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NAFLD Peds DB2: NAFLD Pediatric Database 2

Sponsor
Johns Hopkins Bloomberg School of Public Health (Other)
Overall Status
Completed
CT.gov ID
NCT01061684
Collaborator
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (NIH)
969
Enrollment
12
Locations
124.3
Actual Duration (Months)
80.8
Patients Per Site
0.6
Patients Per Site Per Month

Study Details

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 Intervention/Treatment Phase

    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 :
    Jan 20, 2010
    Actual Primary Completion Date :
    May 31, 2020
    Actual Study Completion Date :
    May 31, 2020

    Arms and Interventions

    Arm Intervention/Treatment
    NAFLD

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

    Outcome Measures

    Primary Outcome Measures

    1. Liver histology scores [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)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 17 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Site City State Country Postal Code
    1 University of California, San Diego San Diego California United States 92103
    2 University of California, San Francisco San Francisco California United States 94143
    3 Emory University Atlanta Georgia United States 30322
    4 Ann & Robert H. Lurie Children's Hospital of Chicago (NWU) Chicago Illinois United States 60614
    5 Riley Hospital for Children Indianapolis Indiana United States 46202
    6 Johns Hopkins University Baltimore Maryland United States 21205
    7 St. Louis University Saint Louis Missouri United States 63104
    8 University at Buffalo-Women and Children's Hospital of Buffalo Buffalo New York United States 14222
    9 Columbia University New York New York United States 10032
    10 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229-3039
    11 Texas Children's Hospital Houston Texas United States 77030
    12 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)

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Johns Hopkins Bloomberg School of Public Health
    ClinicalTrials.gov Identifier:
    NCT01061684
    Other Study ID Numbers:
    • NAFLD Pediatric Database 2
    • U01DK061730
    First Posted:
    Feb 3, 2010
    Last Update Posted:
    Feb 23, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Keywords provided by Johns Hopkins Bloomberg School of Public Health
    fatty liver disease
    NASH
    NAFLD
    non-alcoholic steatohepatitis
    Additional relevant MeSH terms:
    Liver Diseases

    Study Results

    No Results Posted as of Feb 23, 2022