Clinical trial NCT03198104: Assessing Kids for Liver Inflammation and Fibrosis Using Non-invasive MRI
Official title: Assessing Kids for Liver Inflammation and Fibrosis Using Non-invasive MRI
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Non-alcoholic Steatohepatitis
Liver Diseases
Liver Fibroses
Autoimmune Hepatitis
Liver Cirrhoses
Keywords: Multiparametric MR Imaging
Paediatric
Lead sponsor: Perspectum
Sponsor class: INDUSTRY
Collaborators: name: Children's Memorial Health Institute, Poland
First posted: 2017-06-23
Enrollment: 35
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants aged between 6-18 years of age with liver disease who are scheduled to receive a liver biopsy as part of their usual care and age-matched healthy volunteers aged 6-18 years of age. Participants between 1-10 years of age who are scheduled to receive an MRI scan under anesthesia as part of their standard care will also be recruited.

Brief summary: This is a prospective observational study which will recruit up to 100 paediatric participants over a period of 30 months to determine whether MRI is as accurate at detecting, distinguishing, and monitoring liver disease as current standard of care techniques such as liver biopsy and fibroscan.

Detailed description: The most serious pandemic facing Europe is fatty liver disease and non-alcoholic steatohepatitis (NASH). The main causal factor is obesity: according to recent statistics 11.8m-16.3m European children are overweight, of which 2.9m - 4.4m are obese. Unfortunately, current techniques for the diagnosis and monitoring of liver disease in children are poor. Available methods are either insensitive, such as blood tests, or invasive, i.e. liver biopsy, which is risky, painful, and costly. Although it is currently considered to be the gold standard for tissue assessment, liver biopsy has severe limitations, one of which is that it samples only a tiny fraction of the liver, and so it cannot give a representative diagnosis when liver disease is regional. Early determination of liver diseases in children, could have a significant impact on changing the course of illness and on the proper treatment management.

Perspectum Diagnostics have demonstrated that multiparametric MR imaging can, for the first time, allow accurate, non-invasive detection of liver fibrosis/inflammation in adults, and predict clinical outcomes (1,2). However, liver disease in children differs from adults in aetiology, natural history and pathological findings (3,4).

This study will investigate whether non-invasive multiparametric MR imaging of the liver can accurately and reproducibly detect, distinguish and track progression of different forms of liver disease in the paediatric population. To achieve this, children with liver disease who are scheduled to undergo liver biopsy and blood tests as part of their standard care will be recruited. Non-invasive, pain-free MRI scanning will be used to measure fibrosis, iron content and fat content of liver tissue and compare these results to the findings of the liver biopsy that is being performed as standard care. Children who are scheduled to receive repeated liver biopsies to monitor their response to the treatment of autoimmune hepatitis as part of their usual care will also be included. Repeated MRI scans will be performed and compared with the results of biopsy findings.

Eligibility criteria: Inclusion Criteria:

Group 1 (Liver disease):

* Male or female between 6 - 18 years of age
* Male or female aged between 1-10 years of age and scheduled to receive an MRI under anesthesia as part of their standard care.
* Scheduled to receive an ultra-sound guided liver biopsy as part of their usual care
* Participant and guardian are willing and able to give assent and consent for participation in the study

Group 2 (AIH):

* Male or female between 6 - 18 years of age
* Male or female aged between 1-10 years of age and scheduled to receive an MRI under anesthesia as part of their standard care.
* Diagnosed with autoimmune hepatitis and due to begin liver-biopsy monitored treatment as part of their usual care
* Participant and guardian are willing and able to give assent and consent for participation in the study

Group 3 (Healthy controls):

* Male or female between 6 - 18 years of age (age-matched with Group 1)
* Lean, defined as within interquartile range for age and sex-matched childhood BMI growth charts
* Healthy, with no active medical condition requiring treatment, including diabetes, hypertension, psoriasis and any chronic gastrointestinal disorder
* Participant and guardian are willing and able to give assent and consent for participation in the study

Exclusion Criteria:

* The participant may not enter the study if they have any contraindication to magnetic resonance imaging (inc pregnancy, extensive tattoos, pacemaker, shrapnel injury, severe claustrophobia).
* Any other cause, including a significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study

Primary outcomes:
measure: Multiparametric non-invasive MRI of liver tissue in detecting and distinguishing different forms of paediatric liver disease.
timeFrame: 30 months
Secondary outcomes:
measure: Determining use of MRI in measuring changes in liver disease within patient.
timeFrame: 30 months
Other outcomes:
Locations:
city: Warsaw
country: Poland
facility: Children's Memorial Health Institute Poland
Study officials:
affiliation: Perspectum Diagnostics
name: Rajashi Banerjee, MD, PhD
role: STUDY_DIRECTOR
affiliation: Children's Memorial Health Institute, Poland
name: Piotr Socha, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Janowski K, Shumbayawonda E, Dennis A, Kelly M, Bachtiar V, DeBrota D, Langford C, Thomaides-Brears H, Pronicki M, Grajkowska W, Wozniak M, Pawliszak P, Chelstowska S, Jurkiewicz E, Banerjee R, Socha P. Multiparametric MRI as a Noninvasive Monitoring Tool for Children With Autoimmune Hepatitis. J Pediatr Gastroenterol Nutr. 2021 Jan 1;72(1):108-114. doi: 10.1097/MPG.0000000000002930.