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

Idorsia Pharmaceuticals

Status and phase

Active, not recruiting

Phase 3

Conditions

Fabry Disease

Treatments

Drug: Lucerastat

Study type

Interventional

Funder types

Industry

Identifiers

NCT03737214

2018-002210-12 (EudraCT Number)

ID-069A302

Details and patient eligibility

About

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.

Full description

Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country or until all subjects have reached Month 24 (for subjects participating only in Stage 1) or Month 48 (for subjects participating in Stages 1 and 2) or Month 72 (for subjects participating in Stages 1, 2 and 3), whichever is earliest.

Enrollment

107 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Signed ICF prior to any study-mandated procedure;
Subject completed the 6-month, double-blind treatment period in study ID 069A301
Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests.
Fertile male only if agreement 1) to use a condom, 2) to not father a child.

Exclusion criteria

Pregnant / planning to be become pregnant or lactating subject;
Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.

In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:

Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
Subject experienced an event of stroke CTCAE grade 3 or above;
Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.