Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Overview

To determine if transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the prediction of progression to a nodular pattern on US. To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF) To prospectively assess whether TE data are associated with conventional laboratory markers of hepatic fibrosis To determine the variability of TE measurements taken at different sites in the same patient

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: May 9, 2023

Detailed Description

A noninvasive assessment of hepatic fibrosis is desperately needed to advance the care of children with CF significant liver disease and to provide for measurements during clinical trials. That global assessment might serve as both a predictor/descriptor of disease course but also as a critical biomarker for clinical research. FibroScan® measurement of liver stiffness has great potential to fill this void. The underlying hypothesis of this proposal is that elastography in addition to US can improved the prediction of the development of a nodular liver on US and development of portal hypertension over time in children and young adults with CF.

Interventions

  • Procedure: Transient Elastography (TE)
    • Participants enrolled in Cystic Fibrosis Liver Disease Network (CFLD NET)Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers)

Clinical Trial Outcome Measures

Primary Measures

  • Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
    • Time Frame: Baseline
    • Liver stiffness” quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is <30% and success rate is >60%, meaning 10 valid measurements are obtained within 16 attempts).
  • Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
    • Time Frame: Year 1
    • Liver stiffness” quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is <30% and success rate is >60%, meaning 10 valid measurements are obtained within 16 attempts).
  • Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
    • Time Frame: Year 2
    • Liver stiffness” quantifies liver fibrosis and is measured in kPa (median of 10 subsequent

Secondary Measures

  • Liver steatosis obtained via transient elastography
    • Time Frame: Baseline, Year 1, Year 2
    • Controlled Attenuation Parameter (CAP)” quantifies liver steatosis and is measured in dB
  • Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades
    • Time Frame: Baseline, Year 1 and Year 2
    • Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)
  • Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension
    • Time Frame: Baseline, Year 1 and Year 2
    • Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)

Participating in This Clinical Trial

Inclusion Criteria

  • Participants enrolled in CFLD NET PUSH study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers) – Entry criteria for that study were: – CF as determined by sweat chloride >60 meq/l – Pancreatic insufficiency – Age 3-12 years old at entry – For entry into the longitudinal follow up subjects were in one of two groups – A screening US pattern of nodular liver (CIR), heterogeneous increased echogenicity (HTG) or homogeneous increased echogenicity (HMG) – A screening US pattern of normal (NL) matched to a HTG subject (2 NL:1HTG) by age, center and pseudomonas status Exclusion Criteria:

  • Exited from the PUSH Study – Unable / unwilling to sign consent

Gender Eligibility: All

Minimum Age: 6 Years

Maximum Age: 26 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

  • Lead Sponsor
    • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Collaborator
    • Cystic Fibrosis Foundation
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Ed Doo, MD, Study Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
    • Averell Sherker, MD, Study Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

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