A Comprehensive Approach To Relief Of Digestive Symptoms In Cystic Fibrosis: CARDS-CF (CARDS-CF)

Development of a new patient reported outcome measure (PROM) that will measure the daily burden of gastrointestinal symptoms over the previous 24 hour period for people with cystic fibrosis.

Study Overview

• Status: Recruiting
• Conditions: Respiratory Tract Diseases; Lung Diseases; Cystic Fibrosis; Genetic Disease; Digestive System Disease; Pancreatic Disease
• Intervention / Treatment: Other: Focus group; Other: Patient interview (n = approximately 10); Other: Questionnaire (n = 180); Device: Testing of the pilot PROM in a smartphone app (n = 100)

Detailed Description

Development of a Patient Reported Outcome Measure (PROM) in line with FDA guidance. The PROM is intended for daily use to measure the symptom burden of gastrointestinal symptoms for people with CF over the last 24 hours.

• Development of the initial conceptual framework underpinning the PROM and confirmed through a patient focus group.
• Primary item (question) generation for the PROM which will then be refined through a series of interviews with people with CF.
• Testing of the items through an online patient questionnaire for testing of frequency, burden and impact of the items.
• Piloting testing of the draft PROM daily for 2 weeks via smartphone app. The recall period of the PROM will be gastrointestinal symptom burden over the previous 24 hours.

Potential participant's will be identified through 6 UK CF care centres at Nottingham University Hospitals NHS Trust (paediatric and adult CF centres), Leeds Teaching Hospital NHS Trust, Manchester University NHS Foundation trust, Royal Brompton and Harefield NHS Foundation Trust and Kings College Hospital NHS Foundation Trust. In addition, recruitment for the survey and trialing the PROM on a smartphone app will also take place via social media and is therefore also open to people with CF from outside these CF centres.

• Study Type: Observational
• Enrollment (Anticipated): 300

Contacts and Locations

• Study Contact: Name: Alan Smyth; Phone Number: 0115 8230612; Email: alan.smyth@nottingham.ac.uk
• Study Contact Backup: Name: Rebecca Calthorpe; Email: rebecca.calthorpe@nottingham.ac.uk

Study Locations

• United Kingdom
  • Nottingham, United Kingdom, NG7 2UH
    • Recruiting
    • Nottingham University Hospitals NHS Trust
    • Principal Investigator: Alan Smyth
    • Contact: Alan Smyth; Email: alan.smyth@nottingham.ac.uk

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

People with cystic fibrosis age 12 years and over

Description

Inclusion Criteria:

• People with cystic fibrosis age 12 years and over
• Confirmed diagnosis of cystic fibrosis based on genotype or sweat chloride testing
• Capacity to consent, or to understand the requirements of the study where parental consent is required

Exclusion Criteria:

• < 12 years age
• Unable to give informed consent

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

People with CF age 12 years and over; Participants will be identified through one of the 6 participating UK CF centres as well as recruiting via social media. Participants outside the 6 listed CF centres as well as outside the UK are also eligible.

Other: Focus group; Focus group to confirm PROM conceptual framework; Other: Patient interview (n = approximately 10); Patient interviews to refine possible questions for the PROM; Other: Questionnaire (n = 180); Online questionnaire to evaluate the proposed questions as part of PROM development in a larger population; Device: Testing of the pilot PROM in a smartphone app (n = 100); Daily testing of the pilot PROM in a smartphone app for 2 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To develop a new patient reported outcome measure (PROM) which will measure the daily gastrointestinal symptom burden for people with CF, with a recall period of 24 hours.	Development of a new PROM following FDA guidance which will measure the daily impact and burden of gastrointestinal symptoms for people with CF. The recall period will be the previous 24 hours. The name of the PROM will be decided as part of the development process. Question development will be iterative. Floor and ceiling effects for each of the questions will be assessed. The CFAbd score will be used to assess construct validity of the newly developed PROM. This will be completed alongside the PROM on days 1 and day 14.	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of adherence to daily data capture of the PROM through a smartphone app over a 2 week period	Adherence data - Percentage of completed PROM days over the 14 day period. Percentage of completed CFabd scores on days 1 and 14. This data will be collated through the research portal feedback facility in the app.	2 years
Attrition rate of participants	Attrition rate over the 2 weeks, define as people completing the app for two weeks (at least 10 out of 14 days) as a percentage of those initially who gave consent to take part and use the app.	2 years
Usability of the smartphone app	Usability of the app - participant feedback via the app on days 7 and 14 to include questions of ease of data entry on the app, whether they experience any technical issues with the app and whether questions are meaningful to participants. These questions will be developed as part of the PROM development process.	2 years

Collaborators and Investigators

• Sponsor: Nottingham University Hospitals NHS Trust
• Collaborators: King's College Hospital NHS Trust; The Leeds Teaching Hospitals NHS Trust; Manchester University NHS Foundation Trust; Royal Brompton & Harefield NHS Foundation Trust; Medizinische Hochschule Brandenburg Theodor Fontane

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2022
• Primary Completion (Anticipated): September 30, 2023
• Study Completion (Anticipated): September 30, 2023

Study Registration Dates

• First Submitted: February 4, 2022
• First Submitted That Met QC Criteria: February 11, 2022
• First Posted (Actual): February 22, 2022

Study Record Updates

• Last Update Posted (Actual): February 2, 2023
• Last Update Submitted That Met QC Criteria: January 31, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: Abdomen; Gut; Patient reported outcome measure (PROM); Gastrointestinal symptom burden; Symptom score
• Additional Relevant MeSH Terms: Pathologic Processes; Infant, Newborn, Diseases; Fibrosis; Lung Diseases; Gastrointestinal Diseases; Digestive System Diseases; Cystic Fibrosis; Pancreatic Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn
• Other Study ID Numbers: 21CS025

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No