<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/NCT03749083</url>
  </required_header>
  <id_info>
    <org_study_id>18-304</org_study_id>
    <nct_id>NCT03749083</nct_id>
  </id_info>
  <brief_title>A Study of the Role of Circulating Tumor DNA in Predicting the Likelihood of Organ Preservation After Clinical Complete Response to Neoadjuvant Therapy for Rectal Cancer</brief_title>
  <official_title>A Study of the Role of Circulating Tumor DNA in Predicting the Likelihood of Organ Preservation After Clinical Complete Response to Neoadjuvant Therapy for Rectal Cancer</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Massachusetts General Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Clinical Genomics</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Massachusetts General Hospital</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      In this research study, the investigators are looking to see if the circulating tumor DNA&#xD;
      (genetic material), also known as ctDNA, in the blood will help them predict whether the&#xD;
      participant's cancer will come back.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      In this research study, the investigator are performing blood draws to see if the presence or&#xD;
      absence of circulating tumor DNA (ctDNA), the genetic material in the participant's cancer's&#xD;
      cells, will help the investigators predict whether the participant's cancer will come back or&#xD;
      not.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Funding&#xD;
  </why_stopped>
  <start_date type="Anticipated">December 31, 2019</start_date>
  <completion_date type="Anticipated">October 31, 2024</completion_date>
  <primary_completion_date type="Anticipated">October 31, 2021</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>One year local recurrence rate in participants that test positive for ctDNA compared to participants that tested negative for ctDNA</measure>
    <time_frame>1 year</time_frame>
    <description>Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Local recurrence rate at any time up to 5 years in patients who have achieved a clinical complete response after rectal cancer treatment but who test positive for circulating tumor DNA at study entry</measure>
    <time_frame>5 years</time_frame>
    <description>Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall rate of local recurrence by stratified KRAS/BRAF status and possibly other gene targets</measure>
    <time_frame>5 years</time_frame>
    <description>Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Median EORTC QLQ-CR29 Score</measure>
    <time_frame>Baseline, 6 months, 12 months, 24 months</time_frame>
    <description>European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) for Colorectal cancer(CR) 29 item questionnaire. The survey will be given at baseline, 6, 12, and 24 months. Results will be reported as the median score for each question. A score of 1 represents 'Not at All' and 4 represents 'Very Much'.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Median SF-12 Health Survey Score</measure>
    <time_frame>Baseline, 6 months, 12 months, 24 months</time_frame>
    <description>The SF-12 is a 12 item validated questionnaire that assesses a participants view of their health. Scores will be normalized on a 100 point scale with higher scores representing better health.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Median LARS Score</measure>
    <time_frame>Baseline, 6 months, 12 months, 24 months</time_frame>
    <description>The Low Anterior Resection Syndrome (LARS) Score is used to assess bowel dysfunction. The survey is assessed on a scale of 0-42 points, with a higher score indicating worse bowel dysfunction.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Median FIQOL Score</measure>
    <time_frame>Baseline, 6 months, 12 months, 24 months</time_frame>
    <description>Fecal Incontinence Quality of Life (FIQOL) Score. Lower scores represent lower functional status.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Median Fecal Incontinence Severity Index (FISI) score</measure>
    <time_frame>Baseline, 6 months, 12 months, 24 months</time_frame>
    <description>The FISI measures incontinence for gas, mucus, liquid stool,and solid stool. The survey assesses the number of times per day that the participant experiences incontinence for gas, mucus, liquid stool,and solid stool. Higher scores represent a higher degree of fecal incontinence.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">0</enrollment>
  <condition>Gastrointestinal Malignancies</condition>
  <arm_group>
    <arm_group_label>Tumor Sequencing</arm_group_label>
    <description>Quality of life assessments will be collected using The Functional Assessment of Cancer Therapy- Colorectal&#xD;
Blood for circulating tumor DNA will be collected</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>The Functional Assessment of Cancer Therapy- Colorectal</intervention_name>
    <description>The Functional Assessment of Cancer Therapy- Colorectal (FACT-C) questionnaire measures health-related quality of life for people with chronic illnesses.</description>
    <arm_group_label>Tumor Sequencing</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        This prospective, observational, non-interventional study will enroll patients who have&#xD;
        achieved a clinical complete response after treatment for rectal cancer and are undergoing&#xD;
        standard surveillance.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participants must have achieved a clinical complete response (cCR) within 3 months of&#xD;
             last therapy - defined as absence of residual ulceration, mass or mucosal irregularity&#xD;
             at endoscopic assessment - following neoadjuvant therapy with chemoradiation or&#xD;
             chemotherapy followed by radiation for stage I, II, or III non-metastatic rectal&#xD;
             cancer. (Whitening of the mucosa with presence of telangiectasia will be accepted as&#xD;
             cCR.)&#xD;
&#xD;
          -  Participants must have original tumor tissue (formalin-fixed, paraffin-embedded&#xD;
             specimens) available for analysis&#xD;
&#xD;
          -  Participants must be 18 years of age or older.&#xD;
&#xD;
          -  Participants must be able to understand and willing to sign a written informed consent&#xD;
             document.&#xD;
&#xD;
          -  Participants must have received long course chemoradiation to 40-54 Gy.&#xD;
&#xD;
          -  Participants must have received at least 4 cycles of FOLFOX&#xD;
&#xD;
          -  Participant must be no more than 3 months past the conclusion of initial&#xD;
             chemoradiation of rectal cancer (i.e., end of chemoradiation).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Participants may not have any other organ cancer evident at the time of enrollment.&#xD;
&#xD;
          -  Participants may not have any other concurrent serious illness that makes&#xD;
             participation on this study impractical or clinically inappropriate.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Theodore S Hong, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Massachusetts General Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Massachusetts General Hospital Cancer Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02214</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Beth Israel Deaconess Medical Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Brigham and Women Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Newton-Wellesley Hospital</name>
      <address>
        <city>Newton</city>
        <state>Massachusetts</state>
        <zip>02459</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2019</verification_date>
  <study_first_submitted>November 15, 2018</study_first_submitted>
  <study_first_submitted_qc>November 19, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 21, 2018</study_first_posted>
  <last_update_submitted>July 3, 2019</last_update_submitted>
  <last_update_submitted_qc>July 3, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">July 8, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Massachusetts General Hospital</investigator_affiliation>
    <investigator_full_name>Theodore Sunki Hong</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>Gastrointestinal Malignancies</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neoplasms</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03749083
org study id: 18-304
nct id: NCT03749083
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
In this research study, the investigators are looking to see if the circulating tumor DNA
      (genetic material), also known as ctDNA, in the blood will help them predict whether the
      participant's cancer will come back.
In this research study, the investigator are performing blood draws to see if the presence or
      absence of circulating tumor DNA (ctDNA), the genetic material in the participant's cancer's
      cells, will help the investigators predict whether the participant's cancer will come back or
      not.
observational model: Cohort
time perspective: Prospective
measure: One year local recurrence rate in participants that test positive for ctDNA compared to participants that tested negative for ctDNA
time frame: 1 year
description: Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.
measure: Local recurrence rate at any time up to 5 years in patients who have achieved a clinical complete response after rectal cancer treatment but who test positive for circulating tumor DNA at study entry
time frame: 5 years
description: Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.
measure: Overall rate of local recurrence by stratified KRAS/BRAF status and possibly other gene targets
time frame: 5 years
description: Local recurrence will be defined as recurrence of the pathologically confirmed adenocarcinoma. This may be detected by endoscopy for intraluminal recurrence or after radical surgery for radiographic evidence of extraluminal/mesorectal recurrence. The recurrence must be in the peri-anastomic site or rectal stump, or pre-sacral area. Regional nodal recurrence and lateral pelvic lymph node recurrence of rectal cancer are also included in this definition.
measure: Median EORTC QLQ-CR29 Score
time frame: Baseline, 6 months, 12 months, 24 months
description: European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) for Colorectal cancer(CR) 29 item questionnaire. The survey will be given at baseline, 6, 12, and 24 months. Results will be reported as the median score for each question. A score of 1 represents 'Not at All' and 4 represents 'Very Much'.
measure: Median SF-12 Health Survey Score
time frame: Baseline, 6 months, 12 months, 24 months
description: The SF-12 is a 12 item validated questionnaire that assesses a participants view of their health. Scores will be normalized on a 100 point scale with higher scores representing better health.
measure: Median LARS Score
time frame: Baseline, 6 months, 12 months, 24 months
description: The Low Anterior Resection Syndrome (LARS) Score is used to assess bowel dysfunction. The survey is assessed on a scale of 0-42 points, with a higher score indicating worse bowel dysfunction.
measure: Median FIQOL Score
time frame: Baseline, 6 months, 12 months, 24 months
description: Fecal Incontinence Quality of Life (FIQOL) Score. Lower scores represent lower functional status.
measure: Median Fecal Incontinence Severity Index (FISI) score
time frame: Baseline, 6 months, 12 months, 24 months
description: The FISI measures incontinence for gas, mucus, liquid stool,and solid stool. The survey assesses the number of times per day that the participant experiences incontinence for gas, mucus, liquid stool,and solid stool. Higher scores represent a higher degree of fecal incontinence.
arm group label: Tumor Sequencing
description: Quality of life assessments will be collected using The Functional Assessment of Cancer Therapy- Colorectal
Blood for circulating tumor DNA will be collected
intervention type: Other
intervention name: The Functional Assessment of Cancer Therapy- Colorectal
description: The Functional Assessment of Cancer Therapy- Colorectal (FACT-C) questionnaire measures health-related quality of life for people with chronic illnesses.
arm group label: Tumor Sequencing
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
last name: Theodore S Hong, MD
role: Principal Investigator
affiliation: Massachusetts General Hospital
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Massachusetts General Hospital
investigator full name: Theodore Sunki Hong
investigator title: Principal Investigator
mesh term: Neoplasms
sharing ipd: No