The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease.
This study will evaluate the safety of agalsidase alfa in patients with Fabry disease. Patients diagnosed with Fabry disease who meet current Canadian guidelines for enzyme replacement therapy will be eligible to enroll in the study and will receive agalsidase alfa at a dose of 0.2 mg/kg body weight administered by an IV infusion over 40 minutes every week or every other week, based on previous treatment. Shire has implemented a change to the drug substance manufacturing process. Safety data will be collected in patients receiving product manufactured with this process. There are no changes to the drug product formulation, manufacturing site, manufacturing process, and container closure.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
171
Cohort 1: 0.2 mg/kg body weight administered as an intravenous (IV) infusion over 40 minutes every other week (EOW) Cohort 2: 0.2 mg/kg body weight administered as an intravenous (IV) infusion over 40 minutes weekly
Alberta Children's Hospital
Calgary, Alberta, Canada
University of Alberta Hospital
Edmonton, Alberta, Canada
Vancouver General Hospital
Vancouver, British Columbia, Canada
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related.Treatment-emergent adverse events (TEAEs) were defined as those events which occurred or worsened in severity after first treatment with Replagal AF until 30 days after the last dose. A serious AE (SAE) was any AE occurred at any dose that resulted in death, life-threatening, hospitalization, prolongation of existing hospitalization, persistent or significant disability or incapacity and congenital anomaly or birth defect.
Time frame: From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)
Number of Participants With Infusion-Related Reactions (IRR)
An IRR (also referred to as infusion-related adverse event \[IRAE\]) was defined as an AE that began either during the infusion or within 12 hours after the start of the infusion and was judged as possibly or probably related to study drug. The IRRs were classified based on the severity as Mild=No limitation of usual activities, Moderate=Some limitation of usual activities, Severe=Inability to carry out usual activities and Life-threatening=Immediate risk of death. The number of participants with infusion-related reactions was reported.
Time frame: From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)
Number of Participants Who Reported Positive to Immunoglobulin A (IgA)
The IgA status was measured using enzyme-linked immunosorbent assay (ELISA). Number of participants who reported positive to IgA was reported.
Time frame: Baseline (within 6 months prior to first dose) up to Week 129
Number of Participants Who Reported Positive to Immunoglobulin E (IgE)
The IgE status was measured using ELISA. Number of participants who reported positive to IgE was reported.
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University of Manitoba
Winnipeg, Manitoba, Canada
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada
Izaak Walton Killam (IWK) Health Centre
Halifax, Nova Scotia, Canada
Kingston General Hospital
Kingston, Ontario, Canada
London Health Sciences Centre - Victoria Hospital
London, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
The Fred A. Litwin Family Centre in Genetic Medicine
Toronto, Ontario, Canada
...and 2 more locations
Time frame: Baseline (within 6 months prior to first dose) up to Week 129
Number of Participants Who Reported Positive to Immunoglobulin M (IgM)
The IgM status was measured using ELISA. Number of participants who reported positive to IgM was reported.
Time frame: Baseline (within 6 months prior to first dose) up to Week 129
Number of Participants Who Reported Positive to Anti-drug Antibody (ADA)
The ADA status was measured using ELISA and electrochemiluminescent (ECL) immunoassay. Number of participants who reported positive to ADA was reported.
Time frame: Baseline (within 6 months prior to first dose) up to Week 285
Number of Participants Who Reported Positive to Neutralizing Antibody (NAb)
The NAb status was measured using enzyme activity inhibition assay. Number of participants who reported positive to NAb was reported.
Time frame: Baseline (within 6 months prior to first dose) up to Week 285