A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
CompletedPhase 3Results postedLast updated 8 June 2021
What this trial tests
Phase 3 trial testing agalsidase alfa in Fabry Disease in 171 participants. Completed in 25 September 2017.
Eligibility, any sex, with Fabry Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)Primary· From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)
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 ex
Any TEAE
Group
Value
95% CI
Replagal (Agalsidase Alfa)
163
Any Serious TEAE
Group
Value
95% CI
Replagal (Agalsidase Alfa)
74
Any TEAE Leading to Treatment Discontinuation
Group
Value
95% CI
Replagal (Agalsidase Alfa)
7
Number of Participants With Infusion-Related Reactions (IRR)Primary· From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks)
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.
Mild
Group
Value
95% CI
Replagal (Agalsidase Alfa)
21
Moderate
Group
Value
95% CI
Replagal (Agalsidase Alfa)
16
Severe
Group
Value
95% CI
Replagal (Agalsidase Alfa)
3
Life-threatening
Group
Value
95% CI
Replagal (Agalsidase Alfa)
0
Number of Participants Who Reported Positive to Immunoglobulin A (IgA)Primary· Baseline (within 6 months prior to first dose) up to Week 129
The IgA status was measured using enzyme-linked immunosorbent assay (ELISA). Number of participants who reported positive to IgA was reported.
Group
Value
95% CI
Replagal (Agalsidase Alfa)
10
Number of Participants Who Reported Positive to Immunoglobulin E (IgE)Primary· Baseline (within 6 months prior to first dose) up to Week 129
The IgE status was measured using ELISA. Number of participants who reported positive to IgE was reported.
Group
Value
95% CI
Replagal (Agalsidase Alfa)
1
Number of Participants Who Reported Positive to Immunoglobulin M (IgM)Primary· Baseline (within 6 months prior to first dose) up to Week 129
The IgM status was measured using ELISA. Number of participants who reported positive to IgM was reported.
Group
Value
95% CI
Replagal (Agalsidase Alfa)
48
Number of Participants Who Reported Positive to Anti-drug Antibody (ADA)Primary· Baseline (within 6 months prior to first dose) up to Week 285
The ADA status was measured using ELISA and electrochemiluminescent (ECL) immunoassay. Number of participants who reported positive to ADA was reported.
Group
Value
95% CI
Replagal (Agalsidase Alfa)
42
Number of Participants Who Reported Positive to Neutralizing Antibody (NAb)Primary· Baseline (within 6 months prior to first dose) up to Week 285
The NAb status was measured using enzyme activity inhibition assay. Number of participants who reported positive to NAb was reported.
Group
Value
95% CI
Replagal (Agalsidase Alfa)
27
Adverse events — posted to ClinicalTrials.gov
Time frame: From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Shire
Last refreshed: 8 June 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01298141.