This study is in progress, not accepting new patients

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

a study on Fabry Disease

Summary

Eligibility
for people ages 18-60 (full criteria)
Location
at UC Irvine UCSD
Dates
study started
completion around

Description

Summary

The objective of CLI-06657AA1-04 (formerly 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-F03, 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

This is an open-label study. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment is until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.

Keywords

Fabry Disease, Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa

Eligibility

You can join if…

Open to people ages 18-60

  1. Completion of study PB-102-F20, PB-102-F03, or PB-102-F30
  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. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.

You CAN'T join if...

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

Locations

  • University of California San Diego
    La Jolla California 92037 United States
  • University of California Irvine Center
    Orange California 92868 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Chiesi Farmaceutici S.p.A.
ID
NCT03566017
Phase
Phase 3 Fabry Disease Research Study
Study Type
Interventional
Participants
About 97 people participating
Last Updated