Fabry Disease
Conditions
Brief summary
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.
Detailed description
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest. Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
Interventions
DRUGLucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Sponsors
Idorsia Pharmaceuticals Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Multi-center, open-label, uncontrolled, single-arm, extension study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Treatment-emergent adverse events (AEs) | From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up |
Other
| Measure | Time frame |
|---|---|
| Treatment-emergent serious adverse events (SAEs) | From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up |
| Subject estimated glomerular filtration rate (eGFR) slope | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
| Change in left ventricular mass index (LVMI) | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
| Change in plasma globotriaosylceramide (Gb3) | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
Countries
Australia, Austria, Belgium, Canada, France, Germany, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom, United States
Outcome results
None listed