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

Phase 1

• Conditions: Fabry disease; MedDRA version: 20.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 24.1Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2018-002210-12-DE
• Lead Sponsor: Idorsia Pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-10-26
• Last Update Posted: 24 June 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 107

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;

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 103
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

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 clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the long-term safety and tolerability of lucerastat in subjects with Fabry disease;Secondary Objective: To evaluate the effect of lucerastat on renal function and cardiac variables in subjects with Fabry disease;<br>To evaluate the long-term effect of lucerastat on biomarkers of Fabry disease.;Primary end point(s): Treatment-emergent AEs and SAEs;Timepoint(s) of evaluation of this end point: From enrollment up to EOS (which corresponds to FU1)

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): N.A;Timepoint(s) of evaluation of this end point: N.A