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

NCT03801993 | Completed Results

• 1 other identifier: GALAXY-OB-18
• Study Type: observational
• Target: 402
• Locations: 1 country
• Sites: 26

Brief Summary

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

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; CF; GI; nutrition; quality of life; bowel; constipation; gastrointestinal

Outcome Measures

Primary Outcomes (1)

• 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.

  1 month

Secondary Outcomes (3)

• Number of Participants With Period-Prevalence of Constipation

  1 month

• Mean Patient Reported Outcome (PRO) Scores

  At Visit 1 (1 day)

• Number of Participants Treated for GI Symptoms

  At Visit 1 (1 day)

Study Arms (1)

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.

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Chris Goss: lead
• Emory University: collaborator
• University of North Carolina, Charlotte: collaborator
• University of Texas: collaborator
• Cystic Fibrosis Foundation: collaborator

Study Sites (26)

Stanford University Medical Center

Palo Alto, California, 94304, United States

Location

Central Connecticut Cystic Fibrosis Center

Hartford, Connecticut, 06106, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20816, United States

Location

Nemours Children's Clinic - Pensacola

Pensacola, Florida, 32514, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Children's Healthcare of Atlanta and Emory University

Atlanta, Georgia, 30322, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

OSF Saint Francis Medical Center

Peoria, Illinois, 61637, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Kentucky

Lexington, Kentucky, 40506-9983, United States

Location

Maine Medical Center

Portland, Maine, 04102, United States

Location

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

Boston, Massachusetts, 02115, United States

Location

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, 01655, United States

Location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, 55455, United States

Location

SSM Health Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

Location

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08903, United States

Location

Atrium Health Pulmonary Care

Charlotte, North Carolina, 28203, United States

Location

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, 27103, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Oklahoma Cystic Fibrosis Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern / Children's Health

Dallas, Texas, 75247, United States

Location

Intermountain Cystic Fibrosis Center

Salt Lake City, Utah, 84113, United States

Location

The Children's Specialty Center Fletcher Allen Health Care

Burlington, Vermont, 05401, United States

Location

Related Publications (1)

• 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.

  PMID: 36319569 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis; Constipation

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Dr. Christopher H. Goss M.D., M.Sc.
• Organization: Seattle Children's Hospital

cgoss@uw.edu

(206) 987-5725

Study Officials

• A. Jay Freeman, MD, MSc

  Emory University/Children's Healthcare

  PRINCIPAL INVESTIGATOR

• Baharak Moshiree, MD

  University of North Carolina Charlotte, Atrium Health

  PRINCIPAL INVESTIGATOR

• Meghana Sathe, MD

  University of Texas Southwestern/Children's Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine and Pediatrics

Study Record Dates

• First Submitted: January 4, 2019
• First Posted: January 14, 2019
• Study Start: May 1, 2019
• Primary Completion: September 17, 2019
• Study Completion: September 17, 2019
• Last Updated: October 15, 2024
• Results First Posted: October 15, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (26)