Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH)
This study is currently recruiting participants.
Verified April 2012 by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier:
NCT01144507
First received: June 14, 2010
Last updated: April 23, 2012
Last verified: April 2012
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Cystic Fibrosis Pancreatic Insufficiency |
Procedure: Abdominal Ultrasound Other: Sample collection procedures |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) |
Resource links provided by NLM:
Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
Primary Outcome Measures:
- Development of cirrhosis, as defined by imaging criteria [ Time Frame: Five years ] [ Designated as safety issue: No ]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 five year period.
Secondary Outcome Measures:
- Effects on associated pulmonary and nutritional issues [ Time Frame: 5 years ] [ Designated as safety issue: No ]
- 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)
Biospecimen Retention: Samples With DNA
During this study, blood and urine specimens will be obtained, de-identified and shipped to and stored at the NIDDK repositories for use in future CFLD ancillary studies. This "biobanking" is a critical aspect of this longitudinal study to facilitate the creation of a resource of DNA and other specimens from a meaningful number of patients with CFLD. In addition, obtaining and storing DNA or EBV-transformed leukocytes (from which DNA can be extracted) will allow future studies to investigate genetic causes and influences (modifier genes) in CFLD.
| Estimated Enrollment: | 800 |
| Study Start Date: | January 2009 |
| Estimated Study Completion Date: | March 2014 |
| Estimated Primary Completion Date: | March 2014 (Final data collection date for primary outcome measure) |
| Groups/Cohorts | Assigned Interventions |
|---|---|
|
Group A
Approximately 110 subjects with a heterogeneous echo pattern of the liver on abdominal ultrasound (HTG US).
|
Procedure: Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
Other Name: Doppler Ultrasound
Other: Sample collection procedures
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
Other Name: Doppler Ultrasound
|
|
Group B
Approximately 450 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.
|
Procedure: Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
Other Name: Doppler Ultrasound
Other: Sample collection procedures
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
Other Name: Doppler Ultrasound
|
|
Group C
An estimated 30 subjects with other findings, including cirrhosis or a diffusely homogeneous echogenic pattern on abdominal ultrasound (OTHER). These subjects will not be followed beyond their initial visit.
|
Procedure: Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
Other Name: Doppler Ultrasound
Other: Sample collection procedures
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
Other Name: Doppler Ultrasound
|
|
Group D
Subjects with cirrhotic and homogeneous pattern at screening ultrasound will be followed in the study.
|
Procedure: Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
Other Name: Doppler Ultrasound
Other: Sample collection procedures
Samples of urine, serum, plasma, and blood for DNA from children and blood for DNA from parents will be requested from participating subjects
Other Name: Doppler Ultrasound
|
Detailed Description:
For subjects in longitudinal follow up, this study will:
- Collect detailed clinical and demographic information about each subject at enrollment and during follow up,
- Obtain and store imaging data from the subject at entry and during follow up,
- Obtain and store serum, plasma and urine samples from the subject at entry (after matching) and during follow up,
- Obtain and store DNA from the subject,
- Obtain and store DNA from the biological parents,
- Obtain and store quality of life data from the subject and parents at enrollment and during follow up
Eligibility| Ages Eligible for Study: | 3 Years to 12 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
The study population will consist of males and females 3 through 12 years of age with Cystic Fibrosis and pancreatic insufficiency who are enrolled in the CFF or Toronto CF registry studies. All racial and ethnic groups will be included.
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 mg/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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01144507
Locations
| United States, Colorado | |
| Children's Hospital of Colorado | Recruiting |
| Aurora, Colorado, United States, 80045 | |
| Contact: Sheryl Faut 720-777-4691 sheryl.faut@childrenscolorado.org.org | |
| Principal Investigator: Michael Narkewicz, MD | |
| Sub-Investigator: Ron Sokol, MD | |
| United States, Georgia | |
| Emory University School of Medicine | Recruiting |
| Atlanta, Georgia, United States, 30322 | |
| Contact: Eric Hunter 404-727-9960 eahunte@emory.edu | |
| Contact: Diana Diaz 404-712-8586 diana.diaz@emory.edu | |
| Principal Investigator: Rene Romero, MD | |
| United States, Indiana | |
| Riley Hospital for Children | Recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact: Beth Byam, RN 317-944-3774 ebyam@iupui.edu | |
| Contact: Ann Klipsch, RN 317-944-9605 aeye@iupui.edu | |
| Principal Investigator: Jean Molleston, MD | |
| United States, Maryland | |
| John Hopkins School of Medicine | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Karen Callahan, RN, MS, CCRP 443-287-8983 kcallah1@jhmi.edu | |
| Contact: Carolyn Chapman, BA, RN 410-955-9782 cchapma7@jhmi.edu | |
| Principal Investigator: Shruti Paranjape, MD | |
| Sub-Investigator: Wilkrom Karnsakul, MD | |
| Sub-Investigator: Kathy Schwarz, MD | |
| United States, Minnesota | |
| University of Minneapolis Medical Center | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| Contact: Denise Stacklie 612-626-9491 stack046@umn.edu | |
| Principal Investigator: Sarah Jane Schwarzenberg, MD | |
| United States, Missouri | |
| Washington University School of Medicine | Recruiting |
| St. Louis, Missouri, United States, 63110 | |
| Contact: Kathleen Harris 314-747-5708 harris_k@kids.wustl.edu | |
| Contact: Stacy Postma 314-747-5931 postma_s@kids.wustl.edu | |
| Principal Investigator: Alexander Weymann, MD | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45229 | |
| Contact: Jan Dietz 513-636-7266 jan.dietz@cchmc.org | |
| Principal Investigator: Joseph Palermo, MD | |
| United States, Texas | |
| Texas Children's Hospital | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Zoe Apted, BA 832-824-3848 apted@bcm.edu | |
| Contact: Alejandro (Alex) De La Torre, BBA 832-824-3813 atorre@bcm.edu | |
| Principal Investigator: Daniel MD Leung, MD | |
| Canada, Ontario | |
| Hospital for Sick Children | Recruiting |
| Toronto, Ontario, Canada, M5G 1X8 | |
| Contact: Julianne Ling 416-813-5673 ulie.ling@sickkids.ca | |
| Principal Investigator: Simon Ling, MD | |
| Sub-Investigator: Peter Durie, MD | |
Sponsors and Collaborators
Cystic Fibrosis Foundation
Investigators
| Study Chair: | Michael Narkewicz, MD | Children's Hospital Colorado |
| 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:
No publications provided
| Responsible Party: | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| ClinicalTrials.gov Identifier: | NCT01144507 History of Changes |
| Other Study ID Numbers: | CFLD PUSH |
| Study First Received: | June 14, 2010 |
| Last Updated: | April 23, 2012 |
| Health Authority: | United States: Federal Government |
Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
|
Cystic Fibrosis Pancreatic insufficiency |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Liver Cirrhosis Exocrine Pancreatic Insufficiency Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes Liver Diseases |
ClinicalTrials.gov processed this record on May 22, 2013