SMR-3438
Research type
Research Study
Full title
A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease
IRAS ID
255810
Contact name
Amanda Knock
Contact email
Sponsor organisation
Katairo GmbH
Eudract number
2018-001496-20
Duration of Study in the UK
1 years, 6 months, 0 days
Research summary
This is a multi-national (UK, Germany, Italy and the Netherlands), multi-centre (5 sites), double-masked, placebo controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease (a genetic eye disorder that causes progressive vision loss).
There are currently no treatments available to cure Stargardt disease, only approaches that will slow down the progression of the disease. Patients with Stargardt disease have abnormal levels of lipofuscin (fatty yellow pigment) in their retinal pigment epithelium. Soraprazan has been shown to reduce lipofuscin deposits (which causes vision loss in Stargardt disease) in mice and primates.
A total of 90 patients will be included in this study, 60 patients will be included in the soraprazan group and 30 patients will be included in the placebo group. A screening/baseline period will be followed by treatment of oral soraprazan/placebo once a day, with monthly visits up to month 12.REC name
North West - Haydock Research Ethics Committee
REC reference
18/NW/0845
Date of REC Opinion
15 Jan 2019
REC opinion
Further Information Favourable Opinion