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Also known as Replagal, Replagal (agalsidase alfa)
agalsidase alfa · 7 trials · 1 indication
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.
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.
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® | 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 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, 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 |
| Replagal 0.2 mg/kg every other week (EOW) | EXPERIMENTAL | - |
| Agalsidase alfa (Cohort 1) | EXPERIMENTAL | Cohort 1: Patients who completed TKT023. |
| Agalsidase Alfa (Cohort 2) | EXPERIMENTAL | Cohort 2: Treatment-naive patients. |
| Name | Type | Description |
|---|---|---|
| REPLAGAL | BIOLOGICAL | REPLAGAL IV infusion. |
| 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 |
| 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...
Agalsidase alfa is used for Fabry disease, a rare inherited lysosomal storage disorder. It is an enzyme replacement therapy being developed by Takeda Pharmaceutical Company Limited (ticker: TAK). The drug is currently in Phase 3 clinical development and is considered investigational, meaning it has not been approved by regulatory authorities.
Agalsidase alfa is a form of the enzyme alpha-galactosidase A, which is deficient in patients with Fabry disease. By replacing this missing enzyme, the drug aims to address the underlying enzyme deficiency that causes the disease. It is classified as a monoclonal antibody modality in the rare disease therapeutic area.
Agalsidase alfa is developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company listed on the stock exchange under the ticker TAK. Takeda is conducting clinical trials to evaluate the safety and efficacy of this enzyme replacement therapy for Fabry disease.
Agalsidase alfa is in Phase 3 clinical development for Fabry disease. It is an investigational drug, meaning it has not been approved by regulatory authorities. The development program includes completed trials, with the most advanced being a Phase 3 study that enrolled 171 patients in Canada.
Agalsidase alfa has been studied in three completed clinical trials. NCT00084084 was a Phase 2 study in children with Fabry disease in the US and Canada. NCT01298141 was a Phase 3 safety study in Canadian patients. NCT01304277 was a Phase 2 study evaluating manufacturing process effects in adult males.
Yes, agalsidase alfa is the same as Replagal. The clinical trials for this drug reference Replagal in their titles, such as NCT00084084 and NCT01298141, which describe Replagal enzyme replacement therapy. Both names refer to the same investigational treatment for Fabry disease.