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Recruiting NCT04190849

European Paediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNAFLD)

Observational Non-Alcoholic Fatty Liver Disease Non-alcoholic Fatty Liver Non-alcoholic Steatohepatitis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Non-Alcoholic Fatty Liver Disease, Non-alcoholic Fatty Liver, Non-alcoholic Steatohepatitis. Basic parameters: up to 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Netherlands, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

The European Paediatric Non-alcoholic Fatty Liver Disease Registry (EU-PNAFLD): a Prospective, Longitudinal Follow-up of Children With Non-alcoholic Fatty Liver Disease

Overview

The EU-PNAFLD (The European Paediatric NALFD Registry) will be a network composed of European centres involved in the care of children with NAFLD, and will include Hepatologists, Endocrinologists, and Scientists, supported by relevant international specialists. This collaboration will build on existing infrastructure (local databases and bio-repositories) and will align with the adult European NAFLD Registry ("EPoS", Elucidating Pathways of Steatohepatitis study) to allow long-term follow-up supported by translational studies. Through an international, well-characterised large-scale cohort, we hope to: facilitate multi-centre clinical trials; extend our understanding of the key disease mechanisms of NAFLD; and establish the natural history of paediatric NAFLD.

Primary outcome measures

  • Survival [Time frame: 30-year follow-up]
Secondary outcome measures (3)
  • Cardiovascular morbidity [Time frame: 30-year follow-up]
  • Liver morbidity [Time frame: 30-year follow-up]
  • Asymptomatic progression of liver disease [Time frame: 30-year follow-up]

Eligibility criteria

Inclusion criteria

  • Diagnosis made under 18 years of age.
  • Diagnosis of NAFLD spectrum disease (simple steatosis (NAFL), steatosis with abnormal transaminases, NASH ± fibrosis or cirrhosis)
  • Diagnosis established by:
  • Radiological evidence of hepatic steatosis (e.g. increased hepatic echogenicity on ultrasound), with
  • Exclusion of secondary causes (negative serological liver screen for HBV/HCV, caeruloplasmin >0.20g/L, no history of excess alcohol consumption, no evidence of iron overload, and no clinically significant alpha-1 antitrypsin (A1AT) phenotype (i.e. SZ, ZZ, SS), with or without
  • Histology (>5% steatosis and histology consistent with paediatric NAFLD)

Exclusion criteria

  • Secondary fatty liver disease (e.g. glycogen storage diseases, Wilson disease, viral hepatitis, drug-related, autoimmune hepatitis, type 1 diabetes mellitus)
  • Post-transplant fatty liver
  • >20g/day ethanol intake

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United Kingdom · 2 centers
  • Addenbrooke's Hospital — Cambridge
  • Birmingham Children's Hospital — Birmingham
Netherlands · 1 center
  • Maastricht UMC — Maastricht

Publications

  • Mann JP, Vreugdenhil A, Socha P, Janczyk W, Baumann U, Rajwal S, Casswall T, Marcus C, van Mourik I, O'Rahilly S, Savage DB, Noble-Jamieson G, Lacaille F, Dabbas M, Dubern B, Kelly DA, Nobili V, Anstee QM. European paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale. Contemp Clin Trials. 2018 Dec;75:67-71. doi: 10.1016/j.cct.2018.11.003. Epub 2018 Nov 5. PMID 30408605

Identifiers

NCT: NCT04190849 · A094204 · 174534

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗