- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02131623
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
Study Overview
Status
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 Type
Enrollment (Actual)
Contacts and Locations
Study Locations
-
-
Ontario
-
Toronto, Ontario, Canada, M5G 1X8
- The Hospital for Sick Children
-
-
-
-
Arizona
-
Phoenix, Arizona, United States, 85016-7710
- Phoenix Childrens Hospital
-
-
Colorado
-
Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
-
-
Missouri
-
Saint Louis, Missouri, United States, 63110
- St. Louis Children's Hospital
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria for subjects with ALGS or PFIC
- The subject has voluntarily provided verbal assent or written assent in accordance with the site's Institutional Review Board (IRB) guidelines;
- The subject's legal guardian has voluntarily provided permission for study participation and authorization for access to personal health information in conjunction with HIPPA;
- The subject has a diagnosis of ALGS or PFIC;
- The subject is able to speak and comprehend US English (applicable only for children age 5 and older);
- The subject is willing and able to participate in the two-week validation study (applicable only for children age 5 and older); and
- The subject is able to understand the nature, scope, and possible consequences of the study, and/or does not present evidence of an uncooperative attitude (applicable only for children age 5 and older).
Inclusion Criteria for caregivers of subjects with ALGS or PFIC:
- The caregiver voluntarily provided written informed consent;
- The caregiver is 18 years of age or older;
- The caregiver has served as a consistent caregiver for the subject with ALGS or PFIC;
- The caregiver's child or subject with ALGS or PFIC meets all of the study inclusion criteria for subjects with ALGS or PFIC;
- The caregiver is able to speak, read, write, and comprehend US English;
- The caregiver is willing and able to participate in the two-week validation study; and
- The caregiver is able to understand the nature, scope, and possible consequences of the study, and/or does not present evidence of an uncooperative attitude.
Study Plan
How is the study designed?
Design Details
- Observational Models: Case-Only
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
---|
Validation Group
Subjects with ALGS or PFIC and/or their caregivers
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Validation of ItchRO measure
Time Frame: 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.
|
2 weeks
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- LUM1038
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Alagille Syndrome
-
TakedaActive, not recruiting
-
AlbireoActive, not recruitingAlagille SyndromeUnited Kingdom, United States, France, Germany, Netherlands, Italy, Belgium, Malaysia, Poland, Turkey
-
Mirum Pharmaceuticals, Inc.Completed
-
Mirum Pharmaceuticals, Inc.Lumena Pharmaceuticals, Inc.; Childhood Liver Disease Research and Education...Completed
-
Mirum Pharmaceuticals, Inc.CompletedAlagille SyndromeUnited Kingdom
-
Mirum Pharmaceuticals, Inc.RecruitingAlagille SyndromeUnited States
-
AlbireoCompletedAlagille SyndromeUnited States, United Kingdom, France, Germany, Canada, Netherlands, Italy, Malaysia, Belgium, Israel, New Zealand, Poland, Turkey
-
National Human Genome Research Institute (NHGRI)Completed
-
Mirum Pharmaceuticals, Inc.Childhood Liver Disease Research and Education NetworkCompletedAlagille SyndromeUnited States, Canada
-
Mirum Pharmaceuticals, Inc.Clinigen, Inc.Approved for marketingAlagille SyndromeUnited States