Conditions: Fabry Disease
Keywords: Fabry disease Alpha galactosidase deficiency Metabolic storage disease
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 18
Healthy volunteers: no

Brief summary:
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 months.

Detailed description:
Under the PB-102-F01 study protocol, patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) and receive PRX-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months). Patients who finish the PB-102-F01 study will be enrolled in the PB-102-F02 extension study and receive the same dose they had received in the PB-102-F01 study for an additional 38 weeks (9 months).

Arms:
arm 1: PRX-102 0.2 mg/kg every 2 weeks arm 2: PRX-102 1 mg/kg every 2 weeks arm 3: PRX-102 2 mg/kg every 2 weeks

Interventions: intervention 1: PRX-102
Intervention descriptions: intervention 1: None

Protocol document: