Alagille Syndrome Clinical Trial
Official title:
Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
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.
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.
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