Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (Bright51)

May 21, 2026 updated by: Chiesi Farmaceutici S.p.A.

Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.

Study Type

Interventional

Enrollment (Actual)

29

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Edegem, Belgium, 2650
        • UZ Antwerpen
  • Czechia
      • Prague, Czechia, 128 08
        • Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
  • Denmark
      • Copenhagen, Denmark, 2100
        • Medical Endocrinology PE 2132, Rigshospitalet
  • Italy
    • Via Pansini
      • Naples, Via Pansini, Italy, 80131
        • Azienda Ospedaliera Universitaria "Federico II"
  • Norway
      • Bergen, Norway, 5021
        • Helse Bergen HF Haukeland Universitetssykehus
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Addenbrooke's Hospital
      • London, United Kingdom
        • The Royal Free Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Uab Medicine
    • Georgia
      • Atlanta, Georgia, United States, 30307
        • Emory University School of Medicine
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinica
    • Michigan
      • Grand Rapids, Michigan, United States, 49525
        • Infusion Associates
    • Texas
      • Dallas, Texas, United States, 75235
        • Renal Disease Research Institute, LLC
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah Hospitals & Clinics
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • O & O Alpan

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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, effective contraception method. 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.

Exclusion Criteria:

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental open label
Pegunigalsidase alfa
Drug: pegunigalsidase alfa
Recombinant human alpha galactosidase A
Other Names:
  • PRX-102

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of treatment-related adverse events
Time Frame: Throughout the study, 364 weeks
CTCAE v4.03
Throughout the study, 364 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Kidney function 1
Time Frame: Every 6 months throughout the duration of the study, 364 weeks
Estimated glomerular filtration rate (eGFRCKD-EPI)
Every 6 months throughout the duration of the study, 364 weeks
Cardiac assessment
Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Biomarkers for Fabry disease
Time Frame: Every 6 months throughout the duration of the study, 364 weeks
Plasma Lyso-Gb3 and Gb3
Every 6 months throughout the duration of the study, 364 weeks
Kidney function 2
Time Frame: Every 6 months throughout the duration of the study, 364 weeks
Protein/Creatinine ratio (UPCR), spot urine test
Every 6 months throughout the duration of the study, 364 weeks
Clinical assessment
Time Frame: Every four weeks throughout the duration of the study, 364 weeks
Record of pain medication and pre-medication use
Every four weeks throughout the duration of the study, 364 weeks
Pain assessment
Time Frame: Every 6 months throughout the duration of the study, 364 weeks
Short form Brief Pain Inventory (BPI)
Every 6 months throughout the duration of the study, 364 weeks
Symptom assessment
Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Mainz Severity Score Index (MSSI)
Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Quality of life assessment
Time Frame: Every 6 months throughout the duration of the study, 364 weeks
Quality of life (EQ-5D-5L)
Every 6 months throughout the duration of the study, 364 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chiesi Farmaceutici S.p.A.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 13, 2018

Primary Completion (Actual)

April 13, 2026

Study Completion (Actual)

April 13, 2026

Study Registration Dates

First Submitted

July 30, 2018

First Submitted That Met QC Criteria

August 2, 2018

First Posted (Actual)

August 3, 2018

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLI-06657AA1-03

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe