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

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