European Paediatric Non-Alcoholic Fatty Liver Disease Reg... | NCT04190849

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

NCT04190849

Sponsor

Cambridge University Hospitals NHS Foundation Trust

Status

Recruiting

Last Update Posted

Mar 4, 2022Actual

Enrollment

2,000Estimated

Phase

Not provided

Conditions

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

Interventions

Not provided

Countries

Netherlands
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

Annotation Module

Unposted Annotation

Unposted Responsible Party

Document Section

Large Document Module

Document Has No Statistical Analysis Plan (SAP)

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
174534	Other Identifier	Integrated Research Application System

Status Module

Record Verification Date

Feb 2022

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 14, 2017Actual

Primary Completion Date

Nov 2047Estimated

Completion Date

Nov 2047Estimated

First Submitted Date

Dec 5, 2019

First Submission Date that Met QC Criteria

Dec 5, 2019

First Posted Date

Dec 9, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 17, 2022

Last Update Posted Date

Mar 4, 2022Actual

Name	Class
The European Association for the Study of the Liver	UNKNOWN
Children's Liver Disease Foundation	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

30 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

2,000Estimated

Label	Type	Description	Intervention Names
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.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Survival	All-cause survival	30-year follow-up

Secondary Outcomes

Measure	Description	Time Frame
Cardiovascular morbidity	CAD, CVA, PAD	30-year follow-up
Liver morbidity	Decompensated liver disease, transplantation, HCC development

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

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.

Sampling Method

Non-Probability Sample

Name	Role	Phone	Extension	Email
Jake P Mann, MRCPCH	Contact	0044124644		jm2032@cam.ac.uk

Name	Affiliation	Role
David B Savage	University of Cambridge	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Maastricht UMC	Active, not recruiting	Maastricht			Netherlands
Addenbrooke's Hospital

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
30408605	Background	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.

Type	Date	Date Unknown
Release	Jul 27, 2022
Reset	Jun 29, 2023

ID	Term
D065626	Non-alcoholic Fatty Liver Disease
D005355	Fibrosis
D008107	Liver Diseases
D006528	Carcinoma, Hepatocellular
D003924	Diabetes Mellitus, Type 2
D050197	Atherosclerosis

ID	Term
D005234	Fatty Liver
D004066	Digestive System Diseases
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms