NCT01678898

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Months to Adult Fabry Patients

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Protalix · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	PRX-102

Timeline

• Start date: 2012-10-01
• Primary completion: 2016-03-06
• Completion: 2016-03-06
• First posted: 2012-09-05
• Last updated: 2023-09-13
• Results posted: 2020-01-27

Locations

14 sites across 6 countries: United States, Australia, Paraguay, Serbia, Spain, United Kingdom