NAFLD Pediatric Database 2 (NAFLD Peds DB2)

• ClinicalTrials.gov Identifier: NCT01061684
• Recruitment Status: Completed
• First Posted: February 3, 2010
• Last Update Posted: February 23, 2022
• Sponsor: Johns Hopkins Bloomberg School of Public Health
• Collaborator: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Information provided by (Responsible Party): Johns Hopkins Bloomberg School of Public Health

Study Description

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.

Condition or disease
Liver Disease

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 969 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Nonalcoholic Fatty Liver Disease (NAFLD) Pediatric Database 2
• Actual Study Start Date: January 20, 2010
• Actual Primary Completion Date: May 31, 2020
• Actual Study Completion Date: May 31, 2020

Groups and Cohorts

Group/Cohort
NAFLD; pediatric patients with non-alcoholic fatty liver disease (NAFLD).

Outcome Measures

Primary Outcome Measures :

1. Liver histology scores [ Time Frame: varies ]
   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

Eligibility Criteria

• Ages Eligible for Study: 2 Years to 17 Years (Child)
• Sexes Eligible for Study: All
• 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

Locations

United States, California

University of California, San Diego

San Diego, California, United States, 92103

University of California, San Francisco

San Francisco, California, United States, 94143

United States, Georgia

Emory University

Atlanta, Georgia, United States, 30322

United States, Illinois

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

Chicago, Illinois, United States, 60614

United States, Indiana

Riley Hospital for Children

Indianapolis, Indiana, United States, 46202

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21205

United States, Missouri

St. Louis University

Saint Louis, Missouri, United States, 63104

United States, New York

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

Buffalo, New York, United States, 14222

Columbia University

New York, New York, United States, 10032

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229-3039

United States, Texas

Texas Children's Hospital

Houston, Texas, United States, 77030

United States, Washington

Seattle Children's Hospital- UW

Seattle, Washington, United States, 98105

Sponsors and Collaborators

Johns Hopkins Bloomberg School of Public Health

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

Investigators

• Study Director: Ed Doo, MD; National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

More Information

Additional Information:

Nonalcoholic Steatohepatitis Clinical Research Consortium 

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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

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.

• Responsible Party: Johns Hopkins Bloomberg School of Public Health
• ClinicalTrials.gov Identifier: NCT01061684
• Other Study ID Numbers: NAFLD Pediatric Database 2; U01DK061730 ( U.S. NIH Grant/Contract )
• First Posted: February 3, 2010
• Last Update Posted: February 23, 2022
• Last Verified: February 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Data will be uploaded to www.niddkrepository.org at the end of the funding.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (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.
• URL: https://repository.niddk.nih.gov/home/

Keywords provided by Johns Hopkins Bloomberg School of Public Health:

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

Additional relevant MeSH terms:

Liver Diseases; Digestive System Diseases