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

Cystic Fibrosis Clinical Trial

— GALAXY  

Official title:

Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03801993
• Other study ID #: GALAXY-OB-18
• Status: Completed
• Start date: May 1, 2019
• Est. completion date: September 17, 2019

Study information

• Verified date: October 2019
• Source: Seattle Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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 information / eligibility

• Status: Completed
• Enrollment: 402
• Est. completion date: September 17, 2019
• Est. primary completion date: September 17, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

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

Sponsors (5)

Lead Sponsor	Collaborator
Chris Goss	Cystic Fibrosis Foundation, Emory University, University of North Carolina, Charlotte, University of Texas

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of completed assessments	Percentage of scheduled outside-the-clinic assessments with at least one of the four PROs fully completed.	1 month
Secondary	Prevalence of Constipation	Percentage of enrolled subjects with any occurrence of protocol-defined constipation during 1 month of follow-up (the period prevalence)	1 month
Secondary	Mean Patient Reported Outcome (PRO) Scores	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.	At Visit 1 (1 day)
Secondary	Percentage of subjects treated for GI symptoms	Percentage of enrolled subjects receiving treatment for GI symptoms at Visit 1	At Visit 1 (1 day)