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

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 or completed at least 48 months in study PB-102-F03. 


  • Estimated glomerular filtration rate (eGFRCKD-EPI)
  • Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI)
  • Plasma Lyso-Gb3 concentration
  • Plasma Gb3 concentration
  • Protein/Creatinine ratio, spot urine test (UPCR)
  • Frequency of pain medication use
  • Exercise tolerance (Stress Test)
  • Short Form Brief Pain Inventory (BPI)
  • Mainz Severity Score Index (MSSI)
  • Quality of life (EQ-5D-5L)
  • Fabry disease Clinical events

Inclusion Criteria

  1. Completion of study PB-102-F20 (IRB_00093689) or PB-102-F30 (not conducted at University of Utah), or completed at least 48 months in study PB-102-F03 (not conducted at the 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 42 days 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.

Medications having the potential to interfere with the evaluation of efficacy are excluded throughout the trial.

The following medications are strictly prohibited during the study:

•       Fabrazyme® (agalsidase beta)  

•       Replagal® (agalsidase alfa)

•       Galafold™ (migalastat)

•       Any other investigational drug for treating Fabry disease