MRI to Characterize and Predict CF Liver Disease in PUSH Cohort

Sponsor

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

Overall Status

Completed

CT.gov ID

NCT02979340

Collaborator

Cystic Fibrosis Foundation (Other)

115

Enrollment

Locations

Actual Duration (Months)

16.4

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis.

Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound.

Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing.

Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis Liver Disease Pancreatic Insufficiency	Procedure: MRI

Detailed Description

This proposal will be a cross sectional study of the current well-characterized PUSH cohort. Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but Investigators will allow a larger window due to the slow progression of fibrosis).

Blinded radiology review of imaging sequences will be performed by a local study radiologist with central review performed by the Imaging Core. Analysis by the Imaging Core will include calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content for all MRI studies.

Study Design

Study Type:

Observational

Actual Enrollment :

115 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Analysis of Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) Cohort Using Advanced MRI to Characterize and Predict CF Liver Disease

Actual Study Start Date :

Dec 1, 2016

Actual Primary Completion Date :

Dec 31, 2019

Actual Study Completion Date :

Dec 31, 2019

Outcome Measures

Primary Outcome Measures

Change over Time in Liver stiffness and Lipid content over time using MRE [BSL, Year 1, Year 2]
Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 26 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Currently enrolled in longitudinal follow up in PUSH study at a site with MR elastography.

Exclusion Criteria:

Age under 6 years (all current patients at time of opening of this study will be greater than 6 years of age).
Internal appliance or hardware that is not compatible with MR.
Inability to obtain MRI within 6 months of US.
Inability to cooperate with MRI.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Colorado	Aurora	Colorado	United States	80045
2	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30322
3	Riley Hospital for Children	Indianapolis	Indiana	United States	46202
4	Johns Hopkins School of Medicine	Baltimore	Maryland	United States	21217
5	Cincinnati Children's Hospital Medical	Cincinnati	Ohio	United States	45229
6	Texas Children's Hospital	Houston	Texas	United States	77030
7	Seattle Children's Hospital	Seattle	Washington	United States	98105

Sponsors and Collaborators

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

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)