Autoimmune Liver DiseaseNCT03178630
MRI Biomarkers in as Predictor of Clinical Endpoints in Pediatric Autoimmune Liver Disease
An observational study of Autoimmune Liver Disease and Autoimmune Hepatitis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 23 years
- Conditions the study is for
- Autoimmune Liver Disease, Autoimmune Hepatitis, Primary Sclerosing Cholangitis
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Feb 2017to Feb 2031
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 23 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- Age 6-23 years old.
- Established clinical diagnosis of AIH or PSC.
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cincinnati Children's Hospital and Medical Center
Cincinnati, Ohio, 45229
Alexander Miethke, MD, 513-636-9078, alexander.miethke@cchmc.org
Cyd Castro Rojas, PhD, 513-517-0580, cyd.castrorojas@cchmc.org
About this study
Official title: Longitudinal Study for the Assessment of MRI Based Biomarkers as a Predictors of Clinical Endpoints in Pediatric Onset 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.
- Medical subject headings
- Hepatitis, Autoimmune, Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with autoimmune liver disease | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of intrahepatic bile duct irregularities between V0 (baseline visit) and V1 (visit after12 months) or V2 (visit after 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).
Measured: 24 months
Change of extra-hepatic duct irregularities between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 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).
Measured: 24 months
Mean shear stiffness of the liver
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).
Measured: 24 months
long-term clinical outcomes: survival with the native liver
Annual assessment of survival with the native liver (Yes=1, No=0) will be done within 10 years of follow-up.
Measured: 120 months
long-term clinical outcomes: hospital admissions for cholangitis
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.
Measured: 120 months
long-term clinical outcomes:endoscopic interventions for biliary strictures
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.
Measured: 120 months
long-term clinical outcomes:diagnosis of cholangiocarcinoma
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.
Measured: 120 months
long-term clinical outcomes: variceal bleeding
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.
Measured: 120 months
long-term clinical outcomes: ascites
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.
Measured: 120 months
Secondary outcomes (4)
Changes in liver/spleen volumes
Changes in liver/spleen volumes (mL) between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).
Measured: 24 months
Changes in T1rho, T1 and T2 mapping
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.
Measured: 24 months
Clinical endpoints of AILD: Pruritus
Annual assessment for Pruritus (on visual analogue scale of 0-10) will be done within 10 years of follow-up.
Measured: 120 months
Clinical endpoints of AILD
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.
Measured: 120 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2017
- Study start20 Feb 2017
- Primary completionFeb 2030 (estimated)
- Study completionFeb 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Liver DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alexander Miethke, MD, Principal investigator, Cincinnati Childrens Hospital Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03178630
- Sponsor's protocol ID
- CIN002 MRI biomarkers in AILD
- Status last verified
- Aug 2026
- First submitted
- 30 Mar 2017
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Farr R, Rojas CC, Alquraish M, Hommel K, Sahay R, Weymann A, Saarela K, Kulkarni S, Ayers M, Squires J, Taylor A, Kalkwarf HJ, Miethke A. Longitudinal, Multicenter Study of Clinical Factors Impacting Health-Related Quality of Life in Paediatric Autoimmune Liver Disease. Liver Int. 2026 Apr;46(4):e70581. doi: 10.1111/liv.70581.
PubMed 41804276