Extension Study of PRX-102 for up to 60 Months

September 10, 2023 updated by: Protalix

A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients

To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

An open-label study to evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients (≥18 years of age). Patients enrolled received 1.0 mg/kg of PRX-102 as an intravenous infusion every 2 weeks (+/- 3 days) for up to 60 months and no less than 36 months.

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

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

  • Paraguay
      • Asunción, Paraguay
        • Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
  • Spain
      • Zaragoza, Spain, 50012
        • Hospital de Dia Quiron Zaragoza
  • United Kingdom
      • London, United Kingdom, NW3 2QG
        • The Royal Free Hospital
  • United States
    • Florida
      • Gainesville, Florida, United States, 32611
        • University of Florida
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins University School of Medicine
    • Texas
      • Dallas, Texas, United States, 75226
        • Institute of Metabolic Disease
    • 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:

  • Completion of study PB-102-F02
  • The patient signs informed consent
  • Female patients and male patients whose co-partners were 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 throughout the duration of the study and for 3 months after termination of treatment.

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 the patient's 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: PRX-102 (pegunigalsidase alfa)
PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
Time Frame: Every two weeks up to 60 months
Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.
Every two weeks up to 60 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma Lyso-Gb3 Concentration
Time Frame: Baseline and month 60
Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported.
Baseline and month 60
Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline and Month 60
eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported.
Baseline and Month 60

Collaborators and Investigators

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

Sponsor

Protalix

Collaborators

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)

January 16, 2014

Primary Completion (Actual)

August 26, 2020

Study Completion (Actual)

November 9, 2021

Study Registration Dates

First Submitted

November 5, 2013

First Submitted That Met QC Criteria

November 5, 2013

First Posted (Estimated)

November 11, 2013

Study Record Updates

Last Update Posted (Actual)

September 13, 2023

Last Update Submitted That Met QC Criteria

September 10, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PB-102-F03

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

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.

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