NAFLD Pediatric Database 2 (NAFLD Peds DB2)

This study is currently recruiting participants.
Verified October 2013 by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor:
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier:
NCT01061684
First received: February 2, 2010
Last updated: October 17, 2013
Last verified: October 2013
  Purpose

The NAFLD Database 2 will recruit at least 250 new pediatric participants and will also invite pediatric participants from the prior NAFLD Database and TONIC trial to enroll in the NAFLD Pediatric Database 2 study. Combining the new and continuing participants leads to a recruitment goal for the pediatric Database 2 of 300 pediatric participants with liver biopsies and contemporaneous biosamples during the 18 month enrollment period.

An estimated additional 150 pediatric participants continuing from the NAFLD Database study and TONIC trial will also be enrolled, but without a contemporaneous liver biopsy at the time of enrollment.


Condition
Liver Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Nonalcoholic Fatty Liver Disease (NAFLD) Pediatric Database 2

Resource links provided by NLM:


Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Primary Outcome Measures:
  • Liver histology scores [ Time Frame: varies ] [ Designated as safety issue: No ]
    Liver histology scores (derived from central reading of liver biopsy at entry, standard of care biopsy done during screening or follow-up, or liver biopsy obtained for the TONIC trial)


Biospecimen Retention:   Samples With DNA

plasma, serum, liver tissue


Estimated Enrollment: 450
Study Start Date: December 2009
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts
NAFLD
pediatric patients with non-alcoholic fatty liver disease (NAFLD).

Detailed Description:

All the new pediatric participants will have had a liver biopsy within 120 days prior to enrollment coupled with contemporaneous biosamples within 90 days prior to enrollment and up to 90 days before or 4-90 days after the biopsy. We estimate that at least 50 of the continuing pediatric participants will be due for a standard of care liver biopsy at the time of their enrollment into the pediatric Database 2 study, and will, as a result, also have a liver biopsy and contemporaneous biosamples.

  Eligibility

Ages Eligible for Study:   2 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Participants at least 2 years of age and less than 18 years of age with known or suspected NAFLD or NASH-related cirrhosis

Criteria

Inclusion Criteria:

  • Continuing participants:

    • Previously enrolled in the NAFLD Database study or TONIC trial
    • Age at least 2 years and not older than 17 years during the consent process
    • Willingness to continue to be followed for up to 4 years
    • Ability and willingness to give written, informed parental consent and child assent, per local IRB guidelines, to be enrolled into the pediatric Database 2 study
  • New participants:

    • Age at least 2 years of age and not older than 17 years during the consent process
    • Willingness to be followed for up to 4 years
    • Ability and willingness to give written, informed parent consent and child assent to be enrolled into the pediatric Database 2 study
    • Minimal or no alcohol use history consistent with NAFLD
    • Having undergone a liver biopsy that is obtained within 120 days of enrollment
    • Collection of biosamples (serum, plasma, DNA, and, if available, liver tissue) within 90 days prior to enrollment and 0-90 days before or 4-90 days after the standard of care liver biopsy

Exclusion Criteria:

  • Total parenteral nutrition for more than 1 month within a 6 month period before baseline liver biopsy
  • Short bowel syndrome
  • History of gastric or jejunoileal bypass preceding the diagnosis of NAFLD. Bariatric surgery performed following enrollment is not exclusionary. Liver biopsies obtained during bariatric surgery cannot be used for enrollment because of the associated surgical or anesthetic acute changes and the weight loss efforts that precede bariatric surgery
  • History of biliopancreatic diversion
  • Evidence of advanced liver disease defined as a Child-Pugh-Turcotte score equal to or greater than 10
  • Evidence of chronic hepatitis B as marked by the presence of HBsAg in serum (participants with isolated antibody to hepatitis B core antigen, anti-HBc total, are not excluded)
  • Evidence of chronic hepatitis C as marked by the presence of anti-HCV or HCV RNA in serum
  • Low alpha-1-antitrypsin level and ZZ phenotype (both determined at the discretion of the investigator)
  • Wilson's disease
  • Known glycogen storage disease
  • Known dysbetalipoproteinemia
  • Known phenotypic hemochromatosis (HII greater than 1.9 or removal of more than 4 g of iron by phlebotomy)
  • Prominent bile duct injury (florid duct lesions or periductal sclerosis) or bile duct paucity
  • Chronic cholestasis
  • Vascular lesions (vasculitis, cardiac sclerosis, acute or chronic Budd-Chiari, hepatoportal sclerosis, peliosis)
  • Iron overload greater than 3+
  • Zones of confluent necrosis, infarction, massive or sub-massive, pan-acinar necrosis
  • Multiple epithelioid granulomas
  • Congenital hepatic fibrosis
  • Cystic fibrosis
  • Polycystic liver disease
  • Other metabolic or congenital liver disease
  • Evidence of systemic infectious disease
  • Known HIV positive
  • Disseminated or advanced malignancy
  • Concomitant severe underlying systemic illness that in the opinion of the investigator would interfere with completion of follow-up
  • Active drug use or dependence that, in the opinion of the study investigator, would interfere with adherence to study requirements
  • Any other condition, which in the opinion of the investigator would impede compliance or hinder completion of study
  • Inability for parent to provide informed consent and child 8 years or greater to give assent
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01061684

Locations
United States, California
University of California, San Diego Recruiting
San Diego, California, United States, 92103
Contact: Janis Durelle    619-543-5226    jdurelle@ucsd.edu   
Principal Investigator: Jeffrey Schwimmer, MD         
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Camille Langlois    415-476-1756    langloisc@peds.ucsf.edu   
Principal Investigator: Philip Rosenthal, MD         
United States, Illinois
Children's Memorial Hospital (NWU) Recruiting
Chicago, Illinois, United States, 60614
Contact: Katie Amsden    773-868-8009    kamsden@childrensmemorial.org   
Principal Investigator: Peter Whittington, MD         
United States, Indiana
Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Ann Klipsch, RN    317-274-9605    aeye@iupui.edu   
Contact: Elizabeth Byam, RN    (317) 274-3774    ebyam@iupui.edu   
Principal Investigator: Jean Molleston, MD         
United States, Maryland
Johns Hopkins University Active, not recruiting
Baltimore, Maryland, United States, 21205
United States, Missouri
St. Louis University Recruiting
St. Louis, Missouri, United States, 63104
Contact: Susan Stewart    314-977-9337    stewarse@slu.edu   
Contact: Judy Thompson, RN    (314) 977-9407    ThompJA@slu.edu   
Sub-Investigator: Adjay Jain, MD         
United States, New York
Mount Sinai Kravis Children's Hospital Active, not recruiting
New York, New York, United States, 10029
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Elena Reynosa, MD    212-305-6274    er2564@columbia.edu   
Principal Investigator: Joel Lavine, MD, PhD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Kimberlee Bernstein    513-636-4406    Kimberlee.bernstein@cchmc.org   
Principal Investigator: Stavra Xanthakos, MD         
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Sara Barlow, MD    832-822-1013    sbarlow@bcm.edu   
Contact: Lucinda Clarke    (713) 798-7122    lucindac@bcm.edu   
Principal Investigator: Stephanie Abrams, MD         
United States, Washington
Seattle Children's Hospital- UW Recruiting
Seattle, Washington, United States, 98105
Contact: Melissa Young    206-987-1037    melissa.young@seattlechildrens.org   
Principal Investigator: Karen Murray, MD         
Sponsors and Collaborators
Investigators
Study Director: Ed Doo, 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: NCT01061684     History of Changes
Other Study ID Numbers: NAFLD Pediatric Database 2
Study First Received: February 2, 2010
Last Updated: October 17, 2013
Health Authority: United States: Federal Government

Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
fatty liver disease
NASH
NAFLD
non-alcoholic steatohepatitis

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

ClinicalTrials.gov processed this record on April 17, 2014