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

Phase 3

Completed

• Conditions: alpha-galactosidase A deficiency; Fabry's disease; 10027664

• Registration Number: NL-OMON55750
• Lead Sponsor: Idorsia Pharmaceuticals Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

1. Signed and dated ICF prior to any study-mandated procedure;
2. Subject completed the 6-month, double-blind treatment period in study ID
069A301.

Exclusion Criteria

1. Pregnant / planning to become pregnant or lactating subject;
2. 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;
3. 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:
4. Subject*s eGFR per the Chronic Kidney Disease Epidemiology Collaboration
creatinine equation < 15 mL/min/1.73 m2;
5. Subject experienced an event of acute kidney injury Common Terminology
Criteria for Adverse Event (CTCAE) grade 2 or above;
6. Subject experienced an event of stroke CTCAE grade 3 or above;
7. Subject experienced an event of heart failure leading to in-patient
hospitalization or prolongation of ongoing hospitalization.

Study & Design

• Study Type: Interventional
• Study Design: Not specified