Clinical trial NCT03001388: Longitudinal Assessment of Transient Elastography in Cystic Fibrosis
Official title: Longitudinal Assessment of Transient Elastography in Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis Liver Disease
Keywords: Cystic Fibrosis Liver Disease
Pancreatic insufficiency
Cystic Fibrosis
Transient Elastography
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor class: NIH
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2016-12-23
Enrollment: 141
Sex: ALL
Minimum age (years): 6
Maximum age (years): 26
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

Eligibility 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

Primary outcomes:
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
timeFrame: Baseline
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
timeFrame: Year 1
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
timeFrame: Year 2
Secondary outcomes:
measure: Liver steatosis obtained via transient elastography
timeFrame: Baseline, Year 1, Year 2
measure: Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades
timeFrame: Baseline, Year 1 and Year 2
measure: Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension
timeFrame: Baseline, Year 1 and Year 2
Other outcomes:
measure: Invalid measurements and success rate on liver stiffness and liver steatosis measurements
timeFrame: Baseline, Year 1 and Year 2
measure: Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination
timeFrame: Baseline, Year 1 and Year 2
measure: IQR/median ratio of all valid measurements within the examination
timeFrame: Baseline, Year 1 and Year 2
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical
state: Ohio
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: Ed Doo, MD
role: STUDY_DIRECTOR
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: Averell Sherker, MD
role: STUDY_DIRECTOR
References:
citation: 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.