Principal Investigator: Nicola Longo
Keywords: Fabry , Protalix , PB-F102 , Pegunigalsidase Alfa (PRX-102) Department: Pediatric Genetics
IRB Number: 00116016
Specialty: Pediatric Genetics
Sub Specialties: Medical Genetics
Recruitment Status: Enrolling by invitation

Contact Information

Carrie Bailey
carrie.bailey@hsc.utah.edu
8015873605

Brief Summary

STUDY DESIGN: Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa (PRX-102) as intravenous infusions every 2 weeks (±3 days). The duration of treatment is up to 48 months or until pegunigalsidase alfa is available to the patient, or at the discretion of the Sponsor.

 

OBJECTIVES: To evaluate the ongoing safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa (PRX-102) every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30

Inclusion Criteria

  1. Completion of study PB-102-F20 (IRB_00093689) or PB-102-F30 (not conducted at University of Utah) 
  2. The patient signs informed consent
  3. 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.

Exclusion Criteria

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.