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

Summary

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

Timeline

Start

2018-06-21

Primary completion

2021-08-17

Completion

2021-09-02

Outcome

Missed primary endpoint

registry analysis (other test); Lucerastat vs Placebo; p = 0.3189; LS Mean difference vs. placebo 0.42 (95% CI -0.4 to 1.23); ANCOVA NCT03425539 ↗

release “the effect of lucerastat, as an oral substrate reduction therapy for the treatment of adult patients with Fabry disease, did not meet the primary endpoint.” idorsia.com ↗

release “MODIFY, the Phase 3 study of lucerastat for adult patients with Fabry disease did not meet the primary endpoint.” idorsia.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Lucerastat	Small molecule	1000 mg	Oral

Indications