<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/NCT04431479</url>
  </required_header>
  <id_info>
    <org_study_id>RG1006823</org_study_id>
    <secondary_id>NCI-2020-01242</secondary_id>
    <secondary_id>10360</secondary_id>
    <secondary_id>R01CA118953</secondary_id>
    <nct_id>NCT04431479</nct_id>
  </id_info>
  <brief_title>Predicting the Quality of Response to Specific Treatments in Patients With cGVHD, PQRST Study</brief_title>
  <official_title>Predicting the Quality of Response to Specific Treatments (PQRST)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Fred Hutchinson Cancer Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>Fred Hutchinson Cancer Center</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This trial collects clinical data and blood samples to predict the quality of response to&#xD;
      specific treatments in patients with chronic graft-versus-host disease (cGVHD) who are about&#xD;
      to start initial or second-line therapy. Collecting and analyzing clinical data and blood&#xD;
      samples from patients with cGVHD before and after treatment initiation may help doctors&#xD;
      identify changes that may predict treatment response.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      OUTLINE:&#xD;
&#xD;
      Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and&#xD;
      emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months&#xD;
      after starting index treatment, and at start of a new systemic treatment. Patients also&#xD;
      undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after&#xD;
      starting index treatment, or at a treatment change visit if new therapy has not started.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">June 29, 2020</start_date>
  <completion_date type="Anticipated">August 2, 2025</completion_date>
  <primary_completion_date type="Anticipated">August 2, 2025</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>Clinical Response according to the 2014 criteria</measure>
    <time_frame>Up to 6 months</time_frame>
    <description>At the assessments, the 9 provider-reported National Institute of Health (NIH) organ severity scores (skin, eye, mouth, esophagus, upper gastrointestinal [GI], lower GI, liver, lung, and joint manifestations) will be collected reflecting disease activity in the past week. All scored items are single 4-7 point Likert scales. Based on past work, we anticipate it will take approximately 8 minutes to conduct the physical exam and record the relevant results. Pulmonary function testing results will be collected if available otherwise portable spirometry will be performed. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Time to next systemic treatment</measure>
    <time_frame>From the start of the index medication until the addition of another systemic chronic graft versus host disease (cGVHD) treatment with death and treated recurrent malignancy considered competing events, assessed up to 3 years</time_frame>
    <description>Any addition of another systemic cGVHD treatment for medical reasons will be considered a failure, whether added because of a new or worsening manifestation of cGVHD, used as a &quot;steroid sparing agent,&quot; or substituted due to toxicity.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Duration of treatment</measure>
    <time_frame>Up to 3 months</time_frame>
    <description>Duration of treatment is defined as the time until discontinuation of therapeutic systemic immunosuppression (adrenal replacement and topical/local therapies are allowed) without resumption for at least 3 months.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Survival</measure>
    <time_frame>From the start of the index medication to death with patients lost to follow up or alive at the conclusion of the study censored, assessed up to 3 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Non-relapse mortality</measure>
    <time_frame>Up to 3 years</time_frame>
    <description>Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Patient-reported outcomes</measure>
    <time_frame>Up to 3 years</time_frame>
    <description>Will be assessed using Lee symptom scale and Patient Reported Outcomes Measurement Information System (PROMIS). The summary score of the Lee Symptom Scale and the PROMIS Global will be calculated according to the instructions of the developers. For analyses assessing change in quality of life, improvement or worsening of the Summary symptom score by 6 points or more or the PROMIS Physical or Mental Functioning scales by 5 points or more compared to baseline will be considered a clinically significant change.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">200</enrollment>
  <condition>Chronic Graft Versus Host Disease</condition>
  <arm_group>
    <arm_group_label>Observational (questionnaire, biospecimen, chart review)</arm_group_label>
    <description>Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started.</description>
  </arm_group>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Biospecimen Collection</intervention_name>
    <description>Undergo collection of blood sample</description>
    <arm_group_label>Observational (questionnaire, biospecimen, chart review)</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Medical Chart Review</intervention_name>
    <description>Review of medical chart</description>
    <arm_group_label>Observational (questionnaire, biospecimen, chart review)</arm_group_label>
    <other_name>Chart Review</other_name>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Quality-of-Life Assessment</intervention_name>
    <description>Ancillary studies</description>
    <arm_group_label>Observational (questionnaire, biospecimen, chart review)</arm_group_label>
    <other_name>Quality of Life Assessment</other_name>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Questionnaire Administration</intervention_name>
    <description>Complete questionnaire</description>
    <arm_group_label>Observational (questionnaire, biospecimen, chart review)</arm_group_label>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with chronic graft-versus-host disease (GVHD) who are starting first- or&#xD;
        second-line therapy for GVHD&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Prior allogeneic stem cell transplant, with any graft source, donor type, and GVHD&#xD;
             prophylaxis&#xD;
&#xD;
          -  No evidence of persistent or progressive malignancy at the time of enrollment&#xD;
&#xD;
          -  Agrees to be evaluated at the transplant center before initial or second-line&#xD;
             treatment is started (may be concurrent with the enrollment visit), and later between&#xD;
             2-6 weeks, 3 months and 6 months after treatment is started or if a new therapy is&#xD;
             started before 6 months&#xD;
&#xD;
          -  Signed, informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Inability to comply with study procedures&#xD;
&#xD;
          -  Uncontrolled psychiatric disorder&#xD;
&#xD;
          -  Anticipated survival &lt; 6 months&#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>Stephanie J Lee</last_name>
    <role>Principal Investigator</role>
    <affiliation>Fred Hutch/University of Washington Cancer Consortium</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Stephanie J Lee</last_name>
    <phone>206-667-6190</phone>
    <email>sjlee@fredhutch.org</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Monji Bat-Erdene</last_name>
    <phone>206-667-3615</phone>
    <email>mbaterde@fredhutch.org</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Hannah Lange</last_name>
      <phone>352-273-7832</phone>
      <email>hannah.lange@medicine.ufl.edu</email>
    </contact>
    <investigator>
      <last_name>Nosha Farhadfar</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Moffitt Cancer Center</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33612</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Joseph Pidala</last_name>
      <phone>888-663-3488</phone>
      <email>Joseph.Pidala@moffitt.org</email>
    </contact>
    <investigator>
      <last_name>Joseph Pidala</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Dana-Farber Harvard Cancer Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Corey S. Cutler</last_name>
      <phone>617-632-5946</phone>
      <email>corey_cutler@dfci.harvard.edu</email>
    </contact>
    <investigator>
      <last_name>Corey S. Cutler</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Minnesota/Masonic Cancer Center</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Najla El-Jurdi</last_name>
      <email>neljurdi@umn.edu</email>
    </contact>
    <investigator>
      <last_name>Najla El-Jurdi</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Roswell Park Cancer Institute</name>
      <address>
        <city>Buffalo</city>
        <state>New York</state>
        <zip>14263</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Active, not recruiting</status>
  </location>
  <location>
    <facility>
      <name>Cleveland Clinic Foundation</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Betty K. Hamilton</last_name>
      <phone>216-444-6833</phone>
      <email>HAMILTB2@ccf.org</email>
    </contact>
    <investigator>
      <last_name>Betty K. Hamilton</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>UPMC Hillman Cancer Center</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15232</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Barb Stadterman</last_name>
      <phone>412-647-5554</phone>
      <email>stadtermanbm@upmc.edu</email>
    </contact>
    <investigator>
      <last_name>Annie Im</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Vanderbilt University/Ingram Cancer Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Completed</status>
  </location>
  <location>
    <facility>
      <name>Fred Hutch/University of Washington Cancer Consortium</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98109</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Stephanie J. Lee</last_name>
      <phone>206-667-6190</phone>
      <email>sjlee@fredhutch.org</email>
    </contact>
    <investigator>
      <last_name>Stephanie J. Lee</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Vancouver General Hospital/BC Cancer</name>
      <address>
        <city>Vancouver</city>
        <state>British Columbia</state>
        <zip>V5Z 4E6</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jennifer White</last_name>
      <phone>604-875-4863</phone>
      <email>Jennifer.White1@bccancer.bc.ca</email>
    </contact>
    <investigator>
      <last_name>Jennifer White</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>June 11, 2020</study_first_submitted>
  <study_first_submitted_qc>June 11, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 16, 2020</study_first_posted>
  <last_update_submitted>September 6, 2023</last_update_submitted>
  <last_update_submitted_qc>September 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 7, 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>Bronchiolitis Obliterans Syndrome</mesh_term>
    <mesh_term>Graft vs Host Disease</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/NCT04431479
org study id: RG1006823
secondary id: NCI-2020-01242
secondary id: 10360
secondary id: R01CA118953
nct id: NCT04431479
lead sponsor: 
collaborator: 
is fda regulated drug: No
is fda regulated device: No
This trial collects clinical data and blood samples to predict the quality of response to
      specific treatments in patients with chronic graft-versus-host disease (cGVHD) who are about
      to start initial or second-line therapy. Collecting and analyzing clinical data and blood
      samples from patients with cGVHD before and after treatment initiation may help doctors
      identify changes that may predict treatment response.
OUTLINE:

      Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and
      emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months
      after starting index treatment, and at start of a new systemic treatment. Patients also
      undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after
      starting index treatment, or at a treatment change visit if new therapy has not started.
observational model: Cohort
time perspective: Prospective
measure: Clinical Response according to the 2014 criteria
time frame: Up to 6 months
description: At the assessments, the 9 provider-reported National Institute of Health (NIH) organ severity scores (skin, eye, mouth, esophagus, upper gastrointestinal [GI], lower GI, liver, lung, and joint manifestations) will be collected reflecting disease activity in the past week. All scored items are single 4-7 point Likert scales. Based on past work, we anticipate it will take approximately 8 minutes to conduct the physical exam and record the relevant results. Pulmonary function testing results will be collected if available otherwise portable spirometry will be performed. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.
measure: Time to next systemic treatment
time frame: From the start of the index medication until the addition of another systemic chronic graft versus host disease (cGVHD) treatment with death and treated recurrent malignancy considered competing events, assessed up to 3 years
description: Any addition of another systemic cGVHD treatment for medical reasons will be considered a failure, whether added because of a new or worsening manifestation of cGVHD, used as a "steroid sparing agent," or substituted due to toxicity.
measure: Duration of treatment
time frame: Up to 3 months
description: Duration of treatment is defined as the time until discontinuation of therapeutic systemic immunosuppression (adrenal replacement and topical/local therapies are allowed) without resumption for at least 3 months.
measure: Survival
time frame: From the start of the index medication to death with patients lost to follow up or alive at the conclusion of the study censored, assessed up to 3 years
measure: Non-relapse mortality
time frame: Up to 3 years
description: Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.
measure: Patient-reported outcomes
time frame: Up to 3 years
description: Will be assessed using Lee symptom scale and Patient Reported Outcomes Measurement Information System (PROMIS). The summary score of the Lee Symptom Scale and the PROMIS Global will be calculated according to the instructions of the developers. For analyses assessing change in quality of life, improvement or worsening of the Summary symptom score by 6 points or more or the PROMIS Physical or Mental Functioning scales by 5 points or more compared to baseline will be considered a clinically significant change.
arm group label: Observational (questionnaire, biospecimen, chart review)
description: Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started.
intervention type: Procedure
intervention name: Biospecimen Collection
description: Undergo collection of blood sample
arm group label: Observational (questionnaire, biospecimen, chart review)
intervention type: Other
intervention name: Medical Chart Review
description: Review of medical chart
arm group label: Observational (questionnaire, biospecimen, chart review)
other name: Chart Review
intervention type: Other
intervention name: Quality-of-Life Assessment
description: Ancillary studies
arm group label: Observational (questionnaire, biospecimen, chart review)
other name: Quality of Life Assessment
intervention type: Other
intervention name: Questionnaire Administration
description: Complete questionnaire
arm group label: Observational (questionnaire, biospecimen, chart review)
Blood
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Stephanie J Lee
role: Principal Investigator
affiliation: Fred Hutch/University of Washington Cancer Consortium
last name: Stephanie J Lee
phone: 206-667-6190
email: sjlee@fredhutch.org
last name: Monji Bat-Erdene
phone: 206-667-3615
email: mbaterde@fredhutch.org
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Active, not recruiting
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Completed
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
country: Canada
country: United States
responsible party type: Sponsor
mesh term: Bronchiolitis Obliterans Syndrome
mesh term: Graft vs Host Disease
sharing ipd: No