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



Status:Not yet recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:2 - Any
Updated:1/16/2019
Start Date:May 1, 2019
End Date:June 1, 2020
Contact:Carmen Ufret-Vincenty, PhD
Email:carmen.ufret-vincenty@seattlechildrens.org
Phone:206-884-1443

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Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)

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).

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
We found this trial at
26
sites
1 Medical Center Blvd
Winston-Salem, North Carolina 27103
(336) 716-2011
Wake Forest University Baptist Medical Center Welcome to Wake Forest Baptist Medical Center, a fully...
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201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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Atlanta, GA
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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5153 North 9th Avenue
Pensacola, Florida 32504
(850) 505-4700
Nemours Children's Clinic - Pensacola Nemours Children’s Clinic, Pensacola serves children and families in northwest...
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Pensacola, FL
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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22 Bramhall St
Portland, Maine 04102
(207) 662-0111
Maine Medical Center One of the country's consistently highest rated hospitals is right in your...
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Boston, MA
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Burlington, VT
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Charlotte, North Carolina 28277
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303 East Superior Street
Chicago, Illinois 60611
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Dallas, TX
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Grand Rapids, Michigan 49503
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Hartford, Connecticut 06106
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705 Riley Hospital Dr
Indianapolis, Indiana 46202
(317) 944-5000
Riley Hospital for Children Riley Hospital for Children at IU Health is a place of...
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Lexington, Kentucky
859) 257-9000
University of Kentucky The University of Kentucky is a public, land grant university dedicated to...
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516 Delaware Street Southeast
Minneapolis, Minnesota 55455
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New Brunswick, New Jersey 08903
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Oklahoma City, Oklahoma 73104
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Palo Alto, California 94304
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Peoria, Illinois 61637
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Portland, Oregon 97227
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1465 South Grand Boulevard
Saint Louis, Missouri 63104
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Saint Petersburg, Florida 33701
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26 1900 East
Salt Lake City, Utah 84113
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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Worcester, Massachusetts 01605
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