Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or Endstage Renal Disease Treated with Hemodialysis
Conditions
Brief summary
Pharmacokinetic parameters and dosing confirmation will be estimated based on concentrations of migalastat in plasma and urine
Detailed description
Safety endpoints will include: adverse events, clinical laboratory parameters (serum chemistry, hematology, and urinalysis), eGFRMDRD and eGFRCKD-EPI, vital signs (blood pressure, HR, RR, and body temperature), 12-lead ECGs, physical examinations, Tolerability endpoints will include: treatment-emergent adverse events (incidence of SAEs, discontinuation due to AE, and severity of TEAE), PD endpoint will include change from baseline in plasma lyso-Gb3
Interventions
Sponsors
Amicus Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetic parameters and dosing confirmation will be estimated based on concentrations of migalastat in plasma and urine | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety endpoints will include: adverse events, clinical laboratory parameters (serum chemistry, hematology, and urinalysis), eGFRMDRD and eGFRCKD-EPI, vital signs (blood pressure, HR, RR, and body temperature), 12-lead ECGs, physical examinations, Tolerability endpoints will include: treatment-emergent adverse events (incidence of SAEs, discontinuation due to AE, and severity of TEAE), PD endpoint will include change from baseline in plasma lyso-Gb3 | — |
Countries
France, Portugal, Spain
Outcome results
None listed