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

April 20, 2023 updated by: Chiesi Farmaceutici S.p.A.

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

97

Phase

  • Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

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

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3050
        • Royal Melbourne Hospital
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 1V8
        • Capital District Health Authority
  • Czechia
    • Czech Republic
      • Prague, Czech Republic, Czechia, 12808
        • Vseobecna fakultni nemocnice v Praze
  • Finland
      • Turku, Finland, FI-20521
        • Turku University Central Hospital
  • France
      • Garches, France, 92380
        • Hospital Raymond-Poincaré
  • Hungary
      • Budapest, Hungary, 1083
        • Semmelweis Egyetem
  • Italy
    • Via Pansini
      • Napoli, Via Pansini, Italy, 80131
        • Azienda Ospedaliera Universitaria "Federico II"
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academisch Medisch Centrum
  • Norway
      • Bergen, Norway, 5021
        • Haukeland University Hospital Klinisk Forskningspost
  • Slovenia
      • Slovenj Gradec, Slovenia, 2380
        • General Hospital Slovenj Gradec
  • Spain
      • Zaragoza, Spain, 50012
        • Hospital de Dia Quiron Zaragoza
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Addenbrooke's Hospital
      • London, United Kingdom, NW3 2QG
        • The Royal Free Hospital
    • Birmingham
      • Edgbaston, Birmingham, United Kingdom, B152TH
        • University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
    • Greater Manchester
      • Salford, Greater Manchester, United Kingdom, M6 8HD
        • Salford Royal
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Uab Medicine
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • La Jolla, California, United States, 92037
        • University of California San Diego
      • Orange, California, United States, 92868
        • University of California Irvine Center
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University School of Medicine
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Michigan
      • Grand Rapids, Michigan, United States, 49525
        • Infusion Associates
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • University of Pittsburgh
    • Texas
      • Dallas, Texas, United States, 75235
        • Renal Disease Research Institute, LLC - Dallas
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Eccles Primary Children's Outpatient Services Building
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • O+O Alpan LLC
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

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 to 60 years (Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  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.

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 trial, 364 weeks
Estimated glomerular filtration rate (eGFRCKD-EPI)
Every 6 months throughout trial, 364 weeks
Cardiac assessment
Time Frame: Every 12 months to end of the study, 7 years
Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Every 12 months to end of the study, 7 years
Biomarkers for Fabry disease
Time Frame: Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
plasma Lyso-Gb3 and Gb3
Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
Record of pain medication use
Time Frame: Every two weeks for 7 years
Frequency of pain medication use, or pre-infusion medication
Every two weeks for 7 years
Kidney function 2
Time Frame: Every 6 months to the end of the study, 7 years
Protein/Creatinine ratio, spot urine test (UPCR)
Every 6 months to the end of the study, 7 years
Pain assessment
Time Frame: Every 6 months up to the end of the study, 7 years
short form Brief Pain Inventory (BPI)
Every 6 months up to the end of the study, 7 years
Symptom assessment
Time Frame: Every 12 months up to the end of the study, 7 years
Mainz Severity Score Index (MSSI)
Every 12 months up to the end of the study, 7 years
Quality of life assessment
Time Frame: Every 6 months up to the end of the study, 7 years
quality of life (EQ-5D-5L)
Every 6 months up to the end of the study, 7 years

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)

September 16, 2018

Primary Completion (Anticipated)

January 1, 2025

Study Completion (Anticipated)

April 1, 2025

Study Registration Dates

First Submitted

May 24, 2018

First Submitted That Met QC Criteria

June 11, 2018

First Posted (Actual)

June 21, 2018

Study Record Updates

Last Update Posted (Actual)

April 21, 2023

Last Update Submitted That Met QC Criteria

April 20, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLI-06657AA1-04

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.

Clinical Trials on Fabry Disease

Clinical Trials on pegunigalsidase alfa

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Senegal

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe