Alagille Syndrome Clinical Trial
Official title:
Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
NCT number | NCT02131623 |
Other study ID # | LUM1038 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | July 2014 |
Est. completion date | March 2015 |
Verified date | March 2019 |
Source | Mirum Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 23 |
Est. completion date | March 2015 |
Est. primary completion date | March 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 5 Years and older |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
Canada | The Hospital for Sick Children | Toronto | Ontario |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Phoenix Childrens Hospital | Phoenix | Arizona |
United States | St. Louis Children's Hospital | Saint Louis | Missouri |
Lead Sponsor | Collaborator |
---|---|
Mirum Pharmaceuticals, Inc. |
United States, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 |
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