<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/NCT01685619</url>
  </required_header>
  <id_info>
    <org_study_id>AML-MDS 01-2011</org_study_id>
    <nct_id>NCT01685619</nct_id>
  </id_info>
  <brief_title>AML-MDS Novel Prognostic Tests Clinical Study</brief_title>
  <official_title>A Multi-Centre Observational Prospective Cohort Study Involving the Collection of Clinical Information and Biological Specimens for the Evaluation of Novel Prognostic Tests for Myelodysplasia and Acute Myeloid Leukemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Nova Scotia Health Authority</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Terry Fox Research Institute</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Nova Scotia Health Authority</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This clinical study will provide the study specimens (samples of bone marrow and blood) and&#xD;
      the clinical data for a pan-Canadian collaborative research project developed by the MDS/AML&#xD;
      Research Consortium. The goal of this project involves the evaluation and potential&#xD;
      validation of five novel prognostic tests for myelodysplasia (MDS) and/or acute myeloid&#xD;
      leukemia (AML), as well as an analysis of health economic and socio-ethical implications&#xD;
      related to the potential introduction of these tests into the clinical setting. The&#xD;
      over-arching goal is to improve the outcomes of patients with MDS and AML. The primary&#xD;
      hypothesis is that one or more of the laboratory tests being evaluated in conjunction with&#xD;
      this study, either alone or in combination with other laboratory tests (either established or&#xD;
      under investigation in this project), will have statistically significant prognostic value&#xD;
      either alone or in combination with established clinical risk factors.&#xD;
&#xD;
      The clinical study will involve the enrollment of 200 adults with AML and 200 adults with MDS&#xD;
      over a 2.5 year period. Participants will be followed on study for two years. Bone marrow and&#xD;
      blood specimens will be collected at diagnosis and at other time points as required for the&#xD;
      development of the five laboratory tests.&#xD;
&#xD;
      Participants will be assigned to treatment according to local institutional practice and will&#xD;
      be followed for up to 2 years. Health economic and quality of life questionnaires will be&#xD;
      administered at key time points. Data will be collected regarding participant&#xD;
      characteristics, diagnosis, disease features, treatment and clinical outcome.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Two of the tests involve a technology called flow cytometry. Both of the flow cytometry tests&#xD;
      are used to predict whether a person is likely to have a good response to chemotherapy or&#xD;
      not.&#xD;
&#xD;
      Three of the tests involve new genetic-based technology. One of these tests is called&#xD;
      comparative genomic profiling. This test can detect genetic abnormalities that current&#xD;
      testing methods are not able to detect. Another test involves micro-RNA profiling. The final&#xD;
      test involves RNA sequencing. The researchers think these tests might be useful in predicting&#xD;
      how well a person will respond to treatment.&#xD;
&#xD;
      The novel laboratory tests being evaluated as part of this study are still in the early&#xD;
      phases of development and cannot be used for clinical decision making. Participants enrolled&#xD;
      in this study will not be informed regarding their individual results with respect to the&#xD;
      study tests that are conducted using their biospecimens.&#xD;
&#xD;
      The following information (data) will be collected regarding study participants: diagnosis,&#xD;
      results of relevant clinical tests, age, gender, treatment and outcome during the 2 year&#xD;
      study follow-up period. The study also involves the completion of study questionnaires at six&#xD;
      different time points over the course of the two year study follow-up.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 2, 2012</start_date>
  <completion_date type="Actual">January 24, 2018</completion_date>
  <primary_completion_date type="Actual">January 24, 2018</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>Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.</measure>
    <time_frame>Two years following the completion of enrollment.</time_frame>
    <description>For the AML cohort, the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, white blood cell count at presentation, antecedent hematologic disorder, FLT-3 status, Karnofsky Performance Status (KPS), cytogenetic sub-group (using WHO 2008). For the MDS cohort the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, KPS, karyotype, bone marrow blast count and number of cytopenias at diagnosis.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Cost impact of candidate tests.</measure>
    <time_frame>Two years following the completion of enrollment.</time_frame>
    <description>The cost-effectiveness of the candidate tests for AML and MDS treatment will be projected with the aid of economic simulation models.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Societal risks and benefits related to the candidate tests.</measure>
    <time_frame>Two years following the completion of enrollment.</time_frame>
    <description>A comparative analysis of policies and regulations in Canada governing prognostic tests will be undertaken (including tests that utilize RNA and DNA based technologies). In addition, input will be obtained from key stakeholders. This information will be used to develop a set of guidelines and best practices for this type of research.</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">11</enrollment>
  <condition>Acute Myeloid Leukemia (AML)</condition>
  <condition>Myelodysplastic Syndrome (MDS)</condition>
  <arm_group>
    <arm_group_label>AML Cohort</arm_group_label>
    <description>This cohort is comprised of participants who have acute myelogenous leukemia (AML).</description>
  </arm_group>
  <arm_group>
    <arm_group_label>MDS Cohort</arm_group_label>
    <description>This cohort is comprised of participants who have myelodysplastic syndrome (MDS).</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Some of the laboratory tests that will be evaluated as part of this study will utilize RNA&#xD;
      and/or DNA. There may be biospecimens left over after the work related to this study is&#xD;
      complete. At the time of consent for the study, participants will be asked to provide consent&#xD;
      (or decline their consent) regarding the use of leftover specimens (including RNA and DNA)&#xD;
      for other research. The details regarding this other research are provided in the protocol&#xD;
      and consent template.&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Prospective participants will include patients with known or supsected AML or MDS who are&#xD;
        being treated and/or assessed at a participating site.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        There are two parts to the study: Part One (Collection of Biospecimens) and Part Two (Two&#xD;
        year follow-up that includes data collection regarding treatment, outcome and questionnaire&#xD;
        completion).&#xD;
&#xD;
        INCLUSION CRITERIA (PART ONE):&#xD;
&#xD;
        Prospective participants can be included in the study if:&#xD;
&#xD;
          -  The participant is 18 years of age or older&#xD;
&#xD;
          -  The participant is suspected to have a new diagnosis of MDS (including CMML) , OR&#xD;
             suspected to have a new diagnosis of AML excluding acute promyelocytic leukemia (APL),&#xD;
             OR known to have a diagnosis of MDS (including CMML) confirmed by bone marrow aspirate&#xD;
             and biopsy no more than one year prior to the date of enrollment AND without&#xD;
             commencement of definitive therapy prior to enrollment&#xD;
&#xD;
          -  The participant is scheduled to have a diagnostic or confirmatory bone marrow aspirate&#xD;
             and biopsy at a participating site, or in the case of prospective participants with an&#xD;
             established diagnosis of MDS (including CMML), must be able to undergo a bone marrow&#xD;
             aspirate for the study at the participating site&#xD;
&#xD;
          -  The participant must be able to read and/or understand spoken English or French so&#xD;
             that they will be eligible for Part Two of the study&#xD;
&#xD;
          -  The participant must be able to understand and sign the informed consent form&#xD;
             applicable to their situation&#xD;
&#xD;
        EXCLUSION CRITERIA (PART ONE):&#xD;
&#xD;
        Prospective participants should be excluded from the study if:&#xD;
&#xD;
          -  The participant has already received definitive therapy for AML or MDS&#xD;
&#xD;
          -  The participant has a diagnosis of MDS that was confirmed more than one year prior to&#xD;
             the date of enrollment&#xD;
&#xD;
        INCLUSION CRITERIA (PART TWO):&#xD;
&#xD;
        Participants who have been enrolled in Part One will be eligible to participate in the full&#xD;
        two year study follow-up component if they meet the following criteria:&#xD;
&#xD;
          -  Confirmed diagnosis of either MDS, CMML or AML (excluding APL)&#xD;
&#xD;
          -  Sufficient cell count for the MDS/AML Clinical Study requirements as follows:&#xD;
&#xD;
        For participants with suspected (or known) AML:&#xD;
&#xD;
        The blast count of the peripheral blood taken at diagnosis must be greater than 1 x10^6&#xD;
        blast count/mL&#xD;
&#xD;
          -  It must be possible to earmark for the MDS/AML Study:&#xD;
&#xD;
          -  3 vials 1.0 x 10^7/mL mononuclear peripheral blood cells&#xD;
&#xD;
          -  1 vial 0.5 x 10^7/mL mononuclear bone marrow cells&#xD;
&#xD;
        Cells are to be prepared according to the site's local cell bank procedures so that they&#xD;
        can be stored and transported to study labs as needed.&#xD;
&#xD;
        At sites participating in the Hogge Assay:&#xD;
&#xD;
        In addition to the specimens described above, it must be possible to provide 2 mL of fresh&#xD;
        bone marrow or 5 mL of fresh peripheral blood with &gt; 1 x 10^6 blast count/mL&#xD;
&#xD;
        For participants with suspected (or known) MDS:&#xD;
&#xD;
          -  It must be possible to earmark for the MDS/AML Study:&#xD;
&#xD;
          -  2 vials 1.0 x 10^7/mL mononuclear peripheral blood cells&#xD;
&#xD;
          -  2 vials 1.0 x 10^7/mL mononuclear bone marrow cells&#xD;
&#xD;
        Cells are to be prepared according to the site's local cell bank procedures so that they&#xD;
        can be stored and transported to study labs as needed.&#xD;
&#xD;
        EXCLUSION CRITERIA (PART TWO):&#xD;
&#xD;
        Participants who have been enrolled in Part One will not be eligible to participate in the&#xD;
        full two year study follow-up component if they:&#xD;
&#xD;
          -  Do not have sufficient cell count for the MDS/AML Clinical Study requirements as set&#xD;
             out in Section 11.3&#xD;
&#xD;
          -  It is confirmed after enrollment that they do not have a diagnosis of MDS, CMML or AML&#xD;
&#xD;
          -  A diagnosis of APL is confirmed&#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>Stephen Couban, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Nova Scotia Health Authority</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Tom Baker Cancer Centre</name>
      <address>
        <city>Calgary</city>
        <state>Alberta</state>
        <zip>T2N 4N2</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vancouver General Hospital</name>
      <address>
        <city>Vancouver</city>
        <state>British Columbia</state>
        <zip>V5Z 4E3</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CancerCare Manitoba</name>
      <address>
        <city>Winnipeg</city>
        <state>Manitoba</state>
        <zip>R3M 1A5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queen Elizabeth II Health Sciences Centre</name>
      <address>
        <city>Halifax</city>
        <state>Nova Scotia</state>
        <zip>B3H 2Y9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Princess Margaret Cancer Centre (formerly Princess Margaret Hospital)</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 2M9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Maisonneuve-Rosemont</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H1T 2M4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
  </location_countries>
  <verification_date>November 2022</verification_date>
  <study_first_submitted>September 12, 2012</study_first_submitted>
  <study_first_submitted_qc>September 12, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 14, 2012</study_first_posted>
  <last_update_submitted>November 4, 2022</last_update_submitted>
  <last_update_submitted_qc>November 4, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">November 8, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>AML</keyword>
  <keyword>Acute Myeloid Leukemia</keyword>
  <keyword>MDS</keyword>
  <keyword>Myelodysplastic Syndrome</keyword>
  <keyword>Prognostic</keyword>
  <keyword>Economic</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Leukemia, Myeloid</mesh_term>
    <mesh_term>Leukemia, Myeloid, Acute</mesh_term>
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</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/NCT01685619
org study id: AML-MDS 01-2011
nct id: NCT01685619
lead sponsor: 
collaborator: 
has dmc: Yes
This clinical study will provide the study specimens (samples of bone marrow and blood) and
      the clinical data for a pan-Canadian collaborative research project developed by the MDS/AML
      Research Consortium. The goal of this project involves the evaluation and potential
      validation of five novel prognostic tests for myelodysplasia (MDS) and/or acute myeloid
      leukemia (AML), as well as an analysis of health economic and socio-ethical implications
      related to the potential introduction of these tests into the clinical setting. The
      over-arching goal is to improve the outcomes of patients with MDS and AML. The primary
      hypothesis is that one or more of the laboratory tests being evaluated in conjunction with
      this study, either alone or in combination with other laboratory tests (either established or
      under investigation in this project), will have statistically significant prognostic value
      either alone or in combination with established clinical risk factors.

      The clinical study will involve the enrollment of 200 adults with AML and 200 adults with MDS
      over a 2.5 year period. Participants will be followed on study for two years. Bone marrow and
      blood specimens will be collected at diagnosis and at other time points as required for the
      development of the five laboratory tests.

      Participants will be assigned to treatment according to local institutional practice and will
      be followed for up to 2 years. Health economic and quality of life questionnaires will be
      administered at key time points. Data will be collected regarding participant
      characteristics, diagnosis, disease features, treatment and clinical outcome.
Two of the tests involve a technology called flow cytometry. Both of the flow cytometry tests
      are used to predict whether a person is likely to have a good response to chemotherapy or
      not.

      Three of the tests involve new genetic-based technology. One of these tests is called
      comparative genomic profiling. This test can detect genetic abnormalities that current
      testing methods are not able to detect. Another test involves micro-RNA profiling. The final
      test involves RNA sequencing. The researchers think these tests might be useful in predicting
      how well a person will respond to treatment.

      The novel laboratory tests being evaluated as part of this study are still in the early
      phases of development and cannot be used for clinical decision making. Participants enrolled
      in this study will not be informed regarding their individual results with respect to the
      study tests that are conducted using their biospecimens.

      The following information (data) will be collected regarding study participants: diagnosis,
      results of relevant clinical tests, age, gender, treatment and outcome during the 2 year
      study follow-up period. The study also involves the completion of study questionnaires at six
      different time points over the course of the two year study follow-up.
observational model: Cohort
time perspective: Prospective
measure: Prognostic capacity of the candidate tests (alone and in combination) to predict response to treatment, time to relapse, time to death.
time frame: Two years following the completion of enrollment.
description: For the AML cohort, the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, white blood cell count at presentation, antecedent hematologic disorder, FLT-3 status, Karnofsky Performance Status (KPS), cytogenetic sub-group (using WHO 2008). For the MDS cohort the candidate prognostic tests will be analyzed with adjustments for following clinical factors: age, KPS, karyotype, bone marrow blast count and number of cytopenias at diagnosis.
measure: Cost impact of candidate tests.
time frame: Two years following the completion of enrollment.
description: The cost-effectiveness of the candidate tests for AML and MDS treatment will be projected with the aid of economic simulation models.
measure: Societal risks and benefits related to the candidate tests.
time frame: Two years following the completion of enrollment.
description: A comparative analysis of policies and regulations in Canada governing prognostic tests will be undertaken (including tests that utilize RNA and DNA based technologies). In addition, input will be obtained from key stakeholders. This information will be used to develop a set of guidelines and best practices for this type of research.
arm group label: AML Cohort
description: This cohort is comprised of participants who have acute myelogenous leukemia (AML).
arm group label: MDS Cohort
description: This cohort is comprised of participants who have myelodysplastic syndrome (MDS).
Some of the laboratory tests that will be evaluated as part of this study will utilize RNA
      and/or DNA. There may be biospecimens left over after the work related to this study is
      complete. At the time of consent for the study, participants will be asked to provide consent
      (or decline their consent) regarding the use of leftover specimens (including RNA and DNA)
      for other research. The details regarding this other research are provided in the protocol
      and consent template.
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Stephen Couban, M.D.
role: Principal Investigator
affiliation: Nova Scotia Health Authority
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Canada
responsible party type: Sponsor
mesh term: Leukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
mesh term: Preleukemia
mesh term: Myelodysplastic Syndromes