<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/NCT04488263</url>
  </required_header>
  <id_info>
    <org_study_id>200139</org_study_id>
    <secondary_id>20-C-0139</secondary_id>
    <nct_id>NCT04488263</nct_id>
  </id_info>
  <brief_title>Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NEN)s</brief_title>
  <official_title>Natural History Study of Children and Adults With Neuroendocrine Neoplasms (NENs)</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>
  <brief_summary>
    <textblock>
      Background:&#xD;
&#xD;
      Neuroendocrine neoplasms (NENs) are rare tumors that originate in neuroendocrine cells. NENs&#xD;
      can affect almost any part of the body. People with low-grade tumors can live many years. But&#xD;
      high-grade tumors can be very aggressive. Researchers want to learn more about this type of&#xD;
      cancer. This may help them design better treatments and supportive care studies.&#xD;
&#xD;
      Objective:&#xD;
&#xD;
      To gain a better understanding of neuroendocrine neoplasms.&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
      People starting at age 3 and older who have or are suspected of having NENs and are enrolled&#xD;
      in protocol 19C0016, Natural History and Biospecimen Acquisition Study for Children and&#xD;
      Adults with Rare Solid Tumors&#xD;
&#xD;
      Design:&#xD;
&#xD;
      Participants will be screened with questions about their medical history. This may be done&#xD;
      over the telephone or in person.&#xD;
&#xD;
      Participants medical records, test results, and imaging results will be reviewed. They may&#xD;
      have scans and blood tests. They may sign a separate consent form for some of the tests.&#xD;
&#xD;
      Participants will complete paper or electronic surveys. The surveys will ask about the&#xD;
      effects of cancer on their wellbeing.&#xD;
&#xD;
      Participants may give samples of their tumors from previous surgeries or biopsies. These&#xD;
      samples will be used to study their tumor genes.&#xD;
&#xD;
      Participants will get advice on how to manage their NENs. They will also get recommendations&#xD;
      about potential treatment options.&#xD;
&#xD;
      Participants home doctors will be contacted every 6 to 12 months. They will give medical data&#xD;
      such as imaging and test results.&#xD;
&#xD;
      Participants may have follow-up visits at NIH every 6 to 12 months.&#xD;
&#xD;
      Participants will contact researchers if there are any changes in their tumor.&#xD;
&#xD;
      Participants will be followed on this study for life.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Background:&#xD;
&#xD;
        -  Neuroendocrine neoplasm (NENs) are divided into neuroendocrine tumors (NETs) and&#xD;
           neuroendocrine carcinomas (NECs). These are rare malignancies occurring for example in&#xD;
           the gastrointestinal tract, islets of the pancreas, lung, adrenal medulla, thyroid&#xD;
           C-cells, etc. and are heterogeneous group of neoplasms with unique tumor biology,&#xD;
           natural history, and clinical management issues.&#xD;
&#xD;
        -  Their incidence has increased over the last decade, with an incidence of 6 per 100,000&#xD;
           persons a year and they represent 0.46% of all malignancies.&#xD;
&#xD;
        -  Most NETs are sporadic, but they can be part of familial cancer syndromes such as&#xD;
           multiple endocrine neoplasia type 1 (MEN1), neurofibromatosis type 1 (NF1) or Von&#xD;
           Hippel- Lindau (VHL) syndrome. Whereas poorly differentiated neuroendocrine carcinomas&#xD;
           (NECs) are all high-grade carcinomas that resemble small cell carcinoma or large cell&#xD;
           carcinoma of the lung.&#xD;
&#xD;
        -  Treatment for localized NETs is surgical resection, however, a variety of therapeutic&#xD;
           options are available for patients with advanced NETs. When to apply a given option,&#xD;
           what combination therapeutic approach should be used, how long treatment should be&#xD;
           continued should be used is unclear and controversial.&#xD;
&#xD;
      Objective:&#xD;
&#xD;
      -Characterize the natural history of neuroendocrine neoplasms (NENs). Data will include&#xD;
      clinical presentation, patterns of disease progression, response or lack of response to&#xD;
      therapeutic interventions, disease recurrence and overall survival.&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
        -  Subjects with confirmed or suspicion of NENs.&#xD;
&#xD;
        -  Age greater than or equal to 3 years old&#xD;
&#xD;
      Design:&#xD;
&#xD;
        -  This protocol is a subprotocol of protocol 19C0016 Natural History and Biospecimen&#xD;
           Accrual Study for Children and Adults with Rare Solid Tumors . After enrollment on the&#xD;
           master protocol and undergoing evaluations detailed in the master protocol, patients&#xD;
           will be enrolled on this subprotocol specific for NENs.&#xD;
&#xD;
        -  Medical histories will be documented, and patients followed throughout the course of&#xD;
           their illnesses, with particular attention to patterns of disease recurrence and&#xD;
           progression, response to therapies, duration of responses and hormone production in&#xD;
           patients with hormone production as a manifestation of their disease. Tumor growth rates&#xD;
           will also be calculated throughout the course of the disease.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    To avoid the duplication of effort and resources associated with the PI's branch change.&#xD;
  </why_stopped>
  <start_date type="Actual">November 24, 2020</start_date>
  <completion_date type="Actual">January 11, 2023</completion_date>
  <primary_completion_date type="Actual">September 6, 2022</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>natural history of neuroendocrine neoplasms</measure>
    <time_frame>ongoing</time_frame>
    <description>clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">30</enrollment>
  <condition>Neuroendocrine Tumors</condition>
  <condition>Carcinoma, Neuroendocrine</condition>
  <arm_group>
    <arm_group_label>Cohort 1</arm_group_label>
    <description>Subjects with confirmed/suspected NENs.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        primary clinical&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        -  INCLUSION CRITERIA:&#xD;
&#xD;
          -  Enrollment into NCI protocol 19C0016 'Natural History and Biospecimen Acquisition&#xD;
             Study for Children and Adults with Rare Solid Tumors'.&#xD;
&#xD;
          -  Age greater than or equal to 3 years old&#xD;
&#xD;
          -  Ability of subject or parent/guardian to understand and the willingness to sign a&#xD;
             written consent document.&#xD;
&#xD;
          -  Subjects with:&#xD;
&#xD;
             --histologically or cytologically documented NENs, OR&#xD;
&#xD;
          -  biochemical evidence of neuroendocrine tumor (serum/urinary) based on elevated levels&#xD;
             of chromogranin A, pancreatic polypeptide, neuron-specific enolase, vasoactive&#xD;
             intestinal polypeptide, serotonin (urinary 5-HIAA), gastrin, somatostatin,&#xD;
             catecholamines, metanephrines, calcitonin, fasting insulin, C-peptide (proinsulin),&#xD;
             glucagon, anterior pituitary hormones, OR&#xD;
&#xD;
          -  Suspicion of NEN (from any site/origin) on axial imaging (CT/MRI/FDG,&#xD;
             PET/68Ga-DOTATATE scan, FDOPA PET scan).&#xD;
&#xD;
        EXCLUSION CRITERIA:&#xD;
&#xD;
        None&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jaydira Del Rivero, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>National Cancer Institute (NCI)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>National Institutes of Health Clinical Center</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>
  <link>
    <url>https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2020-C-0139.html</url>
    <description>NIH Clinical Center Detailed Web Page</description>
  </link>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>July 24, 2020</study_first_submitted>
  <study_first_submitted_qc>July 24, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 27, 2020</study_first_posted>
  <last_update_submitted>January 13, 2023</last_update_submitted>
  <last_update_submitted_qc>January 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">January 17, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Clinical Outcome</keyword>
  <keyword>Advice in the Management of Cancer</keyword>
  <keyword>Patterns of Disease Progression</keyword>
  <keyword>Response or Lack of Response to Therapeutic Interventions</keyword>
  <keyword>Natural History</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neoplasms</mesh_term>
    <mesh_term>Neuroendocrine Tumors</mesh_term>
    <mesh_term>Carcinoma, Neuroendocrine</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/NCT04488263
org study id: 200139
secondary id: 20-C-0139
nct id: NCT04488263
lead sponsor: 
Background:

      Neuroendocrine neoplasms (NENs) are rare tumors that originate in neuroendocrine cells. NENs
      can affect almost any part of the body. People with low-grade tumors can live many years. But
      high-grade tumors can be very aggressive. Researchers want to learn more about this type of
      cancer. This may help them design better treatments and supportive care studies.

      Objective:

      To gain a better understanding of neuroendocrine neoplasms.

      Eligibility:

      People starting at age 3 and older who have or are suspected of having NENs and are enrolled
      in protocol 19C0016, Natural History and Biospecimen Acquisition Study for Children and
      Adults with Rare Solid Tumors

      Design:

      Participants will be screened with questions about their medical history. This may be done
      over the telephone or in person.

      Participants medical records, test results, and imaging results will be reviewed. They may
      have scans and blood tests. They may sign a separate consent form for some of the tests.

      Participants will complete paper or electronic surveys. The surveys will ask about the
      effects of cancer on their wellbeing.

      Participants may give samples of their tumors from previous surgeries or biopsies. These
      samples will be used to study their tumor genes.

      Participants will get advice on how to manage their NENs. They will also get recommendations
      about potential treatment options.

      Participants home doctors will be contacted every 6 to 12 months. They will give medical data
      such as imaging and test results.

      Participants may have follow-up visits at NIH every 6 to 12 months.

      Participants will contact researchers if there are any changes in their tumor.

      Participants will be followed on this study for life.
Background:

        -  Neuroendocrine neoplasm (NENs) are divided into neuroendocrine tumors (NETs) and
           neuroendocrine carcinomas (NECs). These are rare malignancies occurring for example in
           the gastrointestinal tract, islets of the pancreas, lung, adrenal medulla, thyroid
           C-cells, etc. and are heterogeneous group of neoplasms with unique tumor biology,
           natural history, and clinical management issues.

        -  Their incidence has increased over the last decade, with an incidence of 6 per 100,000
           persons a year and they represent 0.46% of all malignancies.

        -  Most NETs are sporadic, but they can be part of familial cancer syndromes such as
           multiple endocrine neoplasia type 1 (MEN1), neurofibromatosis type 1 (NF1) or Von
           Hippel- Lindau (VHL) syndrome. Whereas poorly differentiated neuroendocrine carcinomas
           (NECs) are all high-grade carcinomas that resemble small cell carcinoma or large cell
           carcinoma of the lung.

        -  Treatment for localized NETs is surgical resection, however, a variety of therapeutic
           options are available for patients with advanced NETs. When to apply a given option,
           what combination therapeutic approach should be used, how long treatment should be
           continued should be used is unclear and controversial.

      Objective:

      -Characterize the natural history of neuroendocrine neoplasms (NENs). Data will include
      clinical presentation, patterns of disease progression, response or lack of response to
      therapeutic interventions, disease recurrence and overall survival.

      Eligibility:

        -  Subjects with confirmed or suspicion of NENs.

        -  Age greater than or equal to 3 years old

      Design:

        -  This protocol is a subprotocol of protocol 19C0016 Natural History and Biospecimen
           Accrual Study for Children and Adults with Rare Solid Tumors . After enrollment on the
           master protocol and undergoing evaluations detailed in the master protocol, patients
           will be enrolled on this subprotocol specific for NENs.

        -  Medical histories will be documented, and patients followed throughout the course of
           their illnesses, with particular attention to patterns of disease recurrence and
           progression, response to therapies, duration of responses and hormone production in
           patients with hormone production as a manifestation of their disease. Tumor growth rates
           will also be calculated throughout the course of the disease.
observational model: Cohort
time perspective: Prospective
measure: natural history of neuroendocrine neoplasms
time frame: ongoing
description: clinical presentation, patterns of disease progression, response or lack of response to therapeutic interventions, disease recurrence and overall survival
arm group label: Cohort 1
description: Subjects with confirmed/suspected NENs.
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 3 Years
maximum age: N/A
healthy volunteers: No
last name: Jaydira Del Rivero, M.D.
role: Principal Investigator
affiliation: National Cancer Institute (NCI)
facility: 
country: United States
url: https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2020-C-0139.html
description: NIH Clinical Center Detailed Web Page
responsible party type: Sponsor
mesh term: Neoplasms
mesh term: Neuroendocrine Tumors
mesh term: Carcinoma, Neuroendocrine