NAFLD Pediatric Database 2

NCT01061684 | Completed DMC

• 2 other identifiers: NAFLD Pediatric Database 2U01DK061730
• Study Type: observational
• Target: 969
• Locations: 1 country
• Sites: 12

Brief Summary

The NAFLD Database 2 will recruit at least 650 new pediatric participants with liver biopsies and contemporaneous biosamples, and will also invite pediatric participants from the prior NAFLD Database and TONIC trial (50 with a recent biopsy and 150 without a contemporaneous biopsy) 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 850 pediatric participants during the enrollment period.

Conditions

Liver Disease

Keywords

fatty liver disease; NASH; NAFLD; non-alcoholic steatohepatitis

Outcome Measures

Primary Outcomes (1)

• Liver histology scores

  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)

  varies

Study Arms (1)

NAFLD

pediatric patients with non-alcoholic fatty liver disease (NAFLD).

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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

You may qualify if:

• 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

You may not qualify if:

• Total parenteral nutrition for more than 1 month within a 6 month period before baseline liver biopsy
• Short bowel syndrome
• 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+

Sponsors & Collaborators

• Johns Hopkins Bloomberg School of Public Health: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (12)

University of California, San Diego

San Diego, California, 92103, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago (NWU)

Chicago, Illinois, 60614, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

St. Louis University

St Louis, Missouri, 63104, United States

Location

University at Buffalo-Women and Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital- UW

Seattle, Washington, 98105, United States

Location

Related Publications (2)

• Rausch JC, Lavine JE, Chalasani N, Guo X, Kwon S, Schwimmer JB, Molleston JP, Loomba R, Brunt EM, Chen YI, Goodarzi MO, Taylor KD, Yates KP, Rotter JI; NASH Clinical Research Network. Genetic Variants Associated With Obesity and Insulin Resistance in Hispanic Boys With Nonalcoholic Fatty Liver Disease. J Pediatr Gastroenterol Nutr. 2018 May;66(5):789-796. doi: 10.1097/MPG.0000000000001926.

  PMID: 29470286 DERIVED

• Molleston JP, Schwimmer JB, Yates KP, Murray KF, Cummings OW, Lavine JE, Brunt EM, Scheimann AO, Unalp-Arida A; NASH Clinical Research Network. Histological abnormalities in children with nonalcoholic fatty liver disease and normal or mildly elevated alanine aminotransferase levels. J Pediatr. 2014 Apr;164(4):707-713.e3. doi: 10.1016/j.jpeds.2013.10.071. Epub 2013 Dec 19.

  PMID: 24360992 DERIVED

Related Links

• Nonalcoholic Steatohepatitis Clinical Research Consortium
• The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Biospecimen

Retention: SAMPLES WITH DNA

plasma, serum, liver tissue

MeSH Terms

Conditions

Liver Diseases; Non-alcoholic Fatty Liver Disease

Condition Hierarchy (Ancestors)

Digestive System Diseases; Fatty Liver

Study Officials

• Ed Doo, MD

  National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2010
• First Posted: February 3, 2010
• Study Start: January 20, 2010
• Primary Completion: May 31, 2020
• Study Completion: May 31, 2020
• Last Updated: February 23, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, ANALYTIC CODE
• Time Frame: Data will become available once the funding cycle ends.
• Access Criteria: Access requires registration and approval from the NIDDK Repository.

Locations

US (12)