A multicenter, open-label, single-arm, baseline-controlled trial to assess the efficacy and safety of lucerastat in treatment-naïve/pseudo-naïve adult male participants with Fabry disease

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2026-525207-27-00

Enrollment

Registered

2026-08-27

Start date

Unknown

Completion date

Unknown

Last updated

2026-08-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry disease

Brief summary

Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).

Detailed description

Change from baseline to Month 18 in plasma Gb3 concentration.

Interventions

DRUGLucerastat

Eligibility

Sex/Gender

Male

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).	—

Secondary

Measure	Time frame
Change from baseline to Month 18 in plasma Gb3 concentration.	—

Outcome results

None listed

Source: EU CTIS · Data processed: Aug 28, 2026