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An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients

Fabry Disease Clinical Trial

Official title:
An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients

Clinical Trial Summary

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg), to receive the same dose they had received in Phase 1/2 study PB-102-F01, and will continue to receive PRX-102 as an intravenous infusion every 2 weeks for 38 weeks.

Clinical Trial Description

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) to receive the same dose they had received in the Phase 1/2 study, PB-102-F01, and will continue to receive intravenous infusions of PRX-102 every 2 weeks for 38 weeks (9 months).

All exploratory endpoints that were evaluated during the Phase 1/2 study PB-102-F01 will continue to be assessed in the extension protocol (study PB-102-F02).

The additional exploratory efficacy parameters that were evaluated at baseline of the Phase 1/2 study PB-102-F01 as a reference point, before the initiation of treatment, will be evaluated after 3 months into the extension study (total treatment of 6 months). These parameters include Gb3 concentration in renal and skin tissues, LVM, MSSI and cardiac function tests (echocardiography and stress test). Cerebrovascular disease (clinical and MRI) will be evaluated at last infusion (total treatment of 12 months). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01769001
Study type Interventional
Source Protalix
Contact
Status Completed
Phase Phase 1/Phase 2
Start date March 2013
Completion date March 6, 2016
View Details
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