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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04190849
- Acronym / sponsor ID
- EU-PNAFLD / A094204
- Conditions
- Non-Alcoholic Fatty Liver Disease, Non-alcoholic Fatty Liver, Non-alcoholic Steatohepatitis
- Keywords
- Cirrhosis, Liver disease, Hepatocellular carcinoma, Type 2 diabetes, Atherosclerosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Cambridge University Hospitals NHS Foundation TrustUniversity, hospital or other
- Collaborators
- European Association for the Study of the Liver, Children's Liver Disease Foundation
- Study start
- 14 Nov 2017 (actual)
- Primary completion
- Nov 2047 (estimated)
- Study completion
- Nov 2047 (estimated)
- First posted
- 9 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 30 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (6 listed)
- 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:
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Survival
Time frame: 30-year follow-up
All-cause survival
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2022.
- Background: PubMed 30408605Mann 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.
- Individual participant dataSharing planned: No. No IPD will be shared with researchers outside of the Registry
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
David B Savage principal investigator
University of Cambridge
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jake P Mann, MRCPCH (0044124644, jm2032@cam.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (time frame: 30-year follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04190849, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2022; AzTrials last refreshed its copy on 8 Nov 2026.