NCT03425539

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 118 (actual)
• Sponsor: Idorsia Pharmaceuticals Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

Detailed description

The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Lucerastat	Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) twice daily (b.i.d.); dose adjusted for renal function.
DRUG	Placebo	Placebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function.

Timeline

• Start date: 2018-06-21
• Primary completion: 2021-08-17
• Completion: 2021-09-02
• First posted: 2018-02-07
• Last updated: 2024-08-09
• Results posted: 2024-08-09

Locations

49 sites across 14 countries: United States, Australia, Austria, Belgium, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study