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

Idorsia Pharmaceuticals

Status and phase

Completed

Phase 1

Conditions

Fabry Disease

Treatments

Drug: Enzyme replacement therapy (ERT)

Drug: Lucerastat

Study type

Interventional

Funder types

Industry

Identifiers

NCT02930655

AC-069-104

Details and patient eligibility

About

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.

Enrollment

14 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

14 participants in 2 patient groups

Lucerastat group

Experimental group

Description:

Ten subjects with Fabry Disease received 1000 mg of oral lucerastat twice daily for 12 weeks in addition to their standard of care treatment (enzyme replace therapy).

Treatment:

Drug: Lucerastat

Drug: Enzyme replacement therapy (ERT)

Control group

Experimental group

Description:

Four subjects with Fabry Disease under enzyme replace therapy (ERT) as standard of care treatment were included as a control group.

Treatment:

Drug: Enzyme replacement therapy (ERT)