LION
Research type
Research Study
Full title
Leigh syndrome: Investigating Outcome measures & Natural history (LION) – a prospective, longitudinal cohort study
IRAS ID
283535
Contact name
Robert McFarland
Contact email
Sponsor organisation
Newcastle upon Tyne Hospitals NHS Foundation Trust
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
Leigh syndrome (LS) is a severe and progressive neurodegenerative disorder that is the commonest presentation of paediatric mitochondrial disease. It is associated with developmental regression (loss of previously acquired cognitive and motor skills) and muscle, movement and balance problems.
Onset of this very rare condition generally occurs in early childhood, however onset in adolescence and early adulthood has also been observed.There are currently no effective treatments for LS and it is often fatal within a few years of onset.
Although understanding of the clinical signs and symptoms of LS has expanded rapidly since it was first described in the early 1950s, the natural history (progression of the condition over time) of LS remains unclear and may hold the key to development of effective treatment strategies.
The aim of the study is to quantify the disease burden of LS and to describe the natural history of the condition. In addition, potential outcomes for future clinical trials and studies of potential treatments will be assessed. It is hoped that the information gained from this study will inform the design of future clinical trials and studies of treatments for LS.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
20/NE/0231
Date of REC Opinion
28 Oct 2020
REC opinion
Favourable Opinion