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

Sponsor

Cambridge University Hospitals NHS Foundation Trust (Other)

Overall Status

Recruiting

CT.gov ID

NCT04190849

Collaborator

The European Association for the Study of the Liver (Other), Children's Liver Disease Foundation (Other)

2,000

Enrollment

Locations

359.6

Anticipated Duration (Months)

666.7

Patients Per Site

1.9

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Non-Alcoholic Fatty Liver Disease Non-alcoholic Fatty Liver Non-alcoholic Steatohepatitis

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

2000 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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

Actual Study Start Date :

Nov 14, 2017

Anticipated Primary Completion Date :

Nov 1, 2047

Anticipated Study Completion Date :

Nov 1, 2047

Arms and Interventions

Arm	Intervention/Treatment
Non-alcoholic fatty liver disease patients Children (<18 years) with a diagnosis of NAFLD with radiological demonstration of increased liver fat and exclusion of other causes.

Outcome Measures

Primary Outcome Measures

Survival [30-year follow-up]
All-cause survival

Secondary Outcome Measures

Cardiovascular morbidity [30-year follow-up]
CAD, CVA, PAD
Liver morbidity [30-year follow-up]
Decompensated liver disease, transplantation, HCC development
Asymptomatic progression of liver disease [30-year follow-up]
Presence of advanced fibrosis (on biopsy or non-invasive imaging)

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Maastricht UMC	Maastricht		Netherlands
2	Addenbrooke's Hospital	Cambridge	Cambridgeshire	United Kingdom	CB2 0QQ
3	Birmingham Children's Hospital	Birmingham		United Kingdom