Fabry disease MedDRA version: 20.0 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Idorsia Pharmaceuticals Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
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 97 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 11
Exclusion criteria
Exclusion criteria: 1. Pregnant / planning to be become pregnant up to 30 days after study treatment discontinuation 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 parameters in subjects with Fabry disease; To evaluate the long-term effect of lucerastat on biomarkers of Fabry disease.;Primary end point(s): • Treatment-emergent AEs and SAEs up to FU1 visit;Timepoint(s) of evaluation of this end point: From enrollment to FU1 visit; for up to 25 months (24 months OL treatment period plus 1 month Follow-up) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): N.A;Timepoint(s) of evaluation of this end point: N.A | — |
Countries
Australia, Austria, Belgium, Canada, Germany, Ireland, Netherlands, Poland, United Kingdom, United States
Contacts
Public ContactClinical Trial Disclosure Desk
Idorsia Pharmaceuticals Ltd
Outcome results
None listed