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

Idorsia Pharmaceuticals Ltd.14 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fabry Disease

Interventions

LucerastatDRUG

Hard gelatin capsules for oral administration formulated at a strength of 250 mg, and administered as 4 capsules in the morning and 4 capsules in the evening.

Enzyme replacement therapy (ERT)DRUG

All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Investigator Site

Würzburg, Germany

Outcomes

Primary Outcomes

Change from baseline in blood pressure

Time frame: Up to Week 12

Change from baseline in heart rate

Time frame: Up to Week 12

Change from baseline in electrocardiogram (ECG) variables

The duration (in ms) of the different ECG variables were measured using a standard 12-lead ECG

Time frame: Up to Week 12

Change from baseline in body weight

Time frame: Up to Week 12

Number of subjects with treatment-emergent adverse events and serious adverse events

Time frame: Up to Week 12

Number of subjects with adverse events leading to premature discontinuation of lucerastat or ERT

Time frame: Up to Week 12

Number of subjects with treatment-emergent abnormalities in laboratory variables

Time frame: Up to Week 12

Secondary Outcomes

Change from baseline in plasma biomarkers of Fabry Disease

Biomarkers reflecting glycolipid metabolism were measured (unit of measure: ng/mL)

Time frame: Up to Week 12

Change from baseline in urine biomarker of Fabry Disease

Biomarker reflecting glycolipid metabolism was measured (unit of measure: ng/mg)

Time frame: Up to Week 12

Change from baseline in left ventricular ejection fraction (LVEF)

Data from ClinicalTrials.gov

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