Clinical trial NCT03178630: MRI Biomarkers in as Predictor of Clinical Endpoints in Pediatric Autoimmune Liver Disease
Official title: Longitudinal Study for the Assessment of MRI Based Biomarkers as a Predictors of Clinical Endpoints in Pediatric Onset Autoimmune Liver Disease
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Autoimmune Liver Disease
Autoimmune Hepatitis
Primary Sclerosing Cholangitis
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2017-06-07
Enrollment: 150
Sex: ALL
Minimum age (years): 6
Maximum age (years): 23
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: A total of 150 patients between 6 and 23 years of age with a diagnosis of PSC or AIH will be enrolled and followed up to 10 Years.

Brief summary: 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.


Eligibility criteria: 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).

Primary outcomes:
measure: Change of intrahepatic bile duct irregularities between V0 (baseline visit) and V1 (visit after12 months) or V2 (visit after 24 months).
timeFrame: 24 months
measure: Change of extra-hepatic duct irregularities between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).
timeFrame: 24 months
measure: Mean shear stiffness of the liver
timeFrame: 24 months
measure: long-term clinical outcomes: survival with the native liver
timeFrame: 120 months
measure: long-term clinical outcomes: hospital admissions for cholangitis
timeFrame: 120 months
measure: long-term clinical outcomes:endoscopic interventions for biliary strictures
timeFrame: 120 months
measure: long-term clinical outcomes:diagnosis of cholangiocarcinoma
timeFrame: 120 months
measure: long-term clinical outcomes: variceal bleeding
timeFrame: 120 months
measure: long-term clinical outcomes: ascites
timeFrame: 120 months
Secondary outcomes:
measure: Changes in liver/spleen volumes
timeFrame: 24 months
measure: Changes in T1rho, T1 and T2 mapping
timeFrame: 24 months
measure: Clinical endpoints of AILD: Pruritus
timeFrame: 120 months
measure: Clinical endpoints of AILD
timeFrame: 120 months
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital and Medical Center
state: Ohio
Study officials:
affiliation: Cincinnati Childrens Hospital Medical Center
name: Alexander Miethke, MD
role: PRINCIPAL_INVESTIGATOR
References: