NCT03737214

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 107 (estimated)
• Sponsor: Idorsia Pharmaceuticals Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.

Detailed description

Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest. Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Lucerastat	Administered in hard gelatin capsules containing 250 mg of lucerastat.

Timeline

• Start date: 2018-12-18
• Primary completion: 2029-08-01
• Completion: 2029-11-01
• First posted: 2018-11-09
• Last updated: 2025-10-21

Locations

42 sites across 13 countries: United States, Australia, Austria, Belgium, Canada, France, Germany, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study