MRI Biomarkers in as Predictor of Clinical Endpoints in Pediatric Autoimmune Liver Disease

Cincinnati Children's Hospital Medical Center

Status: Enrolling

Conditions: Primary Sclerosing Cholangitis; Autoimmune Hepatitis; Autoimmune Liver Disease

Study type: Observational

Funder types: Other

Identifiers

NCT03178630

CIN002 MRI biomarkers in AILD

Details and patient eligibility

About

Autoimmune liver diseases (AILD), which include Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are a common etiological factor for chronic liver disease among adolescents. This is a longitudinal study to identify surrogate endpoints with an accurate predictive value for the progression of hepatobiliary damage in subjects with pediatric onset AILD. This study will involve collection of MRI-based data at the time of enrollment and at year 1 and 2 of follow up, and collection of clinical data for 10 years following enrollment. There is a strong possibility that MRI quantitative techniques may be more sensitive to disease progression than standard clinical and laboratory tests. To investigate predictivity of MRI based biomarkers, summary measures of MRCP/MREL from baseline, Year 1 and Year 2, e.g. change rate, maximum, and average will be calculated as predictors for Year 10 clinical outcomes. The same predictors will also be used to model native liver survival in a proportional hazard regression. Findings from this study may be used to assess disease progression and to predict complications and survival of liver disease patients.

Enrollment: 150 estimated patients

Sex: All

Ages: 6 to 23 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age 6-23 years old.
2. Established clinical diagnosis of AIH or PSC.

Exclusion criteria

1. History of liver transplantation.
2. Chronic Hepatitis B or untreated hepatitis C virus infection.
3. Pregnancy.
4. Absolute contraindication for MRI (e.g. pacemaker, metallic implants, claustrophobia).
5. Diagnosis of cystic fibrosis or biliary atresia
6. Diagnosis of cardiac hepatopathy.
7. Diagnosis of Wilson's disease, Alpha-1 Antitrypsin deficiency, or Glycogen storage disease.
8. Skin conditions which could be aggravated by MREL (i.e. Epidermolysis bullosa).

Trial design

150 participants in 1 patient group

Patients with autoimmune liver disease

Description:

Patients with autoimmune liver disease Patients (6-23 y.o.) with established clinical diagnosis of AIH or suspected diagnosis of AIH based on elevated serum AST or ALT, elevated IgG level \>1.1 ULN, elevated titer of autoantibodies, including ANA, SMA, LKM, LC-1 or SLA, which is consistent with the simplified criteria for the diagnosis of AIH in children will be enrolled. Patients (6-23 y.o.) with established clinical diagnosis of PSC or Suspected diagnosis of PSC supported by abnormal cholangiogram (ERCP or MRCP) or elevated GGT\>1.5 ULN and dilated bile ducts by liver ultrasound will be enrolled.