NCT05251467

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
350

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Feb 2022

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 4, 2022

Completed
18 days until next milestone

First Posted

Study publicly available on registry

February 22, 2022

Completed
6 days until next milestone

Study Start

First participant enrolled

February 28, 2022

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 3, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 3, 2023

Completed
Last Updated

July 22, 2024

Status Verified

July 1, 2024

Enrollment Period

1.8 years

First QC Date

February 4, 2022

Last Update Submit

July 18, 2024

Conditions

Cystic FibrosisDigestive System DiseaseGenetic DiseaseRespiratory Tract DiseasesLung DiseasesPancreatic Disease

Keywords

Patient reported outcome measure (PROM)Gastrointestinal symptom burdenSymptom scoreGutAbdomen

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (3)

  • Measurement of adherence to daily data capture of the PROM through a smartphone app over a 2 week period

    2 years

  • Attrition rate of participants

    2 years

  • Usability of the smartphone app

    2 years

Study Arms (1)

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 groupOther: Patient interview (n = approximately 10)Other: Questionnaire (n = 180)Device: Testing of the pilot PROM in a smartphone app (n = 150)

Interventions

Focus groupOTHER

Focus group to confirm PROM conceptual framework

People with CF age 12 years and over
Patient interview (n = approximately 10)OTHER

Patient interviews to refine possible questions for the PROM

People with CF age 12 years and over
Questionnaire (n = 180)OTHER

Online questionnaire to evaluate the proposed questions as part of PROM development in a larger population

People with CF age 12 years and over
Testing of the pilot PROM in a smartphone app (n = 150)DEVICE

Daily testing of the pilot PROM in a smartphone app for 2 weeks

People with CF age 12 years and over

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

People with cystic fibrosis age 12 years and over

You may qualify if:

  • 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

You may not qualify if:

  • \< 12 years age
  • Unable to give informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nottingham University Hospitals NHS Trust

Nottingham, NG7 2UH, United Kingdom

Location

MeSH Terms

Conditions

Cystic FibrosisDigestive System DiseasesGenetic Diseases, InbornRespiratory Tract DiseasesLung DiseasesPancreatic Diseases

Interventions

Focus GroupsSurveys and Questionnaires

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Data CollectionEpidemiologic MethodsInvestigative TechniquesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public Health

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 4, 2022

First Posted

February 22, 2022

Study Start

February 28, 2022

Primary Completion

December 3, 2023

Study Completion

December 3, 2023

Last Updated

July 22, 2024

Record last verified: 2024-07

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)