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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04974749 | A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease | PHASE3 | COMPLETED | 20 | — | — | May 1, 2022 | Jan 3, 2024 | Oct 8, 2024 | 6 | China |
| NCT01124643 | Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease | PHASE3 | COMPLETED | 35 | — | — | Apr 13, 2010 | Jul 8, 2013 | Jun 8, 2021 | 9 | United States, Australia +5 |
| NCT00864851 | Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease | PHASE3 | COMPLETED | 44 | — | — | Dec 29, 2008 | Jul 5, 2012 | Jun 9, 2021 | 12 | United States, Australia +6 |
| NCT01363492 | Safety Study of Replagal® Therapy in Children With Fabry Disease | PHASE2 | COMPLETED | 15 | — | — | May 12, 2011 | Apr 17, 2013 | Jun 9, 2021 | 5 | United States |
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. Serious AE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, and was an important medical event. A TEAE was defined as any event emerging at or after the initiation of treatment with an IP or any existing event that worsened in either intensity or frequency following exposure to the IP until the end of the safety follow-up period.
Left ventricular mass (LVM) was measured through echocardiography.
Reflects development of Anti-Agalsidase antibodies post baseline
| Arm | Type | Description |
|---|---|---|
| REPLAGAL | EXPERIMENTAL | Participants received REPLAGAL 0.2 milligrams per kilogram (mg/kg) body weight, intravenous (IV) infusion, every other week (EOW) from Day 1 (Week 0) up to Week 52. |
| Replagal 0.2 mg/kg EOW | EXPERIMENTAL | Intravenous, 0.2mg/kg EOW |
| Replagal 0.2 mg/kg, IV, every other week | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks. |
| Replagal 0.2 mg/kg, IV, weekly | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks. |
| Replagal 0.4 mg/kg, IV, weekly | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks. |
| Replagal 0.2 mg/kg every other week (EOW) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| REPLAGAL | BIOLOGICAL | REPLAGAL IV infusion. |
| Replagal (agalsidase alfa) | BIOLOGICAL | 0.2 mg/kg administered over 40 minutes every other week (EOW) |
Inclusion Criteria: * Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. Fo...