Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)11 sites in 2 countries774 target enrollmentJanuary 12, 2010

ConditionsCystic Fibrosis Pancreatic Insufficiency

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Cystic Fibrosis
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Enrollment: 774
Locations: 11
Primary Endpoint: Development of cirrhosis, as defined by imaging criteria
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The specific aims for this study are:

To determine if sonographic findings predict the risk of progression of liver disease to cirrhosis by comparing cystic fibrosis subjects with heterogeneous echogenicity pattern on ultrasound to those with normal echogenicity pattern on ultrasound
To develop a database and biorepository of serum, plasma, urine and DNA to aid the investigations in ascertaining the mechanisms, consequences, genetic risk factors and biomarkers for the development of cirrhosis
To determine if there are differences in health related quality of life, pulmonary or nutritional status in children with cystic fibrosis who have a heterogeneous echo pattern on ultrasound compared to those who have a normal echo pattern on ultrasound
To determine if Doppler velocity measurements of hepatic and splenic vessels predict an increased risk for the development of cirrhosis.
To determine if cirrhosis on ultrasound progresses to portal hypertension during the study period
To determine if homogeneous liver progresses to either cirrhosis or heterogeneous liver.
To determine the frequency of complications of portal hypertension during follow up in those identified with cirrhosis by year 6 of the study

Detailed Description

For subjects in longitudinal follow up, this study will: 1. Collect detailed clinical and demographic information about each subject at enrollment and during follow up, 2. Obtain and store imaging data from the subject at entry and during follow up, 3. Obtain and store serum, plasma and urine samples from the subject at entry (after matching) and during follow up, 4. Obtain and store DNA from the subject, 5. Obtain and store DNA from the biological parents, 6. Obtain and store quality of life data from the subject and parents at enrollment and during follow up

Registry

clinicaltrials.gov

Start Date

January 12, 2010

End Date

June 14, 2023

Last Updated

last year

Study Type

Observational

Sex

All

Investigators

Sponsor

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

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic Fibrosis and pancreatic insufficiency
•Enrolled in the CFF registry study or Toronto CF Registry
•CF defined as sweat chloride of \>60 mEq/L on one occasion (using the value in the CF registry) or two disease-causing mutations of CFTR with evidence of end organ involvement.
•Pancreatic insufficient defined as one of the following:
•CFTR Mutation associated with pancreatic insufficiency
•Fecal elastase \<100 mcg/gm (at any time)
•72 hour fecal fat with coefficient of fat absorption \<85% (at any time)

Exclusion Criteria

•Known cirrhosis
•Presence of Burkholderia cepacia
•Short bowel syndrome defined as not on full enteral feeds by 3 months of age
•Presence of other serious disease precluding participation in this study (This would include patients with known other causes of chronic liver disease)
•If in the opinion of the Investigator the study is not in the best interest of the patient
•Inability to comply with the longitudinal follow-up described below
•Failure of a family to sign the informed consent document or the HIPAA medical record release form

Outcomes

Primary Outcomes

Development of cirrhosis, as defined by imaging criteria

Time Frame: Nine years

The primary objective of this prospective longitudinal study is to determine the utility of abdominal ultrasound (US) at enrollment to predict the development of cirrhosis in subjects with cystic fibrosis (CF) within a nine year period.