VALidation of Imaging-based Liver Biomarkers in PEDiatric Patients

NCT07202910 | Recruiting

• 3 other identifiers: Dnr 634-18, 2018-09-03Dnr 2023-01951ALFGBG-1005247
• Study Type: observational
• Target: 550
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational (diagnostic validation) study is to validate imaging-based biomarkers for assessing liver involvement in pediatric patients with established or suspected liver disease. The projekt involves 3 larger studies, each with specific aims specified in detailed description below. The main study (study 1) compares imaging-based biomarkers-with liver biopsy to evaluate their correlation with histologically confirmed liver disease, including fibrosis, inflammation, and steatosis. The main question the overall project aims to answer is: • Can multiple novel imaging-based biomarkers, such as shear wave elastography (SWE), shear wave dispersion (SWD), and attenuation imaging (ATI), be used for detailed, non-invasive liver characterization in children, and if so, how should they be used?

Conditions

Hepatic Disorders; Intestinal Failure-associated Liver Disease; Cystic Fibrosis Liver Disease; MAFLD; Pediatric Liver Transplantation

Keywords

pediatric hepatic disease,; obesity and overweight; intestinal failiure associated liver disease; liver transplant disorder; Cystic Fibrosis Liver Disease; Liver disease or abnormalities; pediatric liver transplant; pediatric abdominal imaging; elastography; MAFLD

Outcome Measures

Primary Outcomes (6)

• Diagnostic accuracy of ultrasound-based liver biomarkers (SWE, SWD, ATI) (STUDY 1)

  Primary outcome study 1 To evaluate the sensitivity, specificity, and correlation of SWE (for fibrosis), SWD (for inflammation), and ATI (for steatosis) against histologically confirmed liver disease in pediatric patients. Using Area under the ROC curve (AUC), sensitivity, specificity, correlation coefficients

  At time of liver biopsy

• Longitudinal change in SWE/SWD/ATI biomarkers before and after treatment with CFTR modulators, (Study 2A - Children with Cystic Fibrosis)

  Evaluate the effect of CFTR modulator therapy on liver biomarkers. Metric: Change in SWE/SWD/ATI values over time

  baseline (pretreatment) up to 2 years

• Change in liver biomarkers measured by SWE and SWD during routine ultrasound surveillance post-liver transplantation. (Study 2b)

  SWE and SWD will be added to conventional ultrasound to assess liver stiffness and viscocity. These imaging biomarkers will be tracked longitudinally to evaluate their utility in improving post-transplant surveillance.

  Up to 1 year

• Detection of early liver damage using SWE, SWD, and ATI (Attenuation Imaging) in children with intestinal failure. (Study 2c)

  Non-invasive ultrasound biomarkers will be measured during yearly check-ups to identify early signs of liver fibrosis and steatosis in children at risk of IFALD

  up to 2 years

• Change in SWE/SWD/ATI biomarkers following obesity treatment (GLP-1 analogues or bariatric surgery) (Study2d)

  Assess whether imaging biomarkers reflect early liver response to obesity interventions. Metric: Change in SWE/SWD/ATI values over time

  through study completion, an average of 1 years

• Correlation between ultrasound biomarkers and MR biomarkers . (Study 3)

  SWE/SWD/ATI and MRE will be performed within a 2 week period and compared to assess the relationship between regional and global liver stiffness and steatosis measurements.

  periprocedurally

Secondary Outcomes (4)

• Association between imaging biomarkers and blood liver markers and demographic variabels (Study 1)

  periprocedurally

• Correlation between SWE/SWD values and clinical outcomes in livertransplats . (Study 2b)

  From enrollment to 1 year

• Correlation between SWE/SWD/ATI values and serological liver markers (Study 2c)

  from enrollment to 2 years

• Correlation between MRE and histological findings from liver biopsy. (study3)

  periprocedurally

Other Outcomes (8)

• Inter-operator variability in ultrasound measurements (STUDY 1)

  at ultrasound

• Longitudinal changes in imaging biomarkers in patients with repeated biopsies (Study 1)

  through study completion, an average of 5 years

• Comparison of imaging biomarkers between controls and patients (STUDY 1)

  At time of ultrasound

Study Arms (3)

Children with established or suspected liver disease

Study 1: All children with suspected or established liver disease referred for at liver biopsy at Queen Silvias Children Hospital during the inclusion period. Their ultrasound based biomarkers will be compared with histology and with ultrasound biomarkers of controls (children without liver disease or conditions affecting the liver, referred for a renal biopsy during the inclusion period)

Children who need surveillance of disease and treatment by longitudinal monitoring

Study 2: Several diseases/conditions are already monitored with conventional ultrasound on a regular basis in children. Examples include cystic fibrosis, liver transplants, severe obesity and failure-associated liver disease (IFALD).

Children with MR of the liver

This study (study 3) cohort will include around 50 children who are participating in study 1 and 2, and in whom MRI is clinically indicated as a supplement to ultrasound examination - for example, children with obesity for whom ultrasound may be insufficient or children in whom a more global characterization of the liver is desired.

Eligibility Criteria

• Age: Up to 25 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

All participants are children at Queen Silvias Children's hospital, meeting the eligible criteria Study 1-(main study): All children referred for a liver biopsy Study 2a: All children with cystic fibrosis Study 2b: All children liver transplanted Study 2c: All children monitored for intenstinal failiure Study 2d: All children treated with either bariatric surgery or novel appetite-regulating drugs (GLP1 analogues) Study 3: Children who are participating in study 1 and 2, in whom MRI is clinically indicated as a supplement to ultrasound examination - for example, children with obesity for whom ultrasound may be insufficient

You may qualify if:

• all patients scheduled for a clinically liver biopsy at the Queen Silvia Children's Hospital
• any form of suspected or confirmed liver disease
• age between 0-18 years
• consent by the child/parents to participate.
• For controls: children scheduled for a renal biopsy
• age between 0-18 years
• consent by the child/parents to participate.

You may not qualify if:

• technical limitations regarding the measurements
• inability to fully understand the informed consent documents.

Sponsors & Collaborators

• Sahlgrenska University Hospital: lead
• Queen Silvia Children's Hospital, Gothenburg, Sweden: collaborator

Study Sites (1)

Department of Radiology, Queen Silvias Children´s Hospital,Gothenburg Sweden

Gothenburg, Västra Götaland County, 41650, Sweden

RECRUITING

MeSH Terms

Conditions

Digestive System Diseases; Obesity; Overweight; Liver Diseases; Congenital Abnormalities

Condition Hierarchy (Ancestors)

Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Hanna Hebelka

  Institute of clinical sciences, Sahlgrenska Academy, Gothenburg University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hanna M Hebelka, Associate Prof.

CONTACT

+46705667867; hanna.hebelka@vgregion.se

Charlotte de Lange, Assoc.Prof.

CONTACT

+46735367199; charlotte.de.lange@vgregion.se

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: September 16, 2025
• First Posted: October 2, 2025
• Study Start: March 28, 2019
• Primary Completion (Estimated): March 1, 2030
• Study Completion (Estimated): December 1, 2032
• Last Updated: October 2, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1)