Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis
NCT ID: NCT01144507
Last Updated: 2025-04-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
774 participants
OBSERVATIONAL
2010-01-12
2023-06-14
Brief Summary
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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
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Detailed Description
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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
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Group A
Approximately 60 subjects with a heterogeneous echo pattern of the liver on abdominal ultrasound (HTG US).
Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
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
Group B
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
To establish eligibility and/or markers regarding echo pattern types.
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
Group C
An estimated 30 subjects with cirrhosis pattern on abdominal ultrasound. These subjects will be followed in the study.
Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
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
Group D
An estimated 30 subjects with diffusely homogeneous echogenic pattern at screening ultrasound will be followed in the study.
Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
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
Interventions
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Abdominal Ultrasound
To establish eligibility and/or markers regarding echo pattern types.
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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
3 Years
12 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Responsible Party
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Principal Investigators
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Michael Narkewicz, MD
Role: STUDY_CHAIR
Children's Hospital Colorado
Ed Doo, MD
Role: STUDY_DIRECTOR
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Averell Sherker, MD
Role: STUDY_DIRECTOR
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Locations
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Children's Hospital of Colorado
Aurora, Colorado, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
University of Minneapolis Medical Center
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hospital for Sick Children
Toronto, Ontario, Canada
Countries
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References
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Siegel MJ, Freeman AJ, Ye W, Palermo JJ, Molleston JP, Paranjape SM, Stoll J, Leung DH, Masand P, Karmazyn B, Harned R, Ling SC, Navarro OM, Karnsakul W, Alazraki A, Schwarzenberg SJ, Seidel FG, Towbin A, Alonso EM, Nicholas JL, Murray KF, Otto RK, Sherker AH, Magee JC, Narkewicz MR; CFLD Network. Heterogeneous Liver on Research Ultrasound Identifies Children with Cystic Fibrosis at High Risk of Advanced Liver Disease: Interim Results of a Prospective Observational Case-Controlled Study. J Pediatr. 2020 Apr;219:62-69.e4. doi: 10.1016/j.jpeds.2019.12.033. Epub 2020 Feb 12.
Siegel MJ, Leung DH, Molleston JP, Ye W, Paranjape SM, Freeman AJ, Palermo JJ, Stoll J, Masand P, Karmazyn B, Harned R, Ling SC, Navarro OM, Karnsakul W, Alazraki A, Schwarzenberg SJ, Towbin AJ, Alonso EM, Nicholas JL, Green N, Otto RK, Magee JC, Narkewicz MR. Heterogeneous liver on research ultrasound identifies children with cystic fibrosis at high risk of advanced liver disease. J Cyst Fibros. 2023 Jul;22(4):745-755. doi: 10.1016/j.jcf.2023.03.019. Epub 2023 Apr 7.
Leung DH, Ye W, Molleston JP, Weymann A, Ling S, Paranjape SM, Romero R, Schwarzenberg SJ, Palermo J, Alonso EM, Murray KF, Marshall BC, Sherker AH, Siegel MJ, Krishnamurthy R, Harned R, Karmazyn B, Magee JC, Narkewicz MR; Cystic Fibrosis Liver Disease Network (CFLD NET). Baseline Ultrasound and Clinical Correlates in Children with Cystic Fibrosis. J Pediatr. 2015 Oct;167(4):862-868.e2. doi: 10.1016/j.jpeds.2015.06.062. Epub 2015 Aug 5.
Related Links
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PUSH is a study in the ChiLDREN Network
Other Identifiers
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CFLD PUSH
Identifier Type: -
Identifier Source: org_study_id
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