Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

NCT03001388 | Completed DMC

• 2 other identifiers: ELASTIC-CFU01DK062456
• Study Type: observational
• Target: 141
• Locations: 2 countries
• Sites: 8

Brief Summary

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

Conditions

Cystic Fibrosis Liver Disease

Keywords

Cystic Fibrosis Liver Disease; Pancreatic insufficiency; Cystic Fibrosis; Transient Elastography

Outcome Measures

Primary Outcomes (3)

• Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM

  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).

  Baseline

• Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM

  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).

  Year 1

• Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM

  Liver stiffness" quantifies liver fibrosis and is measured in kPa (median of 10 subsequent

  Year 2

Secondary Outcomes (3)

• Liver steatosis obtained via transient elastography

  Baseline, Year 1, Year 2

• Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades

  Baseline, Year 1 and Year 2

• Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension

  Baseline, Year 1 and Year 2

Other Outcomes (3)

• Invalid measurements and success rate on liver stiffness and liver steatosis measurements

  Baseline, Year 1 and Year 2

• Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination

  Baseline, Year 1 and Year 2

• IQR/median ratio of all valid measurements within the examination

  Baseline, Year 1 and Year 2

Interventions

Transient Elastography (TE) PROCEDURE

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)

Eligibility Criteria

• Age: 6 Years - 26 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Participants enrolled in CFLD NET PUSH study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers) All racial and ethnic groups will be included.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (8)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Ann & Robert H. Lurie Children's

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Cincinnati Children's Hospital Medical

Cincinnati, Ohio, 45229, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (1)

• Ye W, Leung DH, Molleston JP, Ling SC, Murray KF, Nicholas JL, Huang S, Karmazyn BW, Harned RK, Masand P, Alazraki AL, Navarro OM, Otto RK, Palermo JJ, Towbin AJ, Alonso EM, Karnsakul WW, Jane Schwarzenberg S, Seidel GF, Siegel M, Magee JC, Narkewicz MR, Jay Freeman A. Association Between Transient Elastography and Controlled Attenuated Parameter and Liver Ultrasound in Children With Cystic Fibrosis. Hepatol Commun. 2021 May 13;5(8):1362-1372. doi: 10.1002/hep4.1719. eCollection 2021 Aug.

  PMID: 34430781 BACKGROUND

Related Links

• ChiLDReN Website

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Ed Doo, MD

  National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

  STUDY DIRECTOR

• Averell Sherker, MD

  National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2016
• First Posted: December 23, 2016
• Study Start: March 8, 2017
• Primary Completion: May 9, 2023
• Study Completion: May 9, 2023
• Last Updated: April 11, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (7)