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Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY)

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ClinicalTrials.gov Identifier: NCT03801993
Recruitment Status : Not yet recruiting
First Posted : January 14, 2019
Last Update Posted : January 14, 2019
Sponsor:
Collaborators:
Emory University
University of North Carolina, Charlotte
University of Texas
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Chris Goss, Seattle Children's Hospital

Brief Summary:
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).

Condition or disease
Cystic Fibrosis

Detailed Description:

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).


Study Type : Observational
Estimated Enrollment : 400 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)
Estimated Study Start Date : May 1, 2019
Estimated Primary Completion Date : June 1, 2020
Estimated Study Completion Date : June 1, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Group/Cohort
All Subjects
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.



Primary Outcome Measures :
  1. Percentage of completed assessments [ Time Frame: 1 month ]
    Percentage of scheduled outside-the-clinic assessments with at least one of the four PROs fully completed.


Secondary Outcome Measures :
  1. Prevalence of Constipation [ Time Frame: 1 month ]
    Percentage of enrolled subjects with any occurrence of protocol-defined constipation during 1 month of follow-up (the period prevalence)

  2. Mean Patient Reported Outcome (PRO) Scores [ Time Frame: At Visit 1 (1 day) ]
    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.

  3. Percentage of subjects treated for GI symptoms [ Time Frame: At Visit 1 (1 day) ]
    Percentage of enrolled subjects receiving treatment for GI symptoms at Visit 1



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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.
Criteria

Inclusion Criteria:

  1. All genders ≥ 2 years of age at time of consent
  2. Documentation of a Cystic Fibrosis (CF) diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:

    • Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
    • Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
    • Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproterenol of less than -5 mV)
  3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled)
  4. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
  5. Willing to complete questionnaires on mobile device
  6. Able to use the Medidata Patient Cloud mobile application for completing the questionnaires

Exclusion Criteria:

  1. Presence of a condition or abnormality that, in the opinion of the Investigator, would complicate interpretation of study outcome data or interfere with achieving the study objectives
  2. Presence of a pulmonary exacerbation at the Enrollment Visit
  3. Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit
  4. Current gastrointestinal (GI) or abdominal/pelvic malignancy
  5. Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit
  6. At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel cleanout in the 28 days after the Enrollment Visit
  7. Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment Visit
  8. Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03801993


Contacts
Contact: Carmen Ufret-Vincenty, PhD 206-884-1443 carmen.ufret-vincenty@seattlechildrens.org

  Show 26 Study Locations
Sponsors and Collaborators
Chris Goss
Emory University
University of North Carolina, Charlotte
University of Texas
Cystic Fibrosis Foundation
Investigators
Principal Investigator: A. Jay Freeman, MD, MSc Emory University/Children's Healthcare
Principal Investigator: Baharak Moshiree, MD University of North Carolina Charlotte, Atrium Health
Principal Investigator: Meghana Sathe, MD University of Texas Southwestern/Children's Health

Responsible Party: Chris Goss, Professor of Medicine and Pediatrics, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT03801993     History of Changes
Other Study ID Numbers: GALAXY-OB-18
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: January 14, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Chris Goss, Seattle Children's Hospital:
Cystic Fibrosis
CF
GI
nutrition
quality of life
bowel
constipation
gastrointestinal

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases