Fabry disease
Conditions
Brief summary
Pharmacokinetics: Derived pharmacokinetic parameters for dosing confirmation will be estimated based on concentrations of migalastat in plasma., Safety: incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug; change from baseline over time in clinical laboratory test results; change from baseline over time in vital signs;change from baseline over time in physical examination findings, change from baseline over time in body weight and height; change from baseline over time in ECG results; change from baseline to Month 12/ET in echocardiogram parameters; change from baseline to Month 12/ET in Tanner stage (for female subjects aged ≥ 8 years and male subjects aged ≥ 9 years)
Interventions
DRUGMigalastat Hydrochloride Dispersible Tablet
Sponsors
Amicus Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetics: Derived pharmacokinetic parameters for dosing confirmation will be estimated based on concentrations of migalastat in plasma., Safety: incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug; change from baseline over time in clinical laboratory test results; change from baseline over time in vital signs;change from baseline over time in physical examination findings, change from baseline over time in body weight and height; change from baseline over time in ECG results; change from baseline to Month 12/ET in echocardiogram parameters; change from baseline to Month 12/ET in Tanner stage (for female subjects aged ≥ 8 years and male subjects aged ≥ 9 years) | — |
Countries
Belgium, Germany, Spain
Outcome results
None listed