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

Study Overview
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Brief Summary
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A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Detailed Description
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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.

Official Title
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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

Conditions
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Fabry Disease

Intervention / Treatment
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* Drug: Lucerastat


Participation Criteria
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Eligibility Criteria
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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 , 3) not to donate ova. Fertile male only if agreement 1) to use a condom, 2) to not father a child, 3) not to donate sperm. 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Multi-center, open-label, uncontrolled, single-arm, extension study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lucerastat<br>Dose will be based on subject's eGFR. | Drug: Lucerastat<br>* Administered in hard gelatin capsules containing 250 mg of lucerastat.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment-emergent adverse events (AEs) |  | From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up) |