A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement Therapy

The primary purpose of this study was to assess the safety and tolerability of lucerastat in adults with Fabry Disease receiving Enzyme Replacement Therapy (ERT).

The secondary objectives were to investigate the effects of lucerastat on plasma and urine levels of biomarkers, to assess its effects on renal and cardiac functions and to determine the pharmacokinetic profile of lucerastat at steady-state.

Inclusion Criteria:

• Signed informed consent form
• Male and female adult subjects with a diagnosis of Fabry Disease (FD) based on historical assessments (residual α-GAL A activity level below lower limit of normal for males and presence of a galactosidase alpha mutation for females) and a history of clinical symptoms of FD
• On ERT for at least 24 months without any change in dose within the last 6 months prior to screening

Exclusion Criteria:

• Severe renal function impairment
• Severe residual neurologic deficit
• Clinically significant unstable cardiac disease
• Any circumstances or conditions, which, in the opinion of the investigator, may have affected full participation in the study or compliance with the protocol