Clinical trial NCT03801993: Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
Official title: Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
CF
GI
nutrition
quality of life
bowel
constipation
gastrointestinal
Lead sponsor: Seattle Children's Hospital
Sponsor class: OTHER
Collaborators: name: Emory University
name: University of North Carolina, Charlotte
name: University of Texas
name: Cystic Fibrosis Foundation
First posted: 2019-01-14
Enrollment: 402
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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).

Eligibility 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

Primary outcomes:
measure: Number of Participants Who Fully Completed at Least One ePRO
timeFrame: 1 month
Secondary outcomes:
measure: Number of Participants With Period-Prevalence of Constipation
timeFrame: 1 month
measure: Mean Patient Reported Outcome (PRO) Scores
timeFrame: At Visit 1 (1 day)
measure: Number of Participants Treated for GI Symptoms
timeFrame: At Visit 1 (1 day)
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: Hartford
country: United States
facility: Central Connecticut Cystic Fibrosis Center
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Pensacola
country: United States
facility: Nemours Children's Clinic - Pensacola
state: Florida
city: Saint Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta and Emory University
state: Georgia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Peoria
country: United States
facility: OSF Saint Francis Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Boston
country: United States
facility: Boston Children's Hospital, Brigham & Women's Hospital
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Memorial Health Care
state: Massachusetts
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Minneapolis
country: United States
facility: The Minnesota Cystic Fibrosis Center
state: Minnesota
city: Saint Louis
country: United States
facility: SSM Health Cardinal Glennon Children's Hospital
state: Missouri
city: New Brunswick
country: United States
facility: Rutgers - Robert Wood Johnson Medical School
state: New Jersey
city: Charlotte
country: United States
facility: Atrium Health Pulmonary Care
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University Baptist Medical Center
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: Oklahoma Cystic Fibrosis Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern / Children's Health
state: Texas
city: Salt Lake City
country: United States
facility: Intermountain Cystic Fibrosis Center
state: Utah
city: Burlington
country: United States
facility: The Children's Specialty Center Fletcher Allen Health Care
state: Vermont
Study officials:
affiliation: Emory University/Children's Healthcare
name: A. Jay Freeman, MD, MSc
role: PRINCIPAL_INVESTIGATOR
affiliation: University of North Carolina Charlotte, Atrium Health
name: Baharak Moshiree, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Texas Southwestern/Children's Health
name: Meghana Sathe, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Moshiree B, Freeman AJ, Vu PT, Khan U, Ufret-Vincenty C, Heltshe SL, Goss CH, Schwarzenberg SJ, Freedman SD, Borowitz D, Sathe M; GALAXY Study Group. Multicenter prospective study showing a high gastrointestinal symptom burden in cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):266-274. doi: 10.1016/j.jcf.2022.10.006. Epub 2022 Oct 29.