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).
Study Type
OBSERVATIONAL
Enrollment
402
Number of Participants Who Fully Completed at Least One ePRO
Number participants with scheduled outside-the-clinic assessments who fully completed at least one of the four PROs. A "fully complete" questionnaire is defined as having responded to all questions with no missing responses.
Time frame: 1 month
Number of Participants With Period-Prevalence of Constipation
This table summarizes the period-prevalence of protocol-defined constipation. For each follow-up response, constipation was defined by the protocol as having fewer than 3 bowel movements and/or Bristol Stool Scale of Type 1 or 2 (hard lumps) in the past 7 days. Period prevalence is defined as occurrence of the symptom at any time from baseline to week 4. Only participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.
Time frame: 1 month
Mean Patient Reported Outcome (PRO) Scores
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.
Time frame: At Visit 1 (1 day)
Number of Participants Treated for GI Symptoms
Number of enrolled participants receiving treatment for GI symptoms at Visit 1
Time frame: At Visit 1 (1 day)
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Stanford University Medical Center
Palo Alto, California, United States
Central Connecticut Cystic Fibrosis Center
Hartford, Connecticut, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nemours Children's Clinic - Pensacola
Pensacola, Florida, United States
All Children's Hospital
St. Petersburg, Florida, United States
Children's Healthcare of Atlanta and Emory University
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
OSF Saint Francis Medical Center
Peoria, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Kentucky
Lexington, Kentucky, United States
...and 16 more locations