Overview

This trial is active, not recruiting.

Conditions cystic fibrosis, pancreatic insufficiency
Treatments abdominal ultrasound, sample collection procedures
Sponsor National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator Cystic Fibrosis Foundation Therapeutics
Start date January 2009
End date November 2018
Trial size 800 participants
Trial identifier NCT01144507, CFLD PUSH

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
Approximately 60 subjects with a heterogeneous echo pattern of the liver on abdominal ultrasound (HTG US).
abdominal ultrasound Doppler Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
sample collection procedures Doppler Ultrasound
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
Approximately 680 subjects with a normal echo pattern on abdominal ultrasound (NL US). Of these subjects, approximately 110 will be matched 1:1 with Group A participants and followed for the duration of the study. The remaining unmatched subjects will not be followed beyond their initial visit.
abdominal ultrasound Doppler Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
sample collection procedures Doppler Ultrasound
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
An estimated 30 subjects with cirrhosis pattern on abdominal ultrasound. These subjects will be followed in the study.
abdominal ultrasound Doppler Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
sample collection procedures Doppler Ultrasound
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
An estimated 30 subjects with diffusely homogeneous echogenic pattern at screening ultrasound will be followed in the study.
abdominal ultrasound Doppler Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
sample collection procedures Doppler Ultrasound
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects

Primary Outcomes

Measure
Development of cirrhosis, as defined by imaging criteria
time frame: Five years

Secondary Outcomes

Measure
Effects on associated pulmonary and nutritional issues
time frame: 5 years

Eligibility Criteria

Male or female participants from 3 years up to 12 years old.

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

Additional Information

Official title Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH)
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
Trial information was received from ClinicalTrials.gov and was last updated in December 2015.
Information provided to ClinicalTrials.gov by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).