Summary

for people ages 18-60 (full criteria)
at Orange, California and other locations
study started
estimated completion

Description

Summary

The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.

Official Title

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

Details

The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment will be no less than 24 months and up to 48 months or until pegunigalsidase alfa is commercially available to the patient at the discretion of the Sponsor. An interim analysis may be performed for administrative purposes during the conduct of the study. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.

Keywords

Fabry DiseaseGlomerular filtration rateProteinuriaPRX-102pegunigalsidase alfa

Eligibility

You can join if…

Open to people ages 18-60

  • Completion of study PB-102-F20 or PB-102-F30
  • The patient signs informed consent
  • Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 1 month after treatment termination.

You CAN'T join if...

  • Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.

Locations

  • University of California Irvine Center
    OrangeCalifornia92868United States
  • Eccles Primary Children's Outpatient Services Building
    Salt Lake CityUtah84132United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Protalix
ID
NCT03566017
Phase
Phase 3
Study Type
Interventional
Last Updated