ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 8 of 14 for:    Alagille Syndrome

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02131623
Recruitment Status : Completed
First Posted : May 6, 2014
Last Update Posted : June 12, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

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.

Condition or disease
Alagille Syndrome Progressive Familial Intrahepatic Cholestasis

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 : Observational
Actual Enrollment : 23 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
Study Start Date : July 2014
Actual Primary Completion Date : March 2015
Actual Study Completion Date : March 2015


Group/Cohort
Validation Group
Subjects with ALGS or PFIC and/or their caregivers



Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects (children) with ALGS or PFIC and/or caregivers of subjects with ALGS or PFIC
Criteria

Inclusion Criteria for subjects with ALGS or PFIC

  1. The subject has voluntarily provided verbal assent or written assent in accordance with the site's Institutional Review Board (IRB) guidelines;
  2. The subject's legal guardian has voluntarily provided permission for study participation and authorization for access to personal health information in conjunction with HIPPA;
  3. The subject has a diagnosis of ALGS or PFIC;
  4. The subject is able to speak and comprehend US English (applicable only for children age 5 and older);
  5. The subject is willing and able to participate in the two-week validation study (applicable only for children age 5 and older); and
  6. 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:

  1. The caregiver voluntarily provided written informed consent;
  2. The caregiver is 18 years of age or older;
  3. The caregiver has served as a consistent caregiver for the subject with ALGS or PFIC;
  4. The caregiver's child or subject with ALGS or PFIC meets all of the study inclusion criteria for subjects with ALGS or PFIC;
  5. The caregiver is able to speak, read, write, and comprehend US English;
  6. The caregiver is willing and able to participate in the two-week validation study; and
  7. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02131623


Locations
United States, Arizona
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016-7710
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Missouri
St. Louis Children's Hospital
St. Louis, Missouri, United States, 63110
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02131623     History of Changes
Other Study ID Numbers: LUM1038
First Posted: May 6, 2014    Key Record Dates
Last Update Posted: June 12, 2015
Last Verified: June 2015

Additional relevant MeSH terms:
Alagille Syndrome
Liver Diseases
Cholestasis
Cholestasis, Intrahepatic
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn