Skip to main content
OpenTrials
Completed

NCT Number: NCT01981720

Extension Study of PRX-102 for up to 60 Months

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C), Asunción, Paraguay

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PRX-102 (pegunigalsidase alfa)

Biological

PRX-102 1 mg/kg every 2 weeks

Other names: pegunigalsidase alfa, Recombinant human alpha galactosidase-A

Primary outcomes

  1. 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.

Other outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Protalix

Industry

Collaborators

  • Chiesi Farmaceutici S.p.A.

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2020
Study completion
2021
First posted
Nov 11, 2013
Registry last updated
Sep 13, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.