<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/NCT03801993</url>
  </required_header>
  <id_info>
    <org_study_id>GALAXY-OB-18</org_study_id>
    <nct_id>NCT03801993</nct_id>
  </id_info>
  <brief_title>Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis</brief_title>
  <acronym>GALAXY</acronym>
  <official_title>Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Chris Goss</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Emory University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>University of North Carolina, Charlotte</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>University of Texas</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Cystic Fibrosis Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Seattle Children's 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>
      This is a prospective, multicenter, observational study designed to collect gastrointestinal&#xD;
      related data in patients with Cystic Fibrosis (CF).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      There are currently no large, multicenter prospective clinical trials examining management of&#xD;
      constipation or other gastrointestinal (GI) symptoms in people with cystic fibrosis (CF).&#xD;
      Current recommendations in the CF literature are largely based on expert consensus and&#xD;
      opinions. Yet, constipation and other GI symptoms are crucial factors in quality of life&#xD;
      (QOL) and maintenance of optimal nutritional state in people with CF. This study will use&#xD;
      GI-symptomatology questionnaires to understand the multiple overlapping GI symptoms in people&#xD;
      with CF.&#xD;
&#xD;
      Eligible subjects will be consented and enrolled in the study at the Enrollment Visit. At the&#xD;
      visit, the subject or parent/guardian will complete the patient reported outcome surveys&#xD;
      (PROs) using a mobile device (e.g., smartphone or tablet). The same questionnaires will be&#xD;
      completed on a mobile device outside the clinic three additional times. The PROs will consist&#xD;
      of four questionnaires: Patient Assessment of Constipation Symptoms (PAC-SYM), Patient&#xD;
      Assessment of Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation&#xD;
      Quality of Life (PAC-QOL) and a disease-specific questionnaire (Bristol Stool Scale and&#xD;
      questions about fecal incontinence, and stool quality and frequency).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">May 1, 2019</start_date>
  <completion_date type="Actual">September 17, 2019</completion_date>
  <primary_completion_date type="Actual">September 17, 2019</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>Percentage of completed assessments</measure>
    <time_frame>1 month</time_frame>
    <description>Percentage of scheduled outside-the-clinic assessments with at least one of the four PROs fully completed.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Prevalence of Constipation</measure>
    <time_frame>1 month</time_frame>
    <description>Percentage of enrolled subjects with any occurrence of protocol-defined constipation during 1 month of follow-up (the period prevalence)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean Patient Reported Outcome (PRO) Scores</measure>
    <time_frame>At Visit 1 (1 day)</time_frame>
    <description>Mean PRO scores (PAC-SYM score range: 0-4, PAGI-SYM score range: 0-5, PAC-QOL score range: 0-4) at time of enrollment (Visit 1) where lower scores correspond to less symptom severity.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of subjects treated for GI symptoms</measure>
    <time_frame>At Visit 1 (1 day)</time_frame>
    <description>Percentage of enrolled subjects receiving treatment for GI symptoms at Visit 1</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">402</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>All Subjects</arm_group_label>
    <description>Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of&#xD;
        the exclusion criteria will be eligible for participation in this study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. All genders ≥ 2 years of age at time of consent&#xD;
&#xD;
          2. Documentation of a Cystic Fibrosis (CF) diagnosis as evidenced by one or more clinical&#xD;
             features consistent with the CF phenotype and one or more of the following criteria:&#xD;
&#xD;
               -  Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by&#xD;
                  quantitative pilocarpine iontophoresis test (QPIT)&#xD;
&#xD;
               -  Two well-characterized mutations in the cystic fibrosis transmembrane conductance&#xD;
                  regulator (CFTR) gene&#xD;
&#xD;
               -  Abnormal nasal potential difference (NPD) (change in NPD in response to a low&#xD;
                  chloride solution and isoproterenol of less than -5 mV)&#xD;
&#xD;
          3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in&#xD;
             the Registry at Enrollment Visit if not previously enrolled)&#xD;
&#xD;
          4. Written informed consent (and assent when applicable) obtained from subject or&#xD;
             subject's legal representative&#xD;
&#xD;
          5. Willing to complete questionnaires on mobile device&#xD;
&#xD;
          6. Able to use the Medidata Patient Cloud mobile application for completing the&#xD;
             questionnaires&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Presence of a condition or abnormality that, in the opinion of the Investigator, would&#xD;
             complicate interpretation of study outcome data or interfere with achieving the study&#xD;
             objectives&#xD;
&#xD;
          2. Presence of a pulmonary exacerbation at the Enrollment Visit&#xD;
&#xD;
          3. Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days&#xD;
             prior to the Enrollment Visit&#xD;
&#xD;
          4. Current gastrointestinal (GI) or abdominal/pelvic malignancy&#xD;
&#xD;
          5. Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit&#xD;
&#xD;
          6. At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel&#xD;
             cleanout in the 28 days after the Enrollment Visit&#xD;
&#xD;
          7. Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment&#xD;
             Visit&#xD;
&#xD;
          8. Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>A. Jay Freeman, MD, MSc</last_name>
    <role>Principal Investigator</role>
    <affiliation>Emory University/Children's Healthcare</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Baharak Moshiree, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of North Carolina Charlotte, Atrium Health</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Meghana Sathe, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Texas Southwestern/Children's Health</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Stanford University Medical Center</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Central Connecticut Cystic Fibrosis Center</name>
      <address>
        <city>Hartford</city>
        <state>Connecticut</state>
        <zip>06106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Medical Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20816</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Clinic - Pensacola</name>
      <address>
        <city>Pensacola</city>
        <state>Florida</state>
        <zip>32514</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>All Children's Hospital</name>
      <address>
        <city>Saint Petersburg</city>
        <state>Florida</state>
        <zip>33701</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Healthcare of Atlanta and Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Northwestern University</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>OSF Saint Francis Medical Center</name>
      <address>
        <city>Peoria</city>
        <state>Illinois</state>
        <zip>61637</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riley Hospital for Children</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kentucky</name>
      <address>
        <city>Lexington</city>
        <state>Kentucky</state>
        <zip>40506-9983</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Maine Medical Center</name>
      <address>
        <city>Portland</city>
        <state>Maine</state>
        <zip>04102</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital, Brigham &amp; Women's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Massachusetts Memorial Health Care</name>
      <address>
        <city>Worcester</city>
        <state>Massachusetts</state>
        <zip>01655</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Helen DeVos Children's Hospital</name>
      <address>
        <city>Grand Rapids</city>
        <state>Michigan</state>
        <zip>49503</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Minnesota Cystic Fibrosis Center</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>SSM Health Cardinal Glennon Children's Hospital</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rutgers - Robert Wood Johnson Medical School</name>
      <address>
        <city>New Brunswick</city>
        <state>New Jersey</state>
        <zip>08903</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Atrium Health Pulmonary Care</name>
      <address>
        <city>Charlotte</city>
        <state>North Carolina</state>
        <zip>28203</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wake Forest University Baptist Medical Center</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27103</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oklahoma Cystic Fibrosis Center</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health Sciences University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Texas Southwestern / Children's Health</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75247</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Intermountain Cystic Fibrosis Center</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84113</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Specialty Center Fletcher Allen Health Care</name>
      <address>
        <city>Burlington</city>
        <state>Vermont</state>
        <zip>05401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2019</verification_date>
  <study_first_submitted>January 4, 2019</study_first_submitted>
  <study_first_submitted_qc>January 9, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 14, 2019</study_first_posted>
  <last_update_submitted>October 1, 2019</last_update_submitted>
  <last_update_submitted_qc>October 1, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">October 2, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Seattle Children's Hospital</investigator_affiliation>
    <investigator_full_name>Chris Goss</investigator_full_name>
    <investigator_title>Professor of Medicine and Pediatrics</investigator_title>
  </responsible_party>
  <keyword>Cystic Fibrosis</keyword>
  <keyword>CF</keyword>
  <keyword>GI</keyword>
  <keyword>nutrition</keyword>
  <keyword>quality of life</keyword>
  <keyword>bowel</keyword>
  <keyword>constipation</keyword>
  <keyword>gastrointestinal</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</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/NCT03801993
org study id: GALAXY-OB-18
nct id: NCT03801993
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a prospective, multicenter, observational study designed to collect gastrointestinal
      related data in patients with Cystic Fibrosis (CF).
There are currently no large, multicenter prospective clinical trials examining management of
      constipation or other gastrointestinal (GI) symptoms in people with cystic fibrosis (CF).
      Current recommendations in the CF literature are largely based on expert consensus and
      opinions. Yet, constipation and other GI symptoms are crucial factors in quality of life
      (QOL) and maintenance of optimal nutritional state in people with CF. This study will use
      GI-symptomatology questionnaires to understand the multiple overlapping GI symptoms in people
      with CF.

      Eligible subjects will be consented and enrolled in the study at the Enrollment Visit. At the
      visit, the subject or parent/guardian will complete the patient reported outcome surveys
      (PROs) using a mobile device (e.g., smartphone or tablet). The same questionnaires will be
      completed on a mobile device outside the clinic three additional times. The PROs will consist
      of four questionnaires: Patient Assessment of Constipation Symptoms (PAC-SYM), Patient
      Assessment of Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation
      Quality of Life (PAC-QOL) and a disease-specific questionnaire (Bristol Stool Scale and
      questions about fecal incontinence, and stool quality and frequency).
observational model: Cohort
time perspective: Prospective
measure: Percentage of completed assessments
time frame: 1 month
description: Percentage of scheduled outside-the-clinic assessments with at least one of the four PROs fully completed.
measure: Prevalence of Constipation
time frame: 1 month
description: Percentage of enrolled subjects with any occurrence of protocol-defined constipation during 1 month of follow-up (the period prevalence)
measure: Mean Patient Reported Outcome (PRO) Scores
time frame: At Visit 1 (1 day)
description: Mean PRO scores (PAC-SYM score range: 0-4, PAGI-SYM score range: 0-5, PAC-QOL score range: 0-4) at time of enrollment (Visit 1) where lower scores correspond to less symptom severity.
measure: Percentage of subjects treated for GI symptoms
time frame: At Visit 1 (1 day)
description: Percentage of enrolled subjects receiving treatment for GI symptoms at Visit 1
arm group label: All Subjects
description: Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: N/A
healthy volunteers: No
last name: A. Jay Freeman, MD, MSc
role: Principal Investigator
affiliation: Emory University/Children's Healthcare
last name: Baharak Moshiree, MD
role: Principal Investigator
affiliation: University of North Carolina Charlotte, Atrium Health
last name: Meghana Sathe, MD
role: Principal Investigator
affiliation: University of Texas Southwestern/Children's Health
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country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Seattle Children's Hospital
investigator full name: Chris Goss
investigator title: Professor of Medicine and Pediatrics
mesh term: Cystic Fibrosis
mesh term: Fibrosis
sharing ipd: No