International NCL Registry (UK)
Research type
Research Database
Full title
International NCL Registry (UK)
IRAS ID
139916
Contact name
Ruth E Williams
Contact email
Research summary
International NCL Registry (UK)
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
14/NE/0116
Date of REC Opinion
5 Sep 2014
REC opinion
Further Information Favourable Opinion
Data collection arrangements
The DEMCHILD
project is funded by the
European Commission and aims to create clinical and molecular tools for experimental therapy of paediatric
neurodegenerative disorders, such as the Neuronal Ceroid Lipofuscinoses (NCLs). These are a group of rare
neurodegenerative diseases that present in childhood. Workpackage 3 of the DEMCHILD
project aims to study
disease prevalence and collect natural history data by means of an International Clinical Registry. This application
seeks approval for the contribution of UK clinical data to this already established International NCL patient Registry
Children and adults with a genetically confirmed diagnosis of NCL will be identified via the Batten Disease Family
Association, the Batten Disease Clinic at ELCH and via the British Paediatric Neurology Surveillance Unit (BPNSU).
Written consent to participate will be obtained from parents/guardians. Clinical and diagnostic information will be
collated from parents/guardians, from existing medical records and by telephone interview where necessary. Clinical
information will be pseudoanonymised
and uploaded to the International NCL Registry (see NCL database
questionnaire for details of the data to be collected and uploaded.Research programme
Members of the DEMCHILD consortium come from four different European countries, Germany, UK, Italy, and Finland. These are the countries with the highest expertise, both in clinical aspects and basic research, with regard to neuronal ceroid lipofuscinoses.
Research database title
International NCL Registry (UK)
Establishment organisation
Evelina London Children's Hospital
Establishment organisation address
Guys and St Thomas' NHS Foundation Trust
Lambeth Palace Raod
London
SE1 7EH