how your lifestyle, medications, and other factors impact the disease and quality of life.
      These insights help you better understand the condition and help researchers know where to
      focus their efforts.

      Due to its rarity, there is less understanding of VHL and the factors that may have an
      impact. The data individuals provide in MyVHL helps researchers identify and uncover factors
      that may increase risk, inhibit or slow tumor growth, or lead to an effective cure.
observational model: Cohort
time perspective: Other
measure: Number of patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.
time frame: Through study completion, an average of 1 year.
description: Data regarding changes in number of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.
measure: Size of tumors in patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.
time frame: Through study completion, an average of 1 year.
description: Data regarding changes in size of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Joshua Mann, MPH
role: Principal Investigator
affiliation: VHL Alliance
last name: Joshua Mann, MPH
phone: 161727756674
phone ext: 4
email: josh.mann@vhl.org
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: VHL Alliance
investigator full name: Joshua Mann, MPH
investigator title: Director of Health
mesh term: Carcinoma, Renal Cell
mesh term: Leiomyomatosis
mesh term: Birt-Hogg-Dube Syndrome
mesh term: Von Hippel-Lindau Disease
sharing ipd: Yes
ipd description: When participant information is stored, the investigators are careful to protect any patient identifying information from discovery by others. Strict security safeguards are in place to reduce the chance of misuse or unplanned release of information.
Researchers will only use participant information in a de-identified manner. De-identified means that the researchers will use participant information without knowing their identity. In some cases, they may use some identifying information about the participant for research purposes, subject to an approval process through the VHL Alliance MyVHL Research Committee. At times, the researchers will use participant information with a code, instead of their name; the code would allow results of the research to be linked back to the participant.
ipd info type: Study Protocol
ipd info type: Informed Consent Form (ICF)