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

Non-Alcoholic Fatty Liver Disease Clinical Trial

— EU-PNAFLD  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04190849
• Other study ID #: A094204
• Secondary ID: 174534
• Status: Recruiting
• Start date: November 14, 2017
• Est. completion date: November 2047

Study information

• Verified date: February 2022
• Source: Cambridge University Hospitals NHS Foundation Trust
• Contact: Jake P Mann, MRCPCH
• Phone: 0044124644
• Email: jm2032[@]cam.ac.uk
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2000
• Est. completion date: November 2047
• Est. primary completion date: November 2047
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Fatty Liver
• Liver Diseases
• Non-alcoholic Fatty Liver
• Non-Alcoholic Fatty Liver Disease
• Non-alcoholic Steatohepatitis

Locations

Country	Name	City	State
Netherlands	Maastricht UMC	Maastricht
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Addenbrooke's Hospital	Cambridge	Cambridgeshire

Sponsors (3)

Lead Sponsor	Collaborator
Cambridge University Hospitals NHS Foundation Trust	Children's Liver Disease Foundation, The European Association for the Study of the Liver

Countries where clinical trial is conducted

Netherlands,  United Kingdom, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Survival	All-cause survival	30-year follow-up
Secondary	Cardiovascular morbidity	CAD, CVA, PAD	30-year follow-up
Secondary	Liver morbidity	Decompensated liver disease, transplantation, HCC development	30-year follow-up
Secondary	Asymptomatic progression of liver disease	Presence of advanced fibrosis (on biopsy or non-invasive imaging)	30-year follow-up