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
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.
Neuropathic pain
Cornea verticillata
Any intercurrent condition or concomitant therapy considered a contraindication for kidney biopsy, as per local standard of care, or in the investigator's opinion may preclude accurate interpretation of trial data.
Urine albumin-to-creatinine ratio > 300 mg/g at screening (central laboratory) unless treated with background therapy, such as Angiotensin-converting enzyme inhibitors, Angiotensin receptor blocker or Sodium-glucose cotransporter 2 inhibitors, as per local practice.
Inherited or acquired coagulopathy, uncorrected bleeding disorders, international normalized ratio > 1.5, platelet count < 50,000/μL or inability to safely hold anticoagulants or antiplatelet therapy as applicable per local practice (usually 1-2 days for anticoagulants and 3-7 days for antiplatelets).
Hemoglobin level < 9.0 g/dL at screening.
Single group
Lucerastat
DrugHard gelatine capsules of 250 mg lucerastat
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
Barisoni Lipid Inclusion Scoring System (BLISS) is a quantitative scoring methodology for determining the number of GB3 inclusions in PTCs. A higher BLISS score is indicative of more severe disease on the histologic level.
Change from baseline to Month 18 in plasma Gb3 concentration.
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This trial is not recruiting at the moment. You can still explore other options:
