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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02930655
• Date of registration: 10/10/2016
• Prospective Registration: No
• Primary sponsor: Idorsia Pharmaceuticals Ltd.
• Public title: A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease
• Scientific title: 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
• Date of first enrolment: February 1, 2015
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02930655
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Germany

Contacts

• Name: Nicolas Guérard
• Affiliation: Actelion

Key inclusion & exclusion criteria

Inclusion Criteria:

- Signed informed consent form

- Male and female adult subjects with a diagnosis of Fabry Disease (FD) based on
historical assessments (residual a-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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Lucerastat

Drug: Enzyme replacement therapy (ERT)

Primary Outcome(s)

Change from baseline in heart rate [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]

Change from baseline in blood pressure [Time Frame: Up to Week 12]

Change from baseline in body weight [Time Frame: Up to Week 12]

Change from baseline in electrocardiogram (ECG) variables [Time Frame: Up to Week 12]

Number of subjects with treatment-emergent abnormalities in laboratory variables [Time Frame: Up to Week 12]

Number of subjects with treatment-emergent adverse events and serious adverse events [Time Frame: Up to Week 12]

Secondary Outcome(s)

Area under the plasma concentration-time curve [AUC(tau)] of lucerastat [Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose]

Time to reach Cmax (tmax) of lucerastat [Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose]

Change from baseline in plasma biomarkers of Fabry Disease [Time Frame: Up to Week 12]

Change from baseline in left ventricular mass index (LVMi) [Time Frame: Up to Week 12]

Maximum plasma concentration (Cmax) of lucerastat [Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose]

Change from baseline in urine albumin-to-creatinine ratio (UACR) [Time Frame: Up to Week 12]

Change from baseline in urine biomarker of Fabry Disease [Time Frame: Up to Week 12]

Terminal half-life [t(1/2)]of lucerastat [Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose]

Change from baseline in estimated glomerular filtration rate (eGFR) [Time Frame: Up to Week 12]

Change from baseline in left ventricular ejection fraction (LVEF) [Time Frame: Up to Week 12]

Secondary ID(s)

AC-069-104

Source(s) of Monetary Support

Please refer to primary and secondary sponsors