Idorsia Pharmaceuticals Ltd.
The purpose of this clinical trial is to learn how well lucerastat works and how safe it is in untreated adult male participants with Fabry disease. The main question this clinical trial aims to answer is: • Does treatment with lucerastat affects the amount of globotriaosylceramide (Gb3), a fatty substance that builds up in the kidneys, in untreated adult men with Fabry disease? This is an open-label, single-arm trial, which means that participants will know which trial medication they receive and only one trial medication will be given. Trial participants will: * Take lucerastat every day for 18 months * Have kidney biopsies at the end and start of the trial * Visit the clinic 10 times for check-up and tests * Take part in the trial for up to 21 months in total
Inclusion Criteria: * Confirmed diagnosis of Fabry disease: * Plasma and/or leukocyte α-galactosidase A (α-GalA) \< 1% mean normal levels or * Known "pathogenic" or "likely pathogenic" Gene coding for α-galactosidase A (GLA) variant with a low level (i.e., \< 30% mean normal levels) of plasma and/or leukocyte α-GalA. * History of at least one of the following clinical manifestations of Fabry disease: * Neuropathic pain * Cornea verticillata * Angiokeratoma * Treatment-naïve or pseudo-naïve, i.e., without treatment with an approved therapy or any investigational therapy for Fabry d…
Hard gelatine capsules of 250 mg lucerastat