This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
|Official Title:||A Phase II Comparability Study Between Replagal® Produced From Agalsidase Alfa Manufactured by 2 Different Processes in Adult Male Patients With Fabry Disease|
- 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) [ Time Frame: Week 0 to Week 14 ]
- Dose-normalized AUC Extrapolated to Infinity (AUC∞/Dose) [ Time Frame: Week 0 to Week 14 ]
- Dose-normalized Maximum Serum Concentration (Cmax/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) [ Time Frame: Week 2 to EOS ]To Assess Safety and Tolerability by Anti-agalsidase Alfa Antibody Status, concomitant medication, vital signs and ECG.
|Study Start Date:||March 2011|
|Study Completion Date:||December 2012|
|Primary Completion Date:||December 2012 (Final data collection date for primary outcome measure)|
Experimental: Replagal® (0.2 mg/kg, IV, EOW)
Screening period of approximately 14 days during which all patients received 1 infusion of 0.2 mg/kg Replagal RB (Week 0)
Treatment period of 14 weeks during which all patients received 7 infusions of 0.2 mg/kg Replagal AF
Biological: agalsidase alfa
Other Name: Replagal
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01304277
|University of Alberta Hospital|
|Edmonton, Alberta, Canada, T6G 2H7|
|Canada, Nova Scotia|
|Queen Elizabeth II Health Sciences Centre|
|Halifax, Nova Scotia, Canada, B3H 1V8|
|The Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Toronto, Ontario, Canada, M5V 2T3|
|Hopital du Sacre-Coeur de Montreal|
|Montreal, Quebec, Canada, H4J 1C5|
|Study Director:||Anna Wijatyk, MD||Shire Human Genetic Therapies, Inc.|