Xolaris
Research type
Research Study
Full title
Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS).
IRAS ID
232021
Contact name
Graeme Black
Contact email
Sponsor organisation
NightstaRx Limited
Duration of Study in the UK
3 years, 2 months, 1 days
Research summary
Retinitis pigmentosa is an incurable genetic disease that causes blindness. X-linked retinitis pigmentosa (XLRP) is a very severe form of the disease, which results in rapid disease progression and severe retinal dysfunction. XLRP is caused by a defect in a certain gene located on the X-chromosome, and this is why the disease affects men and women differently. Women have two X-chromosomes and so a normal gene on one X-chromosome can compensate for a defective gene on the other X-chromosome to some extent. Men, however, only have one X-chromosome.
In XLRP, this defective gene results in a progressive degeneration of the retina. Sight loss in XLRP begins with ‘night blindness’ (i.e. loss of night vision) in the first decade, followed by a loss of peripheral vision which results in
progressively worsening ‘tunnel vision’ and severe visual impairment. Ultimately, most patients are legally blind by their fourth decade.There are currently no effective treatments available for XLRP. This observational study will support a better understanding of disease progression over time in subjects with X-linked retinitis pigmentosa (XLRP).
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
17/WM/0330
Date of REC Opinion
31 Aug 2017
REC opinion
Further Information Favourable Opinion