Non-Alcoholic Fatty Liver DiseaseEU-PNAFLDNCT04190849
European Paediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNAFLD)
An observational study of Non-Alcoholic Fatty Liver Disease and Non-alcoholic Fatty Liver, run by Cambridge University Hospitals NHS Foundation Trust at 3 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Non-Alcoholic Fatty Liver Disease, Non-alcoholic Fatty Liver, Non-alcoholic Steatohepatitis
- How many people
- 2,000 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 14 Nov 2017to Nov 2047
- How long will I take part?
- 30 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
United Kingdom2 sites, 2 recruiting
- Recruiting
Birmingham Children's Hospital
Birmingham
Indra van Mourik
- Recruiting
Addenbrooke's Hospital
Cambridge, Cambridgeshire, CB2 0QQ
Jake P Mann, 00447804124644, jm2032@cam.ac.uk
About this study
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
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
- Medical subject headings
- Non-alcoholic Fatty Liver Disease, Fibrosis, Liver Diseases, Carcinoma, Hepatocellular, Diabetes Mellitus, Type 2, Atherosclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
All-cause survival
Measured: 30-year follow-up
Secondary outcomes (3)
Cardiovascular morbidity
CAD, CVA, PAD
Measured: 30-year follow-up
Liver morbidity
Decompensated liver disease, transplantation, HCC development
Measured: 30-year follow-up
Asymptomatic progression of liver disease
Presence of advanced fibrosis (on biopsy or non-invasive imaging)
Measured: 30-year follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2019
- Study start14 Nov 2017
- Primary completionNov 2047 (estimated)
- Study completionNov 2047 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Non-Alcoholic Fatty Liver DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
- Target duration
- 30 Years
Sponsor and investigators
- Lead sponsor
- Cambridge University Hospitals NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- European Association for the Study of the Liver, Children's Liver Disease Foundation
- Responsible party
- Principal investigator, Jake Mann, Cambridge University Hospitals NHS Foundation Trust
- Investigators
- David B Savage, Principal investigator, University of Cambridge
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04190849
- Sponsor's protocol ID
- A094204
- Other IDs
- 174534
- Acronym
- EU-PNAFLD
- Status last verified
- Feb 2022
- First submitted
- 5 Dec 2019
- Last updated
- 4 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No IPD will be shared with researchers outside of the Registry
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.
PubMed 30408605