This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure. An agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process. This study is designed to provide PD/PK and safety data. The assessment schedule is designed to capture the PK profile of drug uptake in the blood as well the pharmacologic effect which manifests over the course of weeks. Each patient will serve as his own control.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
17
University of Alberta Hospital
Edmonton, Alberta, Canada
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
INC Research
Toronto, Ontario, Canada
Change From Baseline to Week 16 (EOS) in Urine Gb3 Levels
Time frame: Baseline to EOS
Change From Baseline to Week 16 (EOS) in Plasma Gb3 Levels
Time frame: Baseline to EOS
Dose-normalized Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Sample (AUClast/Dose)
The dose-normalized calculation was performed by dividing the pharmacokinetic parameter by the administered dose.
Time frame: Week 0 to Week 14
Dose-normalized AUC Extrapolated to Infinity (AUC∞/Dose)
The dose-normalized calculation was performed by dividing the pharmacokinetic parameter by the administered dose.
Time frame: Week 0 to Week 14
Dose-normalized Maximum Serum Concentration (Cmax/Dose)
The dose-normalized calculation was performed by dividing the pharmacokinetic parameter by the administered dose.
Time frame: Week 0 to Week 14
To Assess Safety and Tolerability by Anti-agalsidase Alfa Antibody Status (in Serum) at End of Study
Time frame: EOS
Overall Summary of TEAEs by Treatment (Replagal RB and Replagal AF)
To Assess Safety and Tolerability by Anti-agalsidase Alfa Antibody Status, concomitant medication, vital signs and ECG.
Time frame: Week 2 to EOS
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Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, Canada