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

NCT04190849 | Recruiting

• 2 other identifiers: A094204174534
• Study Type: observational
• Target: 2,000
• Locations: 2 countries
• Sites: 3

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.

Conditions

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

Keywords

Cirrhosis; Liver disease; Hepatocellular carcinoma; Type 2 diabetes; Atherosclerosis

Outcome Measures

Primary Outcomes (1)

• Survival

  All-cause survival

  30-year follow-up

Secondary Outcomes (3)

• Cardiovascular morbidity

  30-year follow-up

• Liver morbidity

  30-year follow-up

• Asymptomatic progression of liver disease

  30-year follow-up

Study Arms (1)

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.

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children with NAFLD who have been diagnosed either invasively (i.e. using liver biopsy) or using some form of imaging (USS, MRI, CT, MRS) plus exclusion of secondary causes using an appropriate panel of bloods. This aims to facilitate recruitment from a representative population of children, not just those from specialist centres who have undergone biopsy. 'Secondary NAFLD' (i.e. drug-induced, post-transplant) are not eligible. Children with insulin resistance syndromes are not specifically excluded however this is not the primary focus of EU-PNAFLD and other condition-specific registries are in existence.

You may qualify if:

• 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
• Histology (\>5% steatosis and histology consistent with paediatric NAFLD)

You may not qualify if:

• 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

Sponsors & Collaborators

• Cambridge University Hospitals NHS Foundation Trust: lead
• The European Association for the Study of the Liver: collaborator
• Children's Liver Disease Foundation: collaborator

Study Sites (3)

Maastricht UMC

Maastricht, Netherlands

ACTIVE NOT RECRUITING

Addenbrooke's Hospital

Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

RECRUITING

Birmingham Children's Hospital

Birmingham, United Kingdom

RECRUITING

Related 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.

  PMID: 30408605 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

EDTA blood for DNA extraction from patient (+/- both parents) (Fasting) serum for metabolite profiling

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease; Fibrosis; Liver Diseases; Carcinoma, Hepatocellular; Diabetes Mellitus, Type 2; Atherosclerosis

Condition Hierarchy (Ancestors)

Fatty Liver; Digestive System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases

Study Officials

• David B Savage

  University of Cambridge

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jake P Mann, MRCPCH

CONTACT

0044124644; jm2032@cam.ac.uk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 30 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Hepatology registrar

Study Record Dates

• First Submitted: December 5, 2019
• First Posted: December 9, 2019
• Study Start: November 14, 2017
• Primary Completion (Estimated): November 1, 2047
• Study Completion (Estimated): November 1, 2047
• Last Updated: March 4, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); NL (1)