Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Cystic Fibrosis Liver Disease Clinical Trial

Official title:

Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03001388
• Other study ID #: ELASTIC-CF
• Secondary ID: U01DK062456
• Status: Completed
• Start date: March 8, 2017
• Est. completion date: May 9, 2023

Study information

• Verified date: November 2023
• Source: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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 information / eligibility

• Status: Completed
• Enrollment: 141
• Est. completion date: May 9, 2023
• Est. primary completion date: May 9, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 6 Years to 26 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis Liver Disease
• Fibrosis
• Liver Diseases

Intervention

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)

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Ann & Robert H. Lurie Children's	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical	Cincinnati	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Cystic Fibrosis Foundation

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Invalid measurements and success rate on liver stiffness and liver steatosis measurements		Baseline, Year 1 and Year 2
Other	Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination	To reflect the dispersion of stiffness and steatosis	Baseline, Year 1 and Year 2
Other	IQR/median ratio of all valid measurements within the examination	This quantity should remain as low as possible to ensure reliable results (goal < 30%)	Baseline, Year 1 and Year 2
Primary	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
Primary	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
Primary	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	Liver steatosis obtained via transient elastography	Controlled Attenuation Parameter (CAP)" quantifies liver steatosis and is measured in dB	Baseline, Year 1, Year 2
Secondary	Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades	Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)	Baseline, Year 1 and Year 2
Secondary	Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension	Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)	Baseline, Year 1 and Year 2

External Links

•   ChiLDReN Website