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

Completed

Conditions: Cystic Fibrosis

Registration Number: NCT03801993

Lead Sponsor: Chris Goss

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

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).

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 402

Inclusion Criteria

All genders ≥ 2 years of age at time of consent
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)
Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled)
Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
Willing to complete questionnaires on mobile device
Able to use the Medidata Patient Cloud mobile application for completing the questionnaires

Exclusion Criteria

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
Presence of a pulmonary exacerbation at the Enrollment Visit
Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit
Current gastrointestinal (GI) or abdominal/pelvic malignancy
Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit
At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel cleanout in the 28 days after the Enrollment Visit
Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment Visit
Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants Who Fully Completed at Least One ePRO	1 month	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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Period-Prevalence of Constipation	1 month	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.
Mean Patient Reported Outcome (PRO) Scores	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.
Number of Participants Treated for GI Symptoms	At Visit 1 (1 day)	Number of enrolled participants receiving treatment for GI symptoms at Visit 1

Trial Locations

Locations (26): Stanford University Medical Center
🇺🇸
Palo Alto, California, United States
Central Connecticut Cystic Fibrosis Center
🇺🇸
Hartford, Connecticut, United States
Nemours Children's Clinic - Pensacola
🇺🇸
Pensacola, Florida, United States
All Children's Hospital
🇺🇸
Saint Petersburg, Florida, United States
Children's Healthcare of Atlanta and Emory University
🇺🇸
Atlanta, Georgia, United States
OSF Saint Francis Medical Center
🇺🇸
Peoria, Illinois, United States
Northwestern University
🇺🇸
Chicago, Illinois, United States
Riley Hospital for Children
🇺🇸
Indianapolis, Indiana, United States
Maine Medical Center
🇺🇸
Portland, Maine, United States
Helen DeVos Children's Hospital
🇺🇸
Grand Rapids, Michigan, United States
Boston Children's Hospital, Brigham & Women's Hospital
🇺🇸
Boston, Massachusetts, United States
University of Massachusetts Memorial Health Care
🇺🇸
Worcester, Massachusetts, United States
The Minnesota Cystic Fibrosis Center
🇺🇸
Minneapolis, Minnesota, United States
SSM Health Cardinal Glennon Children's Hospital
🇺🇸
Saint Louis, Missouri, United States
Atrium Health Pulmonary Care
🇺🇸
Charlotte, North Carolina, United States
Wake Forest University Baptist Medical Center
🇺🇸
Winston-Salem, North Carolina, United States
Oregon Health Sciences University
🇺🇸
Portland, Oregon, United States
Nationwide Children's Hospital
🇺🇸
Columbus, Ohio, United States
Oklahoma Cystic Fibrosis Center
🇺🇸
Oklahoma City, Oklahoma, United States
University of Texas Southwestern / Children's Health
🇺🇸
Dallas, Texas, United States
Children's Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Intermountain Cystic Fibrosis Center
🇺🇸
Salt Lake City, Utah, United States
The Children's Specialty Center Fletcher Allen Health Care
🇺🇸
Burlington, Vermont, United States
University of Kentucky
🇺🇸
Lexington, Kentucky, United States
Rutgers - Robert Wood Johnson Medical School
🇺🇸
New Brunswick, New Jersey, United States
Children's National Medical Center
🇺🇸
Washington, District of Columbia, United States