Standardised NBIA Patient Registry and Natural History Study
Research type
Research Study
Full title
Standardised NBIA Patient Registry and Natural History Study
IRAS ID
145553
Contact name
Thomas Klopstock
Contact email
Sponsor organisation
University of Munich
Duration of Study in the UK
1 years, 9 months, 18 days
Research summary
An international registry for patients with Neurodegeneration Associated with Brain Iron Accumulation(NBIA) was initiated and funded for the first four years by TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration), an international consortium supported by the European Union between November 1st 2011 and October 31st, 2015. TIRCON aims to improve knowledge in this rare disease group collectively called NBIA.
This international registry and natural history study will be carried out in 6 centres across the world. The aim of this study is to characterise the natural progression of NIBA and will allow for harmonisation of important patient data in one registry. This type of research study is essential for a better understanding of the disease and the success of future clinical trials. The goal is to recruit genetically confirmed NBIA patients. Participants will be assessed yearly, with a set of medical assessments and questionnaires. All of this assessment data, measurements and biological samples will form a valuable resource for research into underlying causes and the progression of NBIA.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
14/NE/0058
Date of REC Opinion
22 May 2014
REC opinion
Further Information Favourable Opinion