Skip to main content
OpenTrials
Recruiting

NCT Number: NCT03178630

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Autoimmune Liver Disease Autoimmune Diseases Autoimmune Hepatitis Bile Duct Diseases Biliary Tract Diseases Cholangitis Cholangitis, Sclerosing Digestive System Diseases Hepatitis Hepatitis, Autoimmune Hepatitis, Chronic Immune System Diseases Liver Diseases Primary Sclerosing Cholangitis

Age range

6 year–23 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Primary outcomes

  1. Change of intrahepatic bile duct irregularities between V0 (baseline visit) and V1 (visit after12 months) or V2 (visit after 24 months).

    Time frame: 24 months

    Change of intrahepatic bile duct irregularities between V0 and V1 or V2 by MRCP (scored by Majoie classification on 4 point scale of 0-3).

  2. Change of extra-hepatic duct irregularities between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).

    Time frame: 24 months

    Change of extra-hepatic duct irregularities between V0 and V1 or V2 by MRCP (scored by Majoie classification on 5 point scale 0-4).

  3. Mean shear stiffness of the liver

    Time frame: 24 months

    Change in mean shear stiffness (kPa) of the liver by MREL between V0 (baseline visit) and V1 ( visit after 12 months) or V2 (visit after 24 months).

  4. long-term clinical outcomes: survival with the native liver

    Time frame: 120 months

    Annual assessment of survival with the native liver (Yes=1, No=0) will be done within 10 years of follow-up.

  5. long-term clinical outcomes: hospital admissions for cholangitis

    Time frame: 120 months

    Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Any hospital admissions for cholangitis (Yes=1, No=0) since last visit will be recorded at the time of follow-up.

  6. long-term clinical outcomes:endoscopic interventions for biliary strictures

    Time frame: 120 months

    Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Endoscopic interventions for biliary strictures (Yes=1, No=0) since last visit will be recorded at the time of follow-up.

  7. long-term clinical outcomes:diagnosis of cholangiocarcinoma

    Time frame: 120 months

    Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. If there is diagnosis of cholangiocarcinoma (Yes=1, No=0) since last visit will be recorded.

  8. long-term clinical outcomes: variceal bleeding

    Time frame: 120 months

    Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Presence or absence of variceal bleeding (Yes=1, No=0) since last visit will be recorded.

  9. long-term clinical outcomes: ascites

    Time frame: 120 months

    Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Presence or absence of ascites (Yes=1, No=0) since last visit will be recorded.

Secondary outcomes

  1. Changes in liver/spleen volumes

    Time frame: 24 months

    Changes in liver/spleen volumes (mL) between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).

  2. Changes in T1rho, T1 and T2 mapping

    Time frame: 24 months

    Readouts of T1rho, T1 and T2 mapping between baseline MRI at V0 (baseline visit) and repeat ones at V1 (after12 months) or V2 (after 24 months) in msec.

  3. Clinical endpoints of AILD: Pruritus

    Time frame: 120 months

    Annual assessment for Pruritus (on visual analogue scale of 0-10) will be done within 10 years of follow-up.

  4. Clinical endpoints of AILD

    Time frame: 120 months

    Annual assessment for Clinical diagnosis of hepatopulmonary syndrome (Yes=1, No=0) and/or hepatic encephalopathy (Yes=1, No=0) will be done within 10 years of follow-up.

Study contacts

Contact information is provided by the study sponsor or research team.

Alexander Miethke, MD

CONTACT

[email protected]

513-636-8948

Cyd Castro Rojas, PhD

CONTACT

[email protected]

513-517-0580

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Registry information

Official study title

Longitudinal Study for the Assessment of MRI Based Biomarkers as a Predictors of Clinical Endpoints in Pediatric Onset Autoimmune Liver Disease

Important dates

Study start
2017
Primary completion
2030
Study completion
2031
First posted
Jun 7, 2017
Registry last updated
Dec 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.