Fabry disease study to determine the safety and efficacy of lucerastat
Research type
Research Study
Full title
A multicenter, double-blind, randomized, placebo controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
IRAS ID
242324
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Idorsia Pharmaceuticals Ltd
Eudract number
2017-003369-85
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
The purpose of the study is to test if lucerastat, the new study drug, can reduce symptoms of Fabry disease such as pain and stomach symptoms when administered alone. The purpose is also to see if lucerastat is safe for people with Fabry disease.
Lucerastat is currently not “on the market” (not available to be prescribed to you) in any country.
So far, lucerastat has been tested in 86 healthy people (who do not have Fabry disease) and 10 patients with Fabry disease. The level of fatty substance in the blood was reduced in people with Fabry disease treated with lucerastat for 12 weeks.REC name
London - Westminster Research Ethics Committee
REC reference
18/LO/0434
Date of REC Opinion
14 May 2018
REC opinion
Further Information Favourable Opinion