Cystic Fibrosis Clinical Trial
— PUSHOfficial title:
Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH)
Verified date | October 2023 |
Source | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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
Status | Completed |
Enrollment | 774 |
Est. completion date | June 14, 2023 |
Est. primary completion date | February 26, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Years to 12 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Canada | Hospital for Sick Children | Toronto | Ontario |
United States | Emory University School of Medicine | Atlanta | Georgia |
United States | Children's Hospital of Colorado | Aurora | Colorado |
United States | Johns Hopkins School of Medicine | Baltimore | Maryland |
United States | Ann & Robert H. Lurie Children's Hospital of | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Texas Children's Hospital | Houston | Texas |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | University of Minneapolis Medical Center | Minneapolis | Minnesota |
United States | Washington University School of Medicine | Saint Louis | Missouri |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Cystic Fibrosis Foundation |
United States, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Development of cirrhosis, as defined by imaging criteria | 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. | Nine years | |
Secondary | Effects on associated pulmonary and nutritional issues | Health related quality of life
Growth (length, weight and BMI Z-score, anthropometrics) AST,ALT,GGTP FEV1, FVC Sputum Culture (Pseudomonas, Burkholderia cepacia) Use of IV antibiotics Hospitalization for treatment of pulmonary exacerbation CBC (WBC, Hbg, ANC, platelet count) Fat soluble vitamin levels (Vitamin E, 25 hydroxy vitamin D, Vitamin A) |
9years |
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