<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/NCT02243592</url>
  </required_header>
  <id_info>
    <org_study_id>NCI-2014-01585</org_study_id>
    <secondary_id>NCI-2014-01585</secondary_id>
    <secondary_id>9671</secondary_id>
    <secondary_id>9671</secondary_id>
    <secondary_id>UM1CA186704</secondary_id>
    <nct_id>NCT02243592</nct_id>
    <nct_alias>NCT02496195</nct_alias>
  </id_info>
  <brief_title>Molecular Profiling in Tissue Samples From Patients With Cancer Who Are Exceptional Responders to Treatment</brief_title>
  <official_title>Exceptional Responders Pilot Study: Molecular Profiling of Tumors From Cancer Patients Who Are Exceptional Responders</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>National Cancer Institute (NCI)</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This pilot research trial studies molecular profiling in tissue samples from patients with&#xD;
      cancer who got better with treatment that didn't work for most other patients with the same&#xD;
      disease. Studying samples of tissue from patients with cancer in the laboratory may help&#xD;
      doctors learn more about changes that occur in deoxyribonucleic acid (DNA) and identify&#xD;
      biomarkers related to how well patients respond to treatment.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      PRIMARY OBJECTIVES:&#xD;
&#xD;
      I. To identify molecular indicators in malignant tissues from patients who were exceptional&#xD;
      responders on clinical trials or standard systemic treatments using whole exome and/or&#xD;
      targeted deep sequencing, as well as potentially other sequencing and other molecular&#xD;
      characterization methods (if adequate tissue exists).&#xD;
&#xD;
      II. To explore associations between the identified molecular indicators and the putative&#xD;
      mechanism of action of the treatment received by the patient.&#xD;
&#xD;
      III. To test the feasibility of identifying &quot;exceptional responders&quot;, obtaining the relevant&#xD;
      tumor and normal tissue and clinical data, and performing whole exome and/or targeted deep&#xD;
      sequencing on these samples.&#xD;
&#xD;
      OUTLINE:&#xD;
&#xD;
      Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted&#xD;
      next generation sequencing (NGS) assay deep sequencing. Cases for which sufficient nucleic&#xD;
      acid amounts are available will undergo additional analyses including whole genome&#xD;
      sequencing, messenger ribonucleic acid (RNA) (mRNA)-sequencing, micro (miRNA) sequencing,&#xD;
      promoter methylation analysis, and single nucleotide polymorphism (SNP) analysis.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">September 24, 2014</start_date>
  <completion_date type="Anticipated">March 22, 2024</completion_date>
  <primary_completion_date type="Actual">March 4, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Molecular features in tissue samples from patients who were exceptional responders</measure>
    <time_frame>Baseline</time_frame>
    <description>The molecular features of the tumors in the patients will be discernible as distinct to the tumor by comparison to (i) samples from normal tissue in the same patient and (ii) databases of similar data for normal and other tumor types.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses</measure>
    <time_frame>Baseline</time_frame>
    <description>The associations between identified molecular features and the putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses will be explored. Statistical analyses will be primarily descriptive.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of cases identified as potential exceptional responders</measure>
    <time_frame>Baseline</time_frame>
    <description>Statistical analyses will be primarily descriptive.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of identified potential cases confirmed to be exceptional responders</measure>
    <time_frame>Baseline</time_frame>
    <description>Statistical analyses will be primarily descriptive.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of confirmed exceptional responders for which adequate tissue with appropriate informed consent is acquired</measure>
    <time_frame>Baseline</time_frame>
    <description>Statistical analyses will be primarily descriptive.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of acquired cases with tissue for which at least the minimum molecular characterization is successfully obtained</measure>
    <time_frame>Baseline</time_frame>
    <description>Statistical analyses will be primarily descriptive.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of molecularly characterized cases for which a Molecular Characterization report identified (without reference to the drug received by the patient) at least one feature judged to have potential therapeutic relevance</measure>
    <time_frame>Baseline</time_frame>
    <description>Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by an expert panel (&quot;Panel&quot;) to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of cases with &gt;= 1 feature on Molecular Characterization report that was judged to have potential therapeutic relevance to the specific class of drug the patient actually received when the exceptional response was experienced</measure>
    <time_frame>Baseline</time_frame>
    <description>Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Percentage of cases with &gt;= 1 feature that correlates with the mechanism of action of the specific grant to which the exceptional response occurred that was found after further analyzing the molecular profile data for relevant molecular abnormalities</measure>
    <time_frame>Baseline</time_frame>
    <description>Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">170</enrollment>
  <condition>Malignant Neoplasm</condition>
  <arm_group>
    <arm_group_label>Ancillary-Correlative (molecular profiling)</arm_group_label>
    <description>Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted NGS assay deep sequencing. Cases for which sufficient nucleic acid amounts are available will undergo additional analyses including whole genome sequencing, mRNA-sequencing, miRNA sequencing, promoter methylation analysis, and SNP analysis.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Laboratory Biomarker Analysis</intervention_name>
    <description>Undergo sequencing and SNP analysis</description>
    <arm_group_label>Ancillary-Correlative (molecular profiling)</arm_group_label>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Tissue, blood, and buccal cells&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with cancer participating in the Exceptional Cases Initiative&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Documented exceptional response, defined as patients meeting the following criteria:&#xD;
&#xD;
               -  Complete response to a regimen in which complete response is expected in &lt; 10% of&#xD;
                  similarly treated patients&#xD;
&#xD;
               -  Partial response (PR) &gt; 6 months in a regimen in which PRs &gt; 6 months are&#xD;
                  expected in &lt; 10% of patients with similar disease treated with same or similar&#xD;
                  regimen&#xD;
&#xD;
               -  Complete response (CR) or PR of unusual duration, such that the internal review&#xD;
                  committee considers it to be an exceptional response; examples below:&#xD;
&#xD;
                    -  PR of duration &gt; 3 x the median expected PR duration (in cases where PR is&#xD;
                       expected in &gt; 10% of patients with the same disease treated with the same&#xD;
                       regimen)&#xD;
&#xD;
                    -  CR or duration &gt; 3 x the median expected CR duration (in cases where CR may&#xD;
                       be seen in &gt; 10% of patients with same disease treated with same regimen)&#xD;
&#xD;
                    -  The observed duration of CR (or PR) is longer than expected for 90% of&#xD;
                       patients with same disease treated with same regimen&#xD;
&#xD;
               -  Note: it is not required that the patient be enrolled on a clinical trial when&#xD;
                  the exceptional response was observed&#xD;
&#xD;
          -  Reports of radiologic scans or other evidence documenting response will be submitted&#xD;
             for review; cases where response is not assessable (e.g. adjuvant treatment) will not&#xD;
             be eligible because the outcome can not be attributed to a specific treatment&#xD;
&#xD;
          -  Treatment history must be available, for prior treatment and for the drug to which the&#xD;
             exceptional response occurred&#xD;
&#xD;
          -  Patient must meet consent criteria; this requires: (i) current exceptional responder&#xD;
             (ER) consent by a living participant not lost to follow-up, (ii) prior consent for&#xD;
             future research by a participant not known to be deceased, but lost to follow-up, or&#xD;
             (iii) if patient is deceased and did not decline to participate in research at the&#xD;
             time of tissue removal for any tissue that would be used in this study, then no&#xD;
             consent is required&#xD;
&#xD;
          -  Tumor sample available that meets study requirements&#xD;
&#xD;
          -  Required tumor samples MUST exist and be able to be submitted; investigators wishing&#xD;
             to submit samples must not have made agreements that would prohibit the free use of&#xD;
             data from such samples; the National Cancer Institute (NCI) will provide investigators&#xD;
             with a letter for the collaborator amending their existing agreement to allow for the&#xD;
             case to be submitted&#xD;
&#xD;
               -  Tumor tissue from prior to administration of the drug to which the exceptional&#xD;
                  response occurred is required; ideally this sample will have been collected just&#xD;
                  prior to treatment, but other prior tissue will be considered; tissue may be&#xD;
                  fresh frozen or formalin-fixed paraffin embedded&#xD;
&#xD;
               -  Tumor tissue amount must be at least a core biopsy, and meet minimum specimen&#xD;
                  requirements&#xD;
&#xD;
          -  Encouraged: normal tissue sample: (optional): blood or other specimen source for&#xD;
             germline sequencing&#xD;
&#xD;
          -  The tumor samples and clinical data submitted to the Exceptional Responders Database&#xD;
             in database in Genotypes and Phenotypes (dbGaP) will need to have appropriate&#xD;
             agreements in place to allow for the submission; the Exceptional Responders Database&#xD;
             can accept clinical data and samples from cases enrolled on a Cancer Therapy&#xD;
             Evaluation Program (CTEP) sponsored clinical trial and cases that were not enrolled on&#xD;
             any clinical trial; if the response occurred on a trial that was not CTEP-sponsored,&#xD;
             there are existing agreements between the submitting site and the pharmaceutical&#xD;
             company; if existing agreements do not allow for the submission of sample and clinical&#xD;
             data, the NCI will provide the investigators with a letter that allows the tissue to&#xD;
             be used for the exceptional responders study if signed by the appropriate&#xD;
             collaborator; the letter modifies the existing agreement to include the CTEP&#xD;
             Intellectual Property (IP) Option language that would allow the case to be submitted&#xD;
             to the Exceptional Responders Database; if the existing agreement cannot be modified&#xD;
             and the letter cannot be signed, the proposed case will not be accepted; Note: as&#xD;
             stated above, the patient does not need to have been enrolled on a clinical trial to&#xD;
             be eligible for the exceptional responders study&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patient's response did not meet criteria for an exceptional response&#xD;
&#xD;
          -  Patient's treatment regimen is expected to lead to CR or durable PR in &gt; 10% of&#xD;
             patients&#xD;
&#xD;
          -  Patient's duration of response is not &gt; 3 x expected median length of response&#xD;
&#xD;
          -  Response not evaluable or not able to be attributed to systemic treatment (e.g.&#xD;
             adjuvant treatment)&#xD;
&#xD;
          -  Patient refused consent for use of tissue for research activities included in the&#xD;
             exceptional responders study&#xD;
&#xD;
          -  Tumor sample from prior to the exceptional response is not available, or does not meet&#xD;
             quality metrics&#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>S. P Ivy</last_name>
    <role>Principal Investigator</role>
    <affiliation>National Cancer Institute (NCI)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>City of Hope Comprehensive Cancer Center</name>
      <address>
        <city>Duarte</city>
        <state>California</state>
        <zip>91010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Fresno</name>
      <address>
        <city>Fresno</city>
        <state>California</state>
        <zip>93720</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Oakland</name>
      <address>
        <city>Oakland</city>
        <state>California</state>
        <zip>94611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Richmond</name>
      <address>
        <city>Richmond</city>
        <state>California</state>
        <zip>94801</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCSF Medical Center-Mission Bay</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94158</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente Medical Center - Santa Clara</name>
      <address>
        <city>Santa Clara</city>
        <state>California</state>
        <zip>95051</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Santa Rosa</name>
      <address>
        <city>Santa Rosa</city>
        <state>California</state>
        <zip>95403</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Stockton</name>
      <address>
        <city>Stockton</city>
        <state>California</state>
        <zip>95210</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente-Walnut Creek</name>
      <address>
        <city>Walnut Creek</city>
        <state>California</state>
        <zip>94596</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCHealth University of Colorado Hospital</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Poudre Valley Hospital</name>
      <address>
        <city>Fort Collins</city>
        <state>Colorado</state>
        <zip>80524</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Greenwich Hospital</name>
      <address>
        <city>Greenwich</city>
        <state>Connecticut</state>
        <zip>06830</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Helen F Graham Cancer Center</name>
      <address>
        <city>Newark</city>
        <state>Delaware</state>
        <zip>19713</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical Oncology Hematology Consultants PA</name>
      <address>
        <city>Newark</city>
        <state>Delaware</state>
        <zip>19713</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sibley Memorial Hospital</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory University Hospital/Winship Cancer Institute</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>John B Amos Cancer Center</name>
      <address>
        <city>Columbus</city>
        <state>Georgia</state>
        <zip>31904</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>South Georgia Medical Center/Pearlman Cancer Center</name>
      <address>
        <city>Valdosta</city>
        <state>Georgia</state>
        <zip>31602</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Alphonsus Cancer Care Center-Boise</name>
      <address>
        <city>Boise</city>
        <state>Idaho</state>
        <zip>83706</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rush University Medical Center</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Presence Resurrection Medical Center</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60631</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cancer Care Specialists of Illinois - Decatur</name>
      <address>
        <city>Decatur</city>
        <state>Illinois</state>
        <zip>62526</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Decatur Memorial Hospital</name>
      <address>
        <city>Decatur</city>
        <state>Illinois</state>
        <zip>62526</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>NorthShore University HealthSystem-Evanston Hospital</name>
      <address>
        <city>Evanston</city>
        <state>Illinois</state>
        <zip>60201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Illinois CancerCare-Peoria</name>
      <address>
        <city>Peoria</city>
        <state>Illinois</state>
        <zip>61615</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Springfield Clinic</name>
      <address>
        <city>Springfield</city>
        <state>Illinois</state>
        <zip>62702</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Physicians' Clinic of Iowa PC</name>
      <address>
        <city>Cedar Rapids</city>
        <state>Iowa</state>
        <zip>52402</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>York Hospital</name>
      <address>
        <city>York</city>
        <state>Maine</state>
        <zip>03909</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National Cancer Institute Developmental Therapeutics Clinic</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>NCI - Center for Cancer Research</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Joseph Mercy Hospital</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Comprehensive Cancer Center</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Joseph Mercy Brighton</name>
      <address>
        <city>Brighton</city>
        <state>Michigan</state>
        <zip>48114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wayne State University/Karmanos Cancer Institute</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Spectrum Health at Butterworth Campus</name>
      <address>
        <city>Grand Rapids</city>
        <state>Michigan</state>
        <zip>49503</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UP Health System Hematology Oncology Marquette</name>
      <address>
        <city>Marquette</city>
        <state>Michigan</state>
        <zip>49855</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UP Health System Marquette</name>
      <address>
        <city>Marquette</city>
        <state>Michigan</state>
        <zip>49855</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ascension Providence Hospitals - Southfield</name>
      <address>
        <city>Southfield</city>
        <state>Michigan</state>
        <zip>48075</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Francis Medical Center</name>
      <address>
        <city>Cape Girardeau</city>
        <state>Missouri</state>
        <zip>63703</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University School of Medicine</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Missouri Baptist Medical Center</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63131</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mercy Hospital Saint Louis</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63141</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mercy Hospital Springfield</name>
      <address>
        <city>Springfield</city>
        <state>Missouri</state>
        <zip>65804</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CoxHealth South Hospital</name>
      <address>
        <city>Springfield</city>
        <state>Missouri</state>
        <zip>65807</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Benefis Healthcare- Sletten Cancer Institute</name>
      <address>
        <city>Great Falls</city>
        <state>Montana</state>
        <zip>59405</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Portsmouth Regional Hospital</name>
      <address>
        <city>Portsmouth</city>
        <state>New Hampshire</state>
        <zip>03802</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of New Mexico Cancer Center</name>
      <address>
        <city>Albuquerque</city>
        <state>New Mexico</state>
        <zip>87102</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Rochester</name>
      <address>
        <city>Rochester</city>
        <state>New York</state>
        <zip>14642</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Duke University Medical Center</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wake Forest University Health Sciences</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27157</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sanford Roger Maris Cancer Center</name>
      <address>
        <city>Fargo</city>
        <state>North Dakota</state>
        <zip>58122</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riverside Methodist Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43214</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cancer Centers of Southwest Oklahoma Research</name>
      <address>
        <city>Lawton</city>
        <state>Oklahoma</state>
        <zip>73505</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Oklahoma Health Sciences Center</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaiser Permanente Northwest</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97227</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Phoenixville Hospital</name>
      <address>
        <city>Phoenixville</city>
        <state>Pennsylvania</state>
        <zip>19460</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Chester County Hospital</name>
      <address>
        <city>West Chester</city>
        <state>Pennsylvania</state>
        <zip>19380</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Reading Hospital</name>
      <address>
        <city>West Reading</city>
        <state>Pennsylvania</state>
        <zip>19611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Prisma Health Cancer Institute - Butternut</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29605</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Prisma Health Cancer Institute - Faris</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29605</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Prisma Health Cancer Institute - Seneca</name>
      <address>
        <city>Seneca</city>
        <state>South Carolina</state>
        <zip>29672</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sanford Cancer Center Oncology Clinic</name>
      <address>
        <city>Sioux Falls</city>
        <state>South Dakota</state>
        <zip>57104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sanford USD Medical Center - Sioux Falls</name>
      <address>
        <city>Sioux Falls</city>
        <state>South Dakota</state>
        <zip>57117-5134</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wellmont Holston Valley Hospital and Medical Center</name>
      <address>
        <city>Kingsport</city>
        <state>Tennessee</state>
        <zip>37660</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ben Taub General Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Texas Health Science Center at San Antonio</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Huntsman Cancer Institute/University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Virginia Cancer Center</name>
      <address>
        <city>Charlottesville</city>
        <state>Virginia</state>
        <zip>22908</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>WVUH-Berkely Medical Center</name>
      <address>
        <city>Martinsburg</city>
        <state>West Virginia</state>
        <zip>25401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Wisconsin Carbone Cancer Center</name>
      <address>
        <city>Madison</city>
        <state>Wisconsin</state>
        <zip>53792</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Aurora Medical Center in Summit</name>
      <address>
        <city>Summit</city>
        <state>Wisconsin</state>
        <zip>53066</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UW Cancer Center at ProHealth Care</name>
      <address>
        <city>Waukesha</city>
        <state>Wisconsin</state>
        <zip>53188</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>September 16, 2014</study_first_submitted>
  <study_first_submitted_qc>September 16, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 18, 2014</study_first_posted>
  <last_update_submitted>September 9, 2023</last_update_submitted>
  <last_update_submitted_qc>September 9, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 13, 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>Neoplasms</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/NCT02243592
org study id: NCI-2014-01585
secondary id: NCI-2014-01585
secondary id: 9671
secondary id: 9671
secondary id: UM1CA186704
nct id: NCT02243592
nct alias: NCT02496195
lead sponsor: 
has dmc: Yes
This pilot research trial studies molecular profiling in tissue samples from patients with
      cancer who got better with treatment that didn't work for most other patients with the same
      disease. Studying samples of tissue from patients with cancer in the laboratory may help
      doctors learn more about changes that occur in deoxyribonucleic acid (DNA) and identify
      biomarkers related to how well patients respond to treatment.
PRIMARY OBJECTIVES:

      I. To identify molecular indicators in malignant tissues from patients who were exceptional
      responders on clinical trials or standard systemic treatments using whole exome and/or
      targeted deep sequencing, as well as potentially other sequencing and other molecular
      characterization methods (if adequate tissue exists).

      II. To explore associations between the identified molecular indicators and the putative
      mechanism of action of the treatment received by the patient.

      III. To test the feasibility of identifying "exceptional responders", obtaining the relevant
      tumor and normal tissue and clinical data, and performing whole exome and/or targeted deep
      sequencing on these samples.

      OUTLINE:

      Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted
      next generation sequencing (NGS) assay deep sequencing. Cases for which sufficient nucleic
      acid amounts are available will undergo additional analyses including whole genome
      sequencing, messenger ribonucleic acid (RNA) (mRNA)-sequencing, micro (miRNA) sequencing,
      promoter methylation analysis, and single nucleotide polymorphism (SNP) analysis.
observational model: Case-Control
time perspective: Retrospective
measure: Molecular features in tissue samples from patients who were exceptional responders
time frame: Baseline
description: The molecular features of the tumors in the patients will be discernible as distinct to the tumor by comparison to (i) samples from normal tissue in the same patient and (ii) databases of similar data for normal and other tumor types.
measure: Putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses
time frame: Baseline
description: The associations between identified molecular features and the putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses will be explored. Statistical analyses will be primarily descriptive.
measure: Number of cases identified as potential exceptional responders
time frame: Baseline
description: Statistical analyses will be primarily descriptive.
measure: Percentage of identified potential cases confirmed to be exceptional responders
time frame: Baseline
description: Statistical analyses will be primarily descriptive.
measure: Percentage of confirmed exceptional responders for which adequate tissue with appropriate informed consent is acquired
time frame: Baseline
description: Statistical analyses will be primarily descriptive.
measure: Percentage of acquired cases with tissue for which at least the minimum molecular characterization is successfully obtained
time frame: Baseline
description: Statistical analyses will be primarily descriptive.
measure: Percentage of molecularly characterized cases for which a Molecular Characterization report identified (without reference to the drug received by the patient) at least one feature judged to have potential therapeutic relevance
time frame: Baseline
description: Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by an expert panel ("Panel") to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
measure: Percentage of cases with >= 1 feature on Molecular Characterization report that was judged to have potential therapeutic relevance to the specific class of drug the patient actually received when the exceptional response was experienced
time frame: Baseline
description: Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
measure: Percentage of cases with >= 1 feature that correlates with the mechanism of action of the specific grant to which the exceptional response occurred that was found after further analyzing the molecular profile data for relevant molecular abnormalities
time frame: Baseline
description: Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
arm group label: Ancillary-Correlative (molecular profiling)
description: Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted NGS assay deep sequencing. Cases for which sufficient nucleic acid amounts are available will undergo additional analyses including whole genome sequencing, mRNA-sequencing, miRNA sequencing, promoter methylation analysis, and SNP analysis.
intervention type: Other
intervention name: Laboratory Biomarker Analysis
description: Undergo sequencing and SNP analysis
arm group label: Ancillary-Correlative (molecular profiling)
Tissue, blood, and buccal cells
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: S. P Ivy
role: Principal Investigator
affiliation: National Cancer Institute (NCI)
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Neoplasms