Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.

Inclusion criteria

• Signed and dated ICF prior to any study-mandated procedure;
• Male or female adult subjects;
• FD diagnosis confirmed with local genetic test results;
• Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
• Enzyme replacement therapy (ERT) status:

• Subject never treated with ERT; or
• Subject has not received ERT for at least 6 months prior to screening; or
• Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
• A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
• Subjects with moderate or severe neuropathic pain during the screening period.

Exclusion criteria

• Pregnant, planning to be become pregnant, or lactating subject.
• Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
• Subject on regular dialysis for the treatment of chronic kidney disease.
• Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
• Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
• Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.