Actively Recruiting
PEARL - Pediatric Evaluation and Registry for Liver Cholestasis in Canada
Led by Children's Hospital of Eastern Ontario · Updated on 2026-05-04
220
Participants Needed
13
Research Sites
39 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Eastern Ontario
Lead Sponsor
M
Mirum Pharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are creating a national, multi-center registry in Canada to follow children under 18 years old with Alagille syndrome (ALGS) or Genetic Intrahepatic Cholestasis (GIC), including various subtypes of Progressive Familial Intrahepatic Cholestasis (PFIC). This observational study aims to collect standardized, high-quality long-term data to better understand these rare genetic liver diseases and their outcomes across pediatric hepatology centers. Participants, diagnosed either retrospectively (since January 1, 2022) or prospectively, will have data collected from medical charts and regular follow-ups. Retrospective data includes demographics, genetic results, clinical history, lab tests, imaging, and major clinical events. Prospective follow-up occurs every 4-6 months with clinical assessments, labs, imaging, and event tracking until transition to adult care or study end. During the study, participants will undergo scheduled visits every 6 months for symptom review, laboratory tests, imaging including elastography, and growth measurements. Researchers will monitor events such as liver transplant, hospitalizations, and complications. Data is securely managed using electronic systems ensuring privacy. The primary outcome measured is event-free survival over 2-5 years, tracking survival without liver transplant, surgery, liver failure, or death.
CONDITIONS
Brief Title
Pediatric Evaluation and Registry for Liver Cholestasis in Canada
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Pediatric participants under 18 years old with genetically confirmed or clinically diagnosed Alagille syndrome or subtypes of Genetic Intrahepatic Cholestasis
- Enrollment at participating Canadian pediatric liver centers
- Written informed consent from participant (if capable) or parent/guardian and assent from participant as appropriate
You will not qualify if you...
- Inability to comply with follow-up requirements or lost to follow-up
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Eligible participants provide informed consent and complete baseline data collection including demographics, clinical symptoms, genetic testing, laboratory tests, imaging, and growth parameters.
1 baseline visit (in-person)
Duration - Up to 8 years or until transition to adult care or withdrawal
Participants are followed prospectively with structured visits every 4 to 6 months for reassessment of clinical symptoms, laboratory and imaging tests, and documentation of clinical events related to liver disease.
Visits every 4 to 6 months
Duration - Variable, up to several years prior to enrollment
For participants diagnosed previously, annual retrospective follow-up forms capture key clinical milestones and outcomes from diagnosis until enrollment.
Annual data collection forms completed retrospectively without participant visits
Trial Site Locations
Total: 13 locations
1
Alberta Children's Hospital
Calgary, Alberta, Canada
Not Yet Recruiting
2
Stollery Children's Hospital
Edmonton, Alberta, Canada
Not Yet Recruiting
3
BC Children's Hospital
Vancouver, British Columbia, Canada
Not Yet Recruiting
4
HSC Winnipeg Children's Hospital
Winnipeg, Manitoba, Canada
Not Yet Recruiting
5
Janeway Children's Health and Rehabilitation Centre
St. John's, Newfoundland and Labrador, Canada
Not Yet Recruiting
6
Children's Hospital IWK Health Centre
Halifax, Nova Scotia, Canada
Not Yet Recruiting
7
McMaster Children's Hospital
Hamilton, Ontario, Canada
Not Yet Recruiting
8
Children's Hospital LHSC
London, Ontario, Canada
Not Yet Recruiting
9
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Actively Recruiting
10
CHU Sainte-Justine
Montreal, Quebec, Canada
Not Yet Recruiting
11
Montreal Children's Hospital
Montreal, Quebec, Canada
Not Yet Recruiting
12
CHU de Quebec - Universite Laval (Centre Mere-Enfant Soleil)
Qubec City, Quebec, Canada
Not Yet Recruiting
13
Jim Pattison Children's Hospital
Saskatoon, Saskatchewan, Canada
Not Yet Recruiting
Research Team
M
Mohit Kehar
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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