Trial results for a study investigating pediatric liver fat quantification in MASH (NASH) were posted on ClinicalTrials.gov on 2026-01-27, with 30 participants enrolled. The study evaluated the clinical feasibility of an investigational ultrasound technique for quantifying liver fat by comparing ultrasound-derived biomarkers with MRI Proton Density Fat Fraction (MRI-PDFF) measurements.
Background
Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), is a severe form of non-alcoholic fatty liver disease (NAFLD) characterized by liver inflammation and damage. It can progress to fibrosis, cirrhosis, and liver cancer. Accurate and non-invasive methods for quantifying liver fat are crucial for diagnosis, monitoring disease progression, and evaluating treatment efficacy, particularly in pediatric populations where liver biopsy can be challenging. MRI-PDFF is considered a reliable non-invasive method for liver fat quantification, and the development of more accessible techniques like ultrasound could significantly impact patient care.
Trial design
This completed study, designated as Phase NA, enrolled 30 participants to investigate conditions including Non-Alcoholic Fatty Liver Disease and Non-Alcoholic Steatohepatitis. The primary objective was to evaluate the clinical feasibility of an investigational ultrasound technique for quantifying liver fat by comparing specific ultrasound-derived biomarkers with the liver fat percentage obtained from MRI Proton Density Fat Fraction (MRI-PDFF) measurements. All enrolled subjects underwent a series of medical imaging procedures of the abdomen using both ultrasound and MRI modalities.
Key results
The trial reported several key measurements and analyses related to ultrasound biomarkers for liver fat quantification:
- Hepatorenal Index (HRI) Ultrasound Biomarker: The mean HRI was 1.7 (Standard Deviation 0.7). Inter-operator variability for HRI showed a mean of -0.5 (Standard Deviation 0.6). The data acquisition failure rate for HRI was 0 participants.
- Acoustic Attenuation (dB/cm/MHz) Ultrasound Biomarker: The mean acoustic attenuation was 0.6 (Standard Deviation 0.1). Inter-operator variability for acoustic attenuation showed a mean of -0.01 (Standard Deviation 0.02). The data acquisition failure rate for acoustic attenuation was 0 participants.
- Tissue Stiffness (kPa) Ultrasound Biomarker: The mean tissue stiffness was 6.6 (Standard Deviation 1.6). Inter-operator variability for tissue stiffness showed a mean of 0.7 (Standard Deviation 1.2). The data acquisition failure rate for tissue stiffness was 2 participants.
Correlation analyses between ultrasound biomarkers and MRI-PDFF fat fraction included:
- For Hepatorenal Index (HRI), Pearson correlation coefficients were reported as 0.4459 and 0.5431. A correlation estimated from a mixed model was 0.4871.
- For Acoustic Attenuation, Pearson correlation coefficients were 0.1742 and 0.177. A correlation estimated from a mixed model was 0.1756.
What this means
The results from this pediatric study suggest that investigational ultrasound techniques show varying degrees of correlation with MRI-PDFF for quantifying liver fat in MASH/NAFLD. The Hepatorenal Index (HRI) demonstrated a moderate correlation with MRI-PDFF, with Pearson correlation coefficients ranging from 0.4459 to 0.5431. Acoustic attenuation showed a weaker correlation, with coefficients around 0.17. The low data acquisition failure rates for HRI and acoustic attenuation, and relatively low inter-operator variability, indicate the potential feasibility of these ultrasound biomarkers as non-invasive tools for assessing liver fat. These findings contribute to the ongoing effort to develop accessible and reliable methods for diagnosing and monitoring liver fat in pediatric patients with MASH.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for the study NCT04800094, titled "Pediatric Liver Fat Quantification (LFQ) Phase 2 Pilot Study", were posted on 2026-01-27 on clinicaltrials.gov.