Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01298141 | A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease | PHASE3 | COMPLETED | 171 | — | — | Aug 10, 2011 | Sep 25, 2017 | Jun 8, 2021 | 12 | Canada |
| NCT01304277 | This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. | PHASE2 | COMPLETED | 17 | — | — | Nov 17, 2011 | Dec 28, 2012 | Jul 19, 2021 | 5 | Canada |
| NCT00084084 | Replagal Enzyme Replacement Therapy for Children With Fabry Disease | PHASE2 | COMPLETED | 17 | — | — | Jun 10, 2004 | Jun 15, 2011 | Jul 30, 2021 | 14 | United States, Canada |
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.
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.
The IgA status was measured using enzyme-linked immunosorbent assay (ELISA). Number of participants who reported positive to IgA was reported.
The IgE status was measured using ELISA. Number of participants who reported positive to IgE was reported.
The IgM status was measured using ELISA. Number of participants who reported positive to IgM was reported.
The ADA status was measured using ELISA and electrochemiluminescent (ECL) immunoassay. Number of participants who reported positive to ADA was reported.
The NAb status was measured using enzyme activity inhibition assay. Number of participants who reported positive to NAb was reported.
| Arm | Type | Description |
|---|---|---|
| Replagal® | EXPERIMENTAL | All eligible patients may receive Replagal produced by the bioreactor process (AF Replagal) on this treatment plan until AF Replagal is commercially available for the patient, the patient's participation is discontinued, or the study is discontinued, whichever comes first. |
| Replagal® (0.2 mg/kg, IV, EOW) | EXPERIMENTAL | 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 |
| Agalsidase alfa (Cohort 1) | EXPERIMENTAL | Cohort 1: Patients who completed TKT023. |
| Agalsidase Alfa (Cohort 2) | EXPERIMENTAL | Cohort 2: Treatment-naive patients. |
| Name | Type | Description |
|---|---|---|
| agalsidase alfa | BIOLOGICAL | 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 |
Inclusion Criteria: Cohort 1: 1. The patient has a documented diagnosis of Fabry disease. 2. The patient is sufficiently compliant with study activities to participate in this treatment plan, as judged by the Investigator. 3. The patient must meet current Canadian guidelines for enzyme replacement...