Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis

Status:	Active, not recruiting
Conditions:	Gastrointestinal, Pulmonary
Therapuetic Areas:	Gastroenterology, Pulmonary / Respiratory Diseases
Healthy:	No
Age Range:	3 - 12
Updated:	4/17/2018
Start Date:	January 2009
End Date:	November 2021

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

The specific aims for this study are:

1. 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

2. 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

3. 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

4. To determine if Doppler velocity measurements of hepatic and splenic vessels predict an
increased risk for the development of cirrhosis.

5. To determine if cirrhosis on ultrasound progresses to portal hypertension during the
study period

6. To determine if homogeneous liver progresses to either cirrhosis or heterogeneous liver.

7. To determine the frequency of complications of portal hypertension during follow up in
those identified with cirrhosis by year 6 of the study

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

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

We found this trial at

sites

Children's Hospital Colorado

13123 E 16th Ave
Aurora, Colorado 80045

(720) 777-1234