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Development of Biomarker for Development of Non-Alcoholic Steatohepatitis (NASH) in Children

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2009 by Medical College of Wisconsin.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT00677183
First received: May 9, 2008
Last updated: May 20, 2009
Last verified: May 2009
  Purpose

The purpose of this study is to document how often specific genotypes known to be associated with adult-onset NASH (Non-Alcoholic Steatohepatitis) occur in a pediatric cohort and investigate whether these genotypes are associated with increased susceptibility to NASH.


Condition
Non-Alcoholic Steatohepatitis

Study Type: Observational
Study Design: Observational Model: Family-Based
Time Perspective: Prospective
Official Title: Development of a Biomarker for Development of Non-Alcoholic Steatohepatitis (NASH) in Children

Resource links provided by NLM:


Further study details as provided by Medical College of Wisconsin:

Primary Outcome Measures:
  • We will compare known frequencies from the Hapmap and specifically compare the proportions for those with NASH in adults to see if there is a difference in the child incidence of NASH. [ Time Frame: three years ] [ Designated as safety issue: No ]
  • We will compare the proportions of those with NASH to those without NASH and those with fibrosis compared to those without fibrosis. [ Time Frame: Three years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

10mL of blood will be retained from each participant and their parents. 5mL of this blood will undergo DNA extraction and genotyping for several known NASH related genes. The other 5mL of blood will be saved as serum for future analysis.


Estimated Enrollment: 150
Study Start Date: May 2008
Estimated Study Completion Date: May 2011
Estimated Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Groups/Cohorts
SN###.#1
All children in this cohort will have biopsy-proven NASH.
SN###.#2
This cohort will be parents (mother and father when possible) of child subjects with biopsy-proven NASH.

Detailed Description:

NASH is a clinico-pathological entity characterized by the development of histological changes of inflammation and fibrosis in the liver that are nearly identical to those induced by excessive alcohol intake, but in the absence of alcohol abuse. Nonalcoholic steatohepatitis occurs commonly children with additional comorbidities such as obesity and diabetes mellitus. Paralleling the increasing prevalence of obesity and type 2 diabetes in the pediatric population, nonalcoholic fatty liver disease (NAFLD) and especially its more severe histological form NASH, is expected to become one of the most common causes of end-stage liver disease in both children and young adults.

Although no genome wide association studies have been conducted in association with NASH to date, individual candidate gene investigations have identified several genes associated with increase susceptibility to NASH in adults including the microsomal triglyceride transfer protein (MTP) which regulates the incorporation of triglycerides into apolipoprotein B and a key enzyme for the assembly and secretion of VLDL from hepatocytes, the manganese superoxide dismutase (MnSOD) gene which catalyzes the conversion of two molecules of superoxide anion, a highly unstable ROS, into hydrogen peroxide and oxygen more stable ROS, and lastly, phosphatidylethanolamine N-methyltransferase (PEMT) which is required for hepatic secretion of triacylglycerol in very low density lipoproteins (VLDL).

We propose the following aim:

Aim 1: To document the frequency of specific genotypes, previously identified to be associated with adult-onset NASH, in a purely pediatric cohort.

Aim 2: To investigate whether these genotypes are associated with increased susceptibility to NASH and increased occurrence of fibrosis in the cohort of pediatric subjects. Our hypothesis would be:

A significantly higher proportion of the polymorphisms would exist in those subjects with NASH compared to controls.

Aim 3: To investigate the presence of other polymorphisms or other biomarker that are indicative of pediatric NASH. Such that our secondary hypothesis would be:

Specific polymorphisms or biomarkers will be identified that will indicate a higher probability of NASH.

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

The child subjects will be recruited from the Hepatology Clinic at Children's Hospital of Wisconsin.

Criteria

Inclusion Criteria:

  • All subjects aged 2-18 with biopsy proven NAFLD and/or NASH undergoing a blood draw and willing to consent to this study will qualify for inclusion in this protocol.

Exclusion Criteria:

  • other causes of chronic liver disease or other chronic diseases, specifically autoimmune disorders, immunodeficiencies, or individuals with congenital/genetic disorders
  • chronic viral hepatitis, Wilson's disease, or alpha -1- antitrypsin deficiency
  • acute life threatening illness or conditions
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00677183

Contacts
Contact: Vincent F Biank, MD 414-266-3690 vbiank@mcw.edu
Contact: Grzegorz Telega, MD 414-266-3690 gtelega@mcw.edu

Locations
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Vincent F Biank, MD    414-266-3690    vbiank@mcw.edu   
Contact: Grzegorz Telega, MD    414-266-3690    gtelega@mcw.edu   
Sub-Investigator: Grzegorz Telega, MD         
Sub-Investigator: Subra Kugathasan, MD         
Sub-Investigator: Pippa Simpson, PhD         
Principal Investigator: Vincent F Biank, MD         
Sponsors and Collaborators
Medical College of Wisconsin
Investigators
Principal Investigator: Vincent F Biank, MD Medical College of Wisconsin
  More Information

Publications:
Responsible Party: Vincent Biank/Principal Investigator, Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00677183     History of Changes
Other Study ID Numbers: CHW 08/36, GC 618
Study First Received: May 9, 2008
Last Updated: May 20, 2009
Health Authority: United States: Institutional Review Board

Keywords provided by Medical College of Wisconsin:
NASH
Biomarker

Additional relevant MeSH terms:
Fatty Liver
Liver Diseases
Digestive System Diseases

ClinicalTrials.gov processed this record on July 29, 2014