Principal Investigator: Nicola Longo
Keywords: Fabry , PRX-102 Department: Pediatric Genetics
IRB Number: 00116015
Specialty: Pediatric Genetics
Sub Specialties: Medical Genetics
Recruitment Status: Enrolling by invitation

Contact Information

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

Brief Summary

OBJECTIVES: To evaluate the ongoing safety and efficacy parameters of 2 mg/kg pegunigalsidase alfa every 4 weeks in adult Fabry patients.

This is an open-label study to assess the safety and efficacy of pegunigalsidase alfa treatment of 2 mg/kg every 4 weeks.

The duration of treatment will be up to 36 months or until pegunigalsidase alfa is available to the patient or at the discretion of the Sponsor.

In the case of clear clinical deterioration, the treatment may be changed to 1 mg/kg every 2 weeks at the Investigator’s discretion and discussion with the Medical Monitor.

Inclusion Criteria

1. Completion of study PB-102-F50.
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 accepted, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence.

Exclusion Criteria

The following criteria excludes a patient from study enrollment: 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.