NCT02930655

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Idorsia Pharmaceuticals Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Lucerastat	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.
DRUG	Enzyme replacement therapy (ERT)	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.

Timeline

• Start date: 2015-02-01
• Primary completion: 2016-02-01
• Completion: 2016-02-01
• First posted: 2016-10-12
• Last updated: 2018-07-10

Locations

1 site across 1 country: Germany