<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/NCT03251989</url>
  </required_header>
  <id_info>
    <org_study_id>999917141</org_study_id>
    <secondary_id>17-C-N141</secondary_id>
    <nct_id>NCT03251989</nct_id>
  </id_info>
  <brief_title>Rare CNS Tumors Outcomes &amp;Risk</brief_title>
  <official_title>Exploring Outcomes and Risk in Patients With Rare Central Nervous System Tumors</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>National Institutes of Health Clinical Center (CC)</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Background:&#xD;
&#xD;
      Primary tumors of the brain and spine are those that start in the brain or spine. These&#xD;
      tumors are rare, accounting for &lt;2% of all cancers diagnosed in the United States. Some of&#xD;
      these tumors occur in less than 2,000 people per year. Researchers want to study a large&#xD;
      group of people with this kind of tumor. They want to learn more about the tumors, including&#xD;
      the risk factors related to how they develop in adults.&#xD;
&#xD;
      Objective: To collect health and gene data to learn about what changes are associated with a&#xD;
      rare CNS Tumors, to eventually screen for these changes or target the genes in treatment.&#xD;
&#xD;
      Eligibility: Adult participants (Bullet) 18 years of age who self- identify as being&#xD;
      diagnosed with one of 12 rare CNS tumors, including: Atypical teratoid rhabdoid tumor (ATRT);&#xD;
      Brainstem and midline gliomas; Choroid plexus tumors; Ependymoma; High grade meningioma;&#xD;
      Gliomatosis cerebri; Medulloblastoma; Oligodendroglioma / Anaplastic oligodendroglioma;&#xD;
      Pineal region tumors; Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic&#xD;
      xanthroastrocytoma; PNET (Supratentorial embryonal tumor); Primary CNS sarcoma / Secondary&#xD;
      CNS sarcoma (Gliosarcoma).&#xD;
&#xD;
      Design: (Registered Trademark)Participants will be invited to participate through an ad on&#xD;
      the CERN Foundation website (ependymoma), information on the Neuro-Oncology Branch website&#xD;
      and other identified advocacy and social media sites and direct mailer to those who have&#xD;
      already participated in the EO projects. (Registered Trademark)&#xD;
&#xD;
        -  Interested participants will complete an enrollment form that will be sent to the study&#xD;
           coordinator.&#xD;
&#xD;
        -  The coordinator will then send the participant a consent form and schedule a time for&#xD;
           phone consent.&#xD;
&#xD;
        -  Participants will complete the Rare CNS tumors Outcomes Survey and once completed, the&#xD;
           Rare CNS tumors Risk survey.&#xD;
&#xD;
      (Registered Trademark)&#xD;
&#xD;
        -  The questions on the Outcomes Survey will include treatment history, symptoms social and&#xD;
           clinical information and it should take about 25-35 minutes. The Risk survey will cover&#xD;
           their demographic information, personal medical history, family medical history and&#xD;
           environmental exposures. This should take about 52 minutes.&#xD;
&#xD;
        -  Participants who have physical problems can have help with the surveys and forms.&#xD;
&#xD;
        -  Once the surveys are completed, participants will be mailed a kit to collect saliva for&#xD;
           germline DNA. Participants will ship the sample to the study team in a prepaid envelope&#xD;
&#xD;
        -  If the sample is not sufficient, participants will be contacted to give provide an&#xD;
           additional sample.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Background:&#xD;
&#xD;
      Rare cancers are defined as those with &lt;40,000 cases per year. There are over 130 separate&#xD;
      types of primary CNS tumors, all of which meet the definition of a rare cancer. However, some&#xD;
      CNS cancers have incidences of less than 1,000 cases per year. Because of its relative&#xD;
      rarity, limited reports of the presentation and clinical course have been completed. With the&#xD;
      support of the CERN-Foundation, the Adult Ependymoma Outcomes Projects (AEO) was launched to&#xD;
      address this lack of information for one type of rare CNS tumor- ependymoma. Using an online&#xD;
      platform, participants and families from across the world have participated, providing&#xD;
      information that has helped elucidate the presentation and ongoing health of individuals with&#xD;
      this disease. To date, over 300 adults have participated with over 95% reporting interest in&#xD;
      continuing and expanding their participation. Results from the AEO baseline survey and a&#xD;
      follow-up survey (AEOII addressing socioeconomic impact of the disease) have been published.&#xD;
      The most recent data from the AEO has been presented at the Society for Neuro-Oncology (SNO)&#xD;
      annual meetings in 2015 and 2016.&#xD;
&#xD;
      An additional consequence of the relative rarity of these CNS tumors, studies to evaluate&#xD;
      risk factors for the occurrence of these rare CNS tumors or predicting the clinical course of&#xD;
      these rare CNS tumors are also limited. Rare CNS tumors like other cancers, occur when there&#xD;
      are changes to genes that control the way cells grow and divide often as a result of exposure&#xD;
      to other environmental risk factors. Therefore, exploring genetic changes in persons with&#xD;
      rare CNS tumors will allow us to begin to understand what changes are associated specifically&#xD;
      with these tumors. To date, these participants are often included as part of larger cohorts&#xD;
      which include other types of brain tumors. We now understand that even among gliomas, the&#xD;
      risk factors differ. Therefore, identifying the risk factors specifically associated with&#xD;
      rare CNS tumors is critical for primary prevention and early detection. This knowledge would&#xD;
      allow scientists and physicians to eventually screen for these changes or target the genes or&#xD;
      the processes they control for treatment purposes.&#xD;
&#xD;
      Objectives:&#xD;
&#xD;
      The primary objectives of this study are to:&#xD;
&#xD;
        -  Obtain self-reported data on treatment, symptoms, functional status, and quality of life&#xD;
           for adult participants with rare CNS tumors.&#xD;
&#xD;
        -  To evaluate the relationship between health status and disease and treatment&#xD;
           characteristics.&#xD;
&#xD;
        -  To evaluate self-reported clinical and demographic risk factors in adult participants in&#xD;
           the rare CNS tumor participant population.&#xD;
&#xD;
        -  To explore genomic susceptibility in participants with rare CNS tumors&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
      The adult rare CNS tumor population for this study are participants &gt;= 18 years of age who&#xD;
      self- identify as being diagnosed with one of 12 rare CNS tumors, including:&#xD;
&#xD;
        -  Atypical teratoid rhabdoid tumor (ATRT)&#xD;
&#xD;
        -  Brainstem and midline gliomas&#xD;
&#xD;
        -  Choroid plexus tumors&#xD;
&#xD;
             -  Choroid plexus carcinoma&#xD;
&#xD;
             -  Choroid plexus papilloma&#xD;
&#xD;
             -  Atypical choroid plexus papilloma&#xD;
&#xD;
        -  Ependymoma&#xD;
&#xD;
             -  Subependymoma&#xD;
&#xD;
             -  Myxopapillary ependymoma&#xD;
&#xD;
             -  Papillary ependymoma&#xD;
&#xD;
             -  Clear cell ependymoma&#xD;
&#xD;
             -  Tanycytic ependymoma&#xD;
&#xD;
             -  RELA fusion-positive&#xD;
&#xD;
             -  Anaplastic ependymoma&#xD;
&#xD;
             -  Posterior Fossa A&#xD;
&#xD;
             -  Posterior Fossa B&#xD;
&#xD;
        -  High grade meningioma&#xD;
&#xD;
             -  Chordoid meningioma&#xD;
&#xD;
             -  Clear cell meningioma&#xD;
&#xD;
             -  Atypical meningioma&#xD;
&#xD;
             -  Papillary meningioma&#xD;
&#xD;
             -  Rhabdoid meningioma&#xD;
&#xD;
             -  Anaplastic (malignant) meningioma&#xD;
&#xD;
        -  Gliomatosis cerebri&#xD;
&#xD;
        -  Medulloblastoma&#xD;
&#xD;
             -  Sonic Hedge Hog (SHH)&#xD;
&#xD;
             -  WNT&#xD;
&#xD;
             -  Group 3&#xD;
&#xD;
             -  Group 4&#xD;
&#xD;
        -  Oligodendroglioma / Anaplastic oligodendroglioma&#xD;
&#xD;
        -  Pineal region Tumors&#xD;
&#xD;
             -  Pineoblastoma&#xD;
&#xD;
             -  Pineocytoma&#xD;
&#xD;
             -  Pineal parenchymal tumor of intermediate differentiation&#xD;
&#xD;
             -  Papillary tumor of the pineal region&#xD;
&#xD;
        -  Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic xanthroastrocytoma&#xD;
&#xD;
        -  PNET (Supratentorial embryonal tumor)&#xD;
&#xD;
        -  Primary CNS sarcoma / Secondary CNS sarcoma (Gliosarcoma)&#xD;
&#xD;
      Design:&#xD;
&#xD;
        -  This study represents an ongoing effort to systematically evaluate outcomes in a large&#xD;
           group of participants with these rare tumors. It will provide descriptive data which can&#xD;
           be used to design further studies evaluating interventions related to identified health&#xD;
           and quality of life related issues for this participant population.&#xD;
&#xD;
        -  Participants will be invited to participate through an ad on the CERN Foundation website&#xD;
           (ependymoma), information on the Neuro-Oncology Branch website and other identified&#xD;
           advocacy and social media sites and direct mailer to those who have already participated&#xD;
           in the EO projects.&#xD;
&#xD;
        -  The goal of adding the Rare CNS tumors Risk Survey is to implement a survey to further&#xD;
           evaluate participants in the EO for several known and suspected risk factors for rare&#xD;
           CNS tumors and to obtain germline DNA to interrogate genomic susceptibility to the&#xD;
           disease.&#xD;
&#xD;
        -  Interested participants will complete an enrollment form that will be sent to the study&#xD;
           coordinator. Once submitted, the study coordinator will then send the participant a&#xD;
           consent form and schedule a time for remote consent if interested.&#xD;
&#xD;
        -  Once consent is obtained, the study coordinator will send the participant instructions&#xD;
           and a unique link that they will use to access and complete the survey(s).&#xD;
&#xD;
        -  Once the surveys are completed, the study coordinator will email the participant a&#xD;
           request for medical records to help us collect more health information related to care&#xD;
           for their Rare CNS tumor diagnosis.&#xD;
&#xD;
        -  Once the survey(s) are completed, the participant will be mailed a kit to collect saliva&#xD;
           for germline DNA.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Suspended</overall_status>
  <why_stopped>
    Strategic planning for the types of tumors is being done. Temp hold until AM is submitted.&#xD;
  </why_stopped>
  <start_date type="Actual">August 21, 2017</start_date>
  <completion_date type="Anticipated">February 26, 2025</completion_date>
  <primary_completion_date type="Anticipated">February 1, 2025</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>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Relationship between health status and disease and treatment characteristics as well as clinical and demographic risk factors as self reported by adult participants with rare CNS tumors; and the relationship of genomic susceptibility of the popu...</measure>
    <time_frame>completion of study</time_frame>
    <description>Obtain self-reported data on treatment, symptoms, functional status, and quality of life for adult participants with rare CNS tumors To evaluate the relationship between health status and disease and treatment characteristics. To evaluate self-reported clinical and demographic risk factors in adult participants in the rare CNS tumors participant population. To explore genomic susceptibility in participants with rare CNS tumors.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">326</enrollment>
  <condition>High Grade Meningioma</condition>
  <condition>Ependymoma</condition>
  <condition>Medulloblastoma</condition>
  <condition>PNET</condition>
  <condition>Primary CNS Sarcoma</condition>
  <arm_group>
    <arm_group_label>1/Patient with a rare CNS diagnosis</arm_group_label>
    <description>A diagnosis of rare CNS Tumors</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Participants will be solicited through the Collaborative Ependymoma Research Network&#xD;
        (CERN), information on the Neuro-Oncology Branch website and other advocacy organization&#xD;
        websites, mailing list and social media sites.@@@&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        -  INCLUSION CRITERIA:&#xD;
&#xD;
        Participants with rare CNS tumors who meet the following criteria will be invited to&#xD;
        participate in the study:&#xD;
&#xD;
          -  A diagnosis of rare CNS tumors, (Atypical teratoid rhabdoid tumor (ATRT); Brainstem&#xD;
             and midline gliomas; Choroid plexus tumors; Ependymoma; High grade meningioma;&#xD;
             Gliomatosis cerebri; Medulloblastoma; Oligodendroglioma / Anaplastic&#xD;
             oligodendroglioma; Pineal region tumors; Pleomorphic xanthroastrocytoma / Anaplastic&#xD;
             pleomorphic xanthroastrocytoma; PNET (Supratentorial embryonal tumor); Primary CNS&#xD;
             sarcoma / Secondary CNS sarcoma (Gliosarcoma) or as reported by the participant&#xD;
&#xD;
          -  Ability to speak, write, and read English, as questionnaires available in English&#xD;
             language only.&#xD;
&#xD;
          -  Ability of participant to understand and the willingness to sign a written informed&#xD;
             consent document.&#xD;
&#xD;
        EXCLUSION CRITERIA: See inclusion criteria&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Terri S Armstrong, C.R.N.P.</last_name>
    <role>Principal Investigator</role>
    <affiliation>National Cancer Institute (NCI)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>National Cancer Institute (NCI)/ Neuro-Oncology Branch</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <reference>
    <citation>Armstrong TS, Vera-Bolanos E, Gilbert MR. Clinical course of adult patients with ependymoma: results of the Adult Ependymoma Outcomes Project. Cancer. 2011 Nov 15;117(22):5133-41. doi: 10.1002/cncr.26181. Epub 2011 Apr 28.</citation>
    <PMID>21538344</PMID>
  </reference>
  <reference>
    <citation>Walbert T, Mendoza TR, Vera-Bolanos E, Acquaye A, Gilbert MR, Armstrong TS. Symptoms and socio-economic impact of ependymoma on adult patients: results of the Adult Ependymoma Outcomes Project 2. J Neurooncol. 2015 Jan;121(2):341-8. doi: 10.1007/s11060-014-1638-4. Epub 2014 Oct 31.</citation>
    <PMID>25359395</PMID>
  </reference>
  <reference>
    <citation>Acquaye AA, Vera E, Gilbert MR, Armstrong TS. Clinical presentation and outcomes for adult ependymoma patients. Cancer. 2017 Feb 1;123(3):494-501. doi: 10.1002/cncr.30355. Epub 2016 Sep 28.</citation>
    <PMID>27679985</PMID>
  </reference>
  <verification_date>January 30, 2023</verification_date>
  <study_first_submitted>August 15, 2017</study_first_submitted>
  <study_first_submitted_qc>August 15, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 16, 2017</study_first_posted>
  <last_update_submitted>January 31, 2023</last_update_submitted>
  <last_update_submitted_qc>January 31, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 1, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Brain Tumor</keyword>
  <keyword>Central Nervous System Tumors</keyword>
  <keyword>Choroid Plexus Tumors</keyword>
  <keyword>Gliomas</keyword>
  <keyword>Atypical Teratoid Rhabdoid Tumor</keyword>
  <keyword>Natural History</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Meningioma</mesh_term>
    <mesh_term>Ependymoma</mesh_term>
    <mesh_term>Medulloblastoma</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>.All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.</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_time_frame>Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active. @@@@@@All collected IPD will be available after primary analysis have been published.</ipd_time_frame>
    <ipd_access_criteria>Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. @@@@@@Genomic data are made available via dbGaP through requests to the data custodians.</ipd_access_criteria>
  </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/NCT03251989
org study id: 999917141
secondary id: 17-C-N141
nct id: NCT03251989
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
Background:

      Primary tumors of the brain and spine are those that start in the brain or spine. These
      tumors are rare, accounting for <2% of all cancers diagnosed in the United States. Some of
      these tumors occur in less than 2,000 people per year. Researchers want to study a large
      group of people with this kind of tumor. They want to learn more about the tumors, including
      the risk factors related to how they develop in adults.

      Objective: To collect health and gene data to learn about what changes are associated with a
      rare CNS Tumors, to eventually screen for these changes or target the genes in treatment.

      Eligibility: Adult participants (Bullet) 18 years of age who self- identify as being
      diagnosed with one of 12 rare CNS tumors, including: Atypical teratoid rhabdoid tumor (ATRT);
      Brainstem and midline gliomas; Choroid plexus tumors; Ependymoma; High grade meningioma;
      Gliomatosis cerebri; Medulloblastoma; Oligodendroglioma / Anaplastic oligodendroglioma;
      Pineal region tumors; Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic
      xanthroastrocytoma; PNET (Supratentorial embryonal tumor); Primary CNS sarcoma / Secondary
      CNS sarcoma (Gliosarcoma).

      Design: (Registered Trademark)Participants will be invited to participate through an ad on
      the CERN Foundation website (ependymoma), information on the Neuro-Oncology Branch website
      and other identified advocacy and social media sites and direct mailer to those who have
      already participated in the EO projects. (Registered Trademark)

        -  Interested participants will complete an enrollment form that will be sent to the study
           coordinator.

        -  The coordinator will then send the participant a consent form and schedule a time for
           phone consent.

        -  Participants will complete the Rare CNS tumors Outcomes Survey and once completed, the
           Rare CNS tumors Risk survey.

      (Registered Trademark)

        -  The questions on the Outcomes Survey will include treatment history, symptoms social and
           clinical information and it should take about 25-35 minutes. The Risk survey will cover
           their demographic information, personal medical history, family medical history and
           environmental exposures. This should take about 52 minutes.

        -  Participants who have physical problems can have help with the surveys and forms.

        -  Once the surveys are completed, participants will be mailed a kit to collect saliva for
           germline DNA. Participants will ship the sample to the study team in a prepaid envelope

        -  If the sample is not sufficient, participants will be contacted to give provide an
           additional sample.
Background:

      Rare cancers are defined as those with <40,000 cases per year. There are over 130 separate
      types of primary CNS tumors, all of which meet the definition of a rare cancer. However, some
      CNS cancers have incidences of less than 1,000 cases per year. Because of its relative
      rarity, limited reports of the presentation and clinical course have been completed. With the
      support of the CERN-Foundation, the Adult Ependymoma Outcomes Projects (AEO) was launched to
      address this lack of information for one type of rare CNS tumor- ependymoma. Using an online
      platform, participants and families from across the world have participated, providing
      information that has helped elucidate the presentation and ongoing health of individuals with
      this disease. To date, over 300 adults have participated with over 95% reporting interest in
      continuing and expanding their participation. Results from the AEO baseline survey and a
      follow-up survey (AEOII addressing socioeconomic impact of the disease) have been published.
      The most recent data from the AEO has been presented at the Society for Neuro-Oncology (SNO)
      annual meetings in 2015 and 2016.

      An additional consequence of the relative rarity of these CNS tumors, studies to evaluate
      risk factors for the occurrence of these rare CNS tumors or predicting the clinical course of
      these rare CNS tumors are also limited. Rare CNS tumors like other cancers, occur when there
      are changes to genes that control the way cells grow and divide often as a result of exposure
      to other environmental risk factors. Therefore, exploring genetic changes in persons with
      rare CNS tumors will allow us to begin to understand what changes are associated specifically
      with these tumors. To date, these participants are often included as part of larger cohorts
      which include other types of brain tumors. We now understand that even among gliomas, the
      risk factors differ. Therefore, identifying the risk factors specifically associated with
      rare CNS tumors is critical for primary prevention and early detection. This knowledge would
      allow scientists and physicians to eventually screen for these changes or target the genes or
      the processes they control for treatment purposes.

      Objectives:

      The primary objectives of this study are to:

        -  Obtain self-reported data on treatment, symptoms, functional status, and quality of life
           for adult participants with rare CNS tumors.

        -  To evaluate the relationship between health status and disease and treatment
           characteristics.

        -  To evaluate self-reported clinical and demographic risk factors in adult participants in
           the rare CNS tumor participant population.

        -  To explore genomic susceptibility in participants with rare CNS tumors

      Eligibility:

      The adult rare CNS tumor population for this study are participants >= 18 years of age who
      self- identify as being diagnosed with one of 12 rare CNS tumors, including:

        -  Atypical teratoid rhabdoid tumor (ATRT)

        -  Brainstem and midline gliomas

        -  Choroid plexus tumors

             -  Choroid plexus carcinoma

             -  Choroid plexus papilloma

             -  Atypical choroid plexus papilloma

        -  Ependymoma

             -  Subependymoma

             -  Myxopapillary ependymoma

             -  Papillary ependymoma

             -  Clear cell ependymoma

             -  Tanycytic ependymoma

             -  RELA fusion-positive

             -  Anaplastic ependymoma

             -  Posterior Fossa A

             -  Posterior Fossa B

        -  High grade meningioma

             -  Chordoid meningioma

             -  Clear cell meningioma

             -  Atypical meningioma

             -  Papillary meningioma

             -  Rhabdoid meningioma

             -  Anaplastic (malignant) meningioma

        -  Gliomatosis cerebri

        -  Medulloblastoma

             -  Sonic Hedge Hog (SHH)

             -  WNT

             -  Group 3

             -  Group 4

        -  Oligodendroglioma / Anaplastic oligodendroglioma

        -  Pineal region Tumors

             -  Pineoblastoma

             -  Pineocytoma

             -  Pineal parenchymal tumor of intermediate differentiation

             -  Papillary tumor of the pineal region

        -  Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic xanthroastrocytoma

        -  PNET (Supratentorial embryonal tumor)

        -  Primary CNS sarcoma / Secondary CNS sarcoma (Gliosarcoma)

      Design:

        -  This study represents an ongoing effort to systematically evaluate outcomes in a large
           group of participants with these rare tumors. It will provide descriptive data which can
           be used to design further studies evaluating interventions related to identified health
           and quality of life related issues for this participant population.

        -  Participants will be invited to participate through an ad on the CERN Foundation website
           (ependymoma), information on the Neuro-Oncology Branch website and other identified
           advocacy and social media sites and direct mailer to those who have already participated
           in the EO projects.

        -  The goal of adding the Rare CNS tumors Risk Survey is to implement a survey to further
           evaluate participants in the EO for several known and suspected risk factors for rare
           CNS tumors and to obtain germline DNA to interrogate genomic susceptibility to the
           disease.

        -  Interested participants will complete an enrollment form that will be sent to the study
           coordinator. Once submitted, the study coordinator will then send the participant a
           consent form and schedule a time for remote consent if interested.

        -  Once consent is obtained, the study coordinator will send the participant instructions
           and a unique link that they will use to access and complete the survey(s).

        -  Once the surveys are completed, the study coordinator will email the participant a
           request for medical records to help us collect more health information related to care
           for their Rare CNS tumor diagnosis.

        -  Once the survey(s) are completed, the participant will be mailed a kit to collect saliva
           for germline DNA.
observational model: Cohort
time perspective: Prospective
measure: Relationship between health status and disease and treatment characteristics as well as clinical and demographic risk factors as self reported by adult participants with rare CNS tumors; and the relationship of genomic susceptibility of the popu...
time frame: completion of study
description: Obtain self-reported data on treatment, symptoms, functional status, and quality of life for adult participants with rare CNS tumors To evaluate the relationship between health status and disease and treatment characteristics. To evaluate self-reported clinical and demographic risk factors in adult participants in the rare CNS tumors participant population. To explore genomic susceptibility in participants with rare CNS tumors.
arm group label: 1/Patient with a rare CNS diagnosis
description: A diagnosis of rare CNS Tumors
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Terri S Armstrong, C.R.N.P.
role: Principal Investigator
affiliation: National Cancer Institute (NCI)
facility: 
country: United States
citation: Armstrong TS, Vera-Bolanos E, Gilbert MR. Clinical course of adult patients with ependymoma: results of the Adult Ependymoma Outcomes Project. Cancer. 2011 Nov 15;117(22):5133-41. doi: 10.1002/cncr.26181. Epub 2011 Apr 28.
PMID: 21538344
citation: Walbert T, Mendoza TR, Vera-Bolanos E, Acquaye A, Gilbert MR, Armstrong TS. Symptoms and socio-economic impact of ependymoma on adult patients: results of the Adult Ependymoma Outcomes Project 2. J Neurooncol. 2015 Jan;121(2):341-8. doi: 10.1007/s11060-014-1638-4. Epub 2014 Oct 31.
PMID: 25359395
citation: Acquaye AA, Vera E, Gilbert MR, Armstrong TS. Clinical presentation and outcomes for adult ependymoma patients. Cancer. 2017 Feb 1;123(3):494-501. doi: 10.1002/cncr.30355. Epub 2016 Sep 28.
PMID: 27679985
responsible party type: Sponsor
mesh term: Meningioma
mesh term: Ependymoma
mesh term: Medulloblastoma
sharing ipd: Yes
ipd description: .All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd time frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active. @@@@@@All collected IPD will be available after primary analysis have been published.
ipd access criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. @@@@@@Genomic data are made available via dbGaP through requests to the data custodians.