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

NCT ID: NCT03801993

Last Updated: 2024-10-15

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 402 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-05-01
• Study Completion Date: 2019-09-17

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).

Conditions

Cystic Fibrosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

All Subjects

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.

No interventions assigned to this group

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

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Emory University

OTHER

Sponsor Role: collaborator

University of North Carolina, Charlotte

OTHER

Sponsor Role: collaborator

University of Texas

OTHER

Sponsor Role: collaborator

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

Chris Goss

OTHER

Sponsor Role: lead

Responsible Party

Chris Goss

Professor of Medicine and Pediatrics

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

A. Jay Freeman, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Emory University/Children's Healthcare

Baharak Moshiree, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina Charlotte, Atrium Health

Meghana Sathe, MD

Role: PRINCIPAL_INVESTIGATOR

University of Texas Southwestern/Children's Health

Locations

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

Maine Medical Center

Portland, Maine, United States

Boston Children's Hospital, Brigham & Women's Hospital

Boston, Massachusetts, United States

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, United States

SSM Health Cardinal Glennon Children's Hospital

St Louis, Missouri, United States

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Atrium Health Pulmonary Care

Charlotte, North Carolina, United States

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Oklahoma Cystic Fibrosis Center

Oklahoma City, Oklahoma, United States

Oregon Health Sciences University

Portland, Oregon, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Texas Southwestern / Children's Health

Dallas, Texas, 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

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 36319569 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

GALAXY-OB-18

Identifier Type: -

Identifier Source: org_study_id