Ignite Creation Date:
2024-05-06 @ 2:48 AM
Last Modification Date:
2024-10-26 @ 11:24 AM
Study NCT ID:
NCT02131623
Status:
COMPLETED
Last Update Posted:
2019-03-18
First Post:
2014-05-01
Brief Title:
Validation of the Itch Reported Outcome ItchRO Diaries in Pediatric Cholestatic Liver Disease
Sponsor:
Mirum Pharmaceuticals Inc
Organization:
Mirum Pharmaceuticals Inc
Study Overview
Official Title:
Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
Status:
COMPLETED
Status Verified Date:
2019-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 childrens 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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None