Canadian Network for Autoimmune Liver Disease

Study Overview
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Brief Summary
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CaNAL is a longitudinal observational cohort study of patients diagnosed with Primary Biliary Cholangitis (PBC), Autoimmune Hepatitis (AIH), or overlap syndrome. This study creates a nationwide registry and network focusing on high quality long-term follow-up of individual patient data from major Canadian centers. Primary Biliary Cholangitis (PBC) and Autoimmune Hepatitis (AIH) are rare and slowly progressive liver diseases associated with development of cirrhosis, liver cancer (HCC) and liver failure requiring liver transplantation or leading to premature death. The rarity and slowly progressive nature of these autoimmune liver diseases make them difficult to study and only a large scale approach combining patient data from multiple centers across Canada will allow new insights. The primary aim of the Canadian Network for Autoimmune Liver Disease is to build a Canadian registry of patients with PBC, AIH, and overlap syndrome. We capture patient characteristics, laboratory assessments and natural history, patient-reported outcomes including quality of life measures and environmental exposures, response to treatment, and pre- and post-transplant outcomes. We will then identify risk factors associated with critical outcomes for the patient, including response to treatment, progression to transplant, risk of liver cancer, and recurrent disease after transplant. We can identify biomarkers (biochemical indicators of progression of disease) to help diagnose autoimmune liver disease at its earliest stages, ensuring timely treatment and preventing disease progression. CaNAL will provide a better understanding of autoimmune liver diseases, biomarkers predictive of disease progression or non-response to therapy as well as better knowledge of the etiology and pathogenesis. CaNAL will also help to serve as a platform for conducting clinical trials or targeted lab-studies to answer important questions that are unlikely to be evaluated by the pharmaceutical industry.

Official Title
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Canadian Network for Autoimmune Liver Disease

Conditions
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Primary Bilary Cirrhosis (PBC), Autoimmune Hepatitis, Overlap Syndrome

Intervention / Treatment
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* Other: Observational; no intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Primary Biliary Cholangitis and/or Autoimmune Hepatitis Exclusion Criteria: Less than 18 years of age

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Observational<br>There is no intervention being administered. This registry only observes patients through their regular standard of care visits. | Other: Observational; no intervention<br>* Observational; no intervention<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver transplant-free survival over time | Time-to-event | Up to 6 years or time of death/liver transplant | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Biochemical values over time (e.g. aspartate aminotransferase, alkaline phosphatase, alanine aminotransferase, Bilirubin) | Investigate trends of different biochemical values over time | Through study completion, an average of 6 months | 
| Short Form 36 (SF-36) | Health-Related Quality of Life | Through study completion, an average of 6 months | 
| PBC-40 | Health-Related Quality of Life, specific to Primary Biliary Cholangitis | Through study completion, an average of 6 months | 
| 5D Pruritus Scale | Itch-Related Quality of Life through 5 dimensions: duration, degree, direction, disability, and distribution. Each dimension is scored from 1(lowest) to 5(highest) for a total score range of 5-25 | Through study completion, an average of 6 months | 
| Itch Visual Analog Scale | Itch-Related Quality of Life; Continuous unitless itch scale from no itch to worst itch imaginable | Through study completion, an average of 6 months | 
| Itch Numeric Rating Scale | Itch-Related Quality of Life; Categorical integer scale from 0 (no itch) to 10 (worst itch imaginable) | Through study completion, an average of 6 months |