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

NCT02131623 | Completed

• 1 other identifier: LUM1038
• Study Type: observational
• Target: 23
• Locations: 2 countries
• Sites: 5

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.

Conditions

Alagille Syndrome; Progressive Familial Intrahepatic Cholestasis

Outcome Measures

Primary Outcomes (1)

• Validation of ItchRO measure

  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

Study Arms (1)

Validation Group

Subjects with ALGS or PFIC and/or their caregivers

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects (children) with ALGS or PFIC and/or caregivers of subjects with ALGS or PFIC

You may qualify if:

• 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).
• 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 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.

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (5)

Phoenix Childrens Hospital

Phoenix, Arizona, 85016-7710, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Alagille Syndrome; Cholestasis, progressive familial intrahepatic 1

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Study Director

  Mirum

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2014
• First Posted: May 6, 2014
• Study Start: July 1, 2014
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: March 18, 2019

Record last verified: 2019-03

Locations

US (4); CA (1)