MRI to Characterize and Predict CF Liver Disease in PUSH Cohort

• ClinicalTrials.gov Identifier: NCT02979340
• Recruitment Status: Completed
• First Posted: December 1, 2016
• Last Update Posted: January 28, 2020
• Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Collaborator: Cystic Fibrosis Foundation
• Information provided by (Responsible Party): National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

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
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: December 2016
• Actual Primary Completion Date: December 31, 2019
• Actual Study Completion Date: December 31, 2019

Groups and Cohorts

Intervention Details:

• Procedure: MRI
  non-invasive assessment of hepatic fibrosis and steatosis

Outcome Measures

Primary Outcome Measures :

1. Change over Time in Liver stiffness and Lipid content over time using MRE [ Time Frame: 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

• Ages Eligible for Study: 6 Years to 26 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic Fibrosis and pancreatic insufficiency and enrolled in the CFLD PUSH longitudinal study.

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

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, Indiana

Riley Hospital for Children

Indianapolis, Indiana, United States, 46202

United States, Maryland

Johns Hopkins School of Medicine

Baltimore, Maryland, United States, 21217

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

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)

More Information

Additional Information:

MRE is a study in the ChiLDReN Network 

• Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• ClinicalTrials.gov Identifier: NCT02979340
• Other Study ID Numbers: CFLD-MRE; U01DK062456 ( U.S. NIH Grant/Contract )
• First Posted: December 1, 2016
• Last Update Posted: January 28, 2020
• Last Verified: July 2019

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

Additional relevant MeSH terms:

Cystic Fibrosis; Liver Diseases; Exocrine Pancreatic Insufficiency; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases