Open Label Extension study (OLE) of the Modify study
Research type
Research Study
Full title
A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
IRAS ID
256040
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Idorsia Pharmaceuticals Ltd
Eudract number
2018-002210-12
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 8 months, 15 days
Research summary
This study is an extension of the MODIFY study (ID-069A301). The purpose of this new study is to evaluate if the long-term intake of lucerastat is safe and well-tolerated in adults with Fabry disease.
Lucerastat is currently not “on the market” (not available to be prescribed) in any country.
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.
In addition, lucerastat has been tested in 14 healthy people in another study plus in approximately 72 patients with Fabry Disease in the Modify study. For both studies, the results are not yet available.REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/0111
Date of REC Opinion
19 Feb 2019
REC opinion
Favourable Opinion