<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/NCT02242552</url>
  </required_header>
  <id_info>
    <org_study_id>Triceps</org_study_id>
    <nct_id>NCT02242552</nct_id>
  </id_info>
  <brief_title>Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood</brief_title>
  <official_title>Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood(TRICEPS Study)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>St. Justine's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>St. Justine's Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      A new research paradigm that involves sequencing tumor DNA/RNA to identify driver mutations,&#xD;
      select among the Health Canada approved drugs (for adult cancers) known to block certain&#xD;
      oncogenic pathways, and recommend these drugs to the treating physician, without taking into&#xD;
      account the tumor histology.&#xD;
&#xD;
      In this paradigm, the treatment is targeted to the actionable mutation(s) i.e. those driving&#xD;
      oncogenesis. It is also personalized to the molecular signature of the patient's tumor,&#xD;
      irrespective of its histopathological subtype. The experience of the investigators team in&#xD;
      genomics, including next generation sequencing and bioinformatic analysis combined with the&#xD;
      clinical expertise, bring at last this approach within our technical capacities. In parallel,&#xD;
      the number of Health Canada-approved drugs (which have been tested in a pediatric setting)&#xD;
      designed to interfere with oncogenesis pathways is increasing exponentially.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>June 2014</start_date>
  <completion_date type="Anticipated">June 2017</completion_date>
  <primary_completion_date type="Anticipated">June 2016</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.</measure>
    <time_frame>24 months</time_frame>
    <description>The study team will evaluate the timeline between decision of biopsy, the actual biopsy, availability of results of the whole-genome analysis, interpretation of results and divulgation of results to patient and family.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Feasibility of performing whole genome sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within 10-week time frame from diagnosis</measure>
    <time_frame>24 months</time_frame>
  </secondary_outcome>
  <enrollment type="Anticipated">30</enrollment>
  <condition>Pediatric Cancer</condition>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood, saliva, tumor sample (bone marrow or solid tumors)&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Children and adolescents with Relapse or refractory leukemia and solid tumor&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        At the time of enrollment:&#xD;
&#xD;
          -  21 year-old or less&#xD;
&#xD;
          -  Poor prognosis biopsy-proven cancer of any type :&#xD;
&#xD;
          -  Cancer (at initial diagnosis) known to be refractory to treatment&#xD;
&#xD;
          -  Or cancer refractory to treatment&#xD;
&#xD;
          -  Or relapsed cancer&#xD;
&#xD;
          -  Written informed consent by patient, parents, or the legal guardians&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Estimated life expectancy of less than 3 months.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>21 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Monia Marzouki, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>St. Justine's Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Monia Marzouki, MD</last_name>
    <phone>514-345-4931</phone>
    <phone_ext>6316</phone_ext>
    <email>monia.marzouki@umontreal.ca</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Hemrique Bittencourt, MD</last_name>
    <phone>514-345-4931</phone>
    <phone_ext>2724</phone_ext>
    <email>henrique.bittencourt@umontreal.ca</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>CHU Sainte-Justine</name>
      <address>
        <city>Montréal</city>
        <state>Quebec</state>
        <zip>H3T1C5</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dominique Lafrenière, BScN, DESS</last_name>
      <phone>514-345-4969</phone>
      <email>dominique.lafreniere@recherche-ste-justine.qc.ca</email>
    </contact>
    <contact_backup>
      <last_name>Mary-Ellen French, RN</last_name>
      <phone>514-345-4969</phone>
      <email>mary-ellen.french@recherche-ste-justine.qc.ca</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>Canada</country>
  </location_countries>
  <verification_date>August 2014</verification_date>
  <study_first_submitted>August 15, 2014</study_first_submitted>
  <study_first_submitted_qc>September 15, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 17, 2014</study_first_posted>
  <last_update_submitted>September 15, 2014</last_update_submitted>
  <last_update_submitted_qc>September 15, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">September 17, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>St. Justine's Hospital</investigator_affiliation>
    <investigator_full_name>Dr Monia Marzouki</investigator_full_name>
    <investigator_title>Hematologist/Oncologist</investigator_title>
  </responsible_party>
  <!-- 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/NCT02242552
org study id: Triceps
nct id: NCT02242552
lead sponsor: 
has dmc: Yes
A new research paradigm that involves sequencing tumor DNA/RNA to identify driver mutations,
      select among the Health Canada approved drugs (for adult cancers) known to block certain
      oncogenic pathways, and recommend these drugs to the treating physician, without taking into
      account the tumor histology.

      In this paradigm, the treatment is targeted to the actionable mutation(s) i.e. those driving
      oncogenesis. It is also personalized to the molecular signature of the patient's tumor,
      irrespective of its histopathological subtype. The experience of the investigators team in
      genomics, including next generation sequencing and bioinformatic analysis combined with the
      clinical expertise, bring at last this approach within our technical capacities. In parallel,
      the number of Health Canada-approved drugs (which have been tested in a pediatric setting)
      designed to interfere with oncogenesis pathways is increasing exponentially.
observational model: Case-Only
time perspective: Prospective
measure: Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.
time frame: 24 months
description: The study team will evaluate the timeline between decision of biopsy, the actual biopsy, availability of results of the whole-genome analysis, interpretation of results and divulgation of results to patient and family.
measure: Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.
time frame: 24 months
measure: Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.
time frame: 24 months
measure: Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.
time frame: 24 months
measure: Feasibility of performing whole genome sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within 10-week time frame from diagnosis
time frame: 24 months
Blood, saliva, tumor sample (bone marrow or solid tumors)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 1 Month
maximum age: 21 Years
healthy volunteers: No
last name: Monia Marzouki, MD
role: Principal Investigator
affiliation: St. Justine's Hospital
last name: Monia Marzouki, MD
phone: 514-345-4931
phone ext: 6316
email: monia.marzouki@umontreal.ca
last name: Hemrique Bittencourt, MD
phone: 514-345-4931
phone ext: 2724
email: henrique.bittencourt@umontreal.ca
facility: 
status: Recruiting
contact: 
contact backup: 
country: Canada
responsible party type: Principal Investigator
investigator affiliation: St. Justine's Hospital
investigator full name: Dr Monia Marzouki
investigator title: Hematologist/Oncologist