Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease

Completed

• Conditions: Progressive Familial Intrahepatic Cholestasis; Alagille Syndrome

• Registration Number: NCT02131623
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary

The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.

Detailed Description

This research will examine the psychometric performance of the electronic diaries for the itch-reported outcome (ItchRO) measure for both patients and caregivers in a stand-alone 2-week validation study in the target patient population. The study is non-interventional in nature; the purpose of the study is to validate the ItchRO prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials. Following training and initial completion of the electronic diaries, families will return home and complete the diaries twice daily over the course of two weeks. Additional assessments include the numerical rating scale to assess itching, the patient impression of change (PIC), the caregiver impression of change (CIC), PedsQL (pediatric quality of life), children's sleep habits questionnaire (CSHQ), and clinical scratch scale.

Longitudinal data will be used to follow the natural (non-interventional) course of the disease and the quality of the data. Test-retest reliability will be assessed using diary data from Day 1 to Day 14 in stable subjects as measured by the PIC for the child-completed measure and the CIC for the caregiver measure. Convergent validity (correlations between the ItchRO and similar concepts on the other measures included in the study) will also be explored (such as the correlation between sleep and morning ItchRO diary scores). Clinical validity (correlation between the ItchRO and clinician reports) and preliminary responsiveness of those who changed in their itching status over the two week period will also be assessed if sample sizes allow.

Study Timeline

• Study First Submitted: 2014-05-01
• Study First Submitted QC: 2014-05-02
• Study First Posted: 2014-05-06
• Study Start: 2014-07
• Primary Completion: 2015-03
• Study Completion: 2015-03
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-14
• Last Update Posted: 2019-03-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Validation of ItchRO measure	2 weeks	Validate the ItchRO measure with subjects with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) and/or their caregivers through the completion of the electronic diary twice daily over the course of two weeks.

Trial Locations

Locations (5)

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

Phoenix Childrens Hospital; 🇺🇸 Phoenix, Arizona, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

St. Louis Children's Hospital; 🇺🇸 Saint Louis, Missouri, United States