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Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03001388
Recruitment Status : Enrolling by invitation
First Posted : December 23, 2016
Last Update Posted : March 30, 2018
Sponsor:
Collaborator:
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Description
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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


Condition or disease Intervention/treatment
Cystic Fibrosis Liver Disease Procedure: Transient Elastography (TE)

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 251 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Longitudinal Assessment of Transient Elastography in Cystic Fibrosis
Actual Study Start Date : April 1, 2017
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • 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)

Outcome Measures
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Primary Outcome Measures :
  1. 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).

  2. 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).

  3. 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 Outcome Measures :
  1. 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

  2. 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)

  3. 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)


Other Outcome Measures:
  1. Invalid measurements and success rate on liver stiffness and liver steatosis measurements [ Time Frame: Baseline, Year 1 and Year 2 ]
  2. Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination [ Time Frame: Baseline, Year 1 and Year 2 ]
    To reflect the dispersion of stiffness and steatosis

  3. IQR/median ratio of all valid measurements within the examination [ Time Frame: Baseline, Year 1 and Year 2 ]
    This quantity should remain as low as possible to ensure reliable results (goal < 30%)


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 26 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
Criteria

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
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03001388


Locations
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
United States, Illinois
Ann & Robert H. Lurie Children's
Chicago, Illinois, United States, 60611
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Ohio
Cincinnati Children's Hospital Medical
Cincinnati, Ohio, United States, 45229
United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Cystic Fibrosis Foundation Therapeutics
Investigators
Study Director: 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)
More Information
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Additional Information:
ChiLDReN Website  This link exits the ClinicalTrials.gov site

Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier: NCT03001388     History of Changes
Other Study ID Numbers: ELASTIC-CF
U01DK062456 ( U.S. NIH Grant/Contract )
First Posted: December 23, 2016    Key Record Dates
Last Update Posted: March 30, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
Cystic Fibrosis Liver Disease
Pancreatic insufficiency
Cystic Fibrosis
Transient Elastography

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Liver Diseases
Pathologic Processes
Pancreatic Diseases
 Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases