Enzyme replacement therapy (ERT)

DRUG2 trials

Sponsors

Idorsia Pharmaceuticals Ltd., Amicus Therapeutics

Conditions

Fabry Disease

Phase 1

A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

CompletedNCT02930655

Idorsia Pharmaceuticals Ltd.Fabry Disease

Start: 2015-02-01End: 2016-02-01Updated: 2018-07-10

Unknown Phase

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

CompletedNCT04281537

Amicus TherapeuticsFabry Disease

Start: 2020-03-01End: 2022-05-18Updated: 2026-02-05

Related Papers

A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta.

2025-08-11

Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects.

2017-01-1430 citations