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Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Completed
Conditions
Cystic Fibrosis Liver Disease
Registration Number
NCT03001388
Lead Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
141
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

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTMYear 2

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

Secondary Outcome Measures
NameTimeMethod
Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertensionBaseline, 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 grayscale ultrasound gradesBaseline, Year 1 and Year 2

Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)

Liver steatosis obtained via transient elastographyBaseline, Year 1, Year 2

Controlled Attenuation Parameter (CAP)" quantifies liver steatosis and is measured in dB

Trial Locations

Locations (8)

Texas Children's Hospital

🇺🇸

Houston, Texas, United States

Children's Hospital Colorado

🇺🇸

Aurora, Colorado, United States

Ann & Robert H. Lurie Children's

🇺🇸

Chicago, Illinois, United States

Seattle Children's Hospital

🇺🇸

Seattle, Washington, United States

Cincinnati Children's Hospital Medical

🇺🇸

Cincinnati, Ohio, United States

Riley Hospital for Children

🇺🇸

Indianapolis, Indiana, United States

The Hospital for Sick Children

🇨🇦

Toronto, Ontario, Canada

Children's Healthcare of Atlanta

🇺🇸

Atlanta, Georgia, United States

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