Development of CELIAC-Q KIDS: A Patient-Reported Outcome Measure for Pediatric Celiac Disease

Phase 1 Protocol to Develop a Patient-reported Outcome Measure for Children and Adolescents With Celiac Disease: CELIAC-Q KIDS

A multicentre, prospective observational study to develop the CELIAC-Q KIDS patient reported outcome measure for children and adolescents with celiac disease. The CELIAC- Q KIDS will contain a comprehensive set of independently functioning scales designed to measure outcomes that matter to children with celiac disease, as well as scales to measure patients experience with the gluten-free diet.

Study Overview

• Status: Active, not recruiting
• Conditions: Celiac Disease; Celiac Disease in Children; Patient-reported Outcome Measures
• Intervention / Treatment: Other: Interview and scale development

Detailed Description

The study will be conducted in three stages:

Stage I - Conceptual framework development: A scoping review will be carried out to map initial concepts that have been measured by patient report in pediatric patients with celiac disease. Findings will be used to develop a preliminary conceptual framework.

Stage II - Item generation: Approximately 20 interviews will be conducted with children and adolescents with celiac disease and their caregivers to capture their experiences including quality of life, symptom experience, and other relevant themes that may emerge. The interviews will be audio recorded and transcribed verbatim. The qualitative data will be analyzed thematically, and key quotes extracted which will be used to create a draft item list. Interviews will be conducted either in person or virtually depending on patient preference. The data from Stage I will be used to develop a preliminary version of the CELIAC-Q KIDS patient-reported outcome measure.

Stage II - Scale refinement:

The CELIAC-Q KIDS scales will be refined through multiple rounds of cognitive debriefing interviews using the "think aloud method." During each interview round, approximately 7 children and adolescents with celiac disease will be interviewed to determine if patients understand the instructions, response options and items of the CELIAC-Q KIDS instrument and to identify missing content. Interviews will take place in a series of rounds to allow time to make changes to the instrument and then obtain feedback on those changes until data saturation is achieved.

Between rounds of cognitive debriefing interviews, the CELIAC-Q KIDS will be shown to experts for feedback. A multidisciplinary, international group of approximately 10 healthcare providers who care for children and adolescents with celiac disease will be surveyed to determine if healthcare providers feel there are any items missing from the patient-reported outcome measure, if any items are not relevant and to provide feedback on the instructions and response options. Once again, feedback provided by the experts will be used to revise the CELIAC-Q KIDS scales.

• Study Type: Observational
• Enrollment (Anticipated): 100

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • McMaster Children's Hospital
    • Toronto, Ontario, Canada, M5G 1X8
      • SickKids | The Hospital for Sick Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 17 years (CHILD)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

This study will include participants from the McMaster Children's Hospital Pediatric Celiac Disease Clinic and the Hospital for Sick Children (SickKids) Children's Celiac Clinic. Patients who have confirmed celiac disease (based on biopsy and/or serology) will be recruited to participant. Patients aged 1 to 8 years of age will participate in the interviews with their caregiver, whereas caregivers will participate on their own when their child is under the age of 8. Both genders will be recruited in patient and parent groups.

Description

Inclusion Criteria:

• Patients diagnosed with celiac disease.
• Pediatric patients (18 years and younger).
• Ability to understand and communicate in the English language

Exclusion Criteria:

• Patients who do not have celiac disease.
• Non-pediatric patients (over 18).
• Unable to understand and communicate in the English language

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pediatric Patients; Pediatric patients with celiac disease.	Other: Interview and scale development; Interview and scale development
Caregivers and parents; Caregivers and parents of pediatric patients with celiac disease	Other: Interview and scale development; Interview and scale development
Experts; Group of experts in pediatric celiac disease to provide feedback on scale development.	Other: Interview and scale development; Interview and scale development

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Development of the patient-reported outcome measure: Consensus on items that will comprise the newly developed disease-specific patient-reported outcome measure for pediatric celiac disease	Development of the CELIAC-Q KIDS scales: qualitative interview and survey-based data will be analyzed qualitatively to develop and refine the items that comprise the scales	48 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Qualitative interviews - for item generation	Patient participants	24 months
Cognitive debriefing interviews - qualitative feedback on scale instructions, response options and items	Patient participants	24 months
Expert survey data - qualitative feedback on scale instructions, response options and items	Expert participant questionnaire	24 months

Collaborators and Investigators

• Sponsor: McMaster University
• Collaborators: The Hospital for Sick Children; Canadian Celiac Association

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 26, 2020
• Primary Completion (ANTICIPATED): December 31, 2025
• Study Completion (ANTICIPATED): December 31, 2026

Study Registration Dates

• First Submitted: September 16, 2022
• First Submitted That Met QC Criteria: September 16, 2022
• First Posted (ACTUAL): September 21, 2022

Study Record Updates

• Last Update Posted (ACTUAL): November 4, 2022
• Last Update Submitted That Met QC Criteria: November 2, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Metabolic Diseases; Gastrointestinal Diseases; Intestinal Diseases; Malabsorption Syndromes; Celiac Disease
• Other Study ID Numbers: 11126 (DAIDS ES Registry Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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