Last updated on February 2018

Study of the Safety Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients

Brief description of study

This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase alfa or agalsidase beta for at least 3 years. Safety and efficacy exploratory endpoints will be evaluated throughout the study period and pharmacokinetics will be obtained on Day 1 and Week 52.

Detailed Study Description

This is an open-label switchover study to assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa treatment of 2 mg/kg every 4 weeks in patients previously treated with enzyme-replacement therapy (ERT): agalsidase alfa or agalsidase beta, for at least 3 years and on a stable dose (>80% labelled dose/kg) for at least the last 6 months. Following screening, patients will be enrolled and switched from their current ERT (approximately 15 patients on each ERT) to receive intravenous (IV) infusions of pegunigalsidase alfa 2 mg/kg every 4 weeks for 52 weeks (total of 14 infusions). At the time of enrollment, premedication, if used for the agalsidase alfa or agalsidase beta infusions before enrollment, will be continued using the same premedication regimen during the first infusion with pegunigalsidase alfa and then will be gradually tapered down at the Investigator's discretion during the next infusions based on protocol-specified criteria. First infusions of pegunigalsidase alfa will be administered under controlled conditions at the investigation site. Based on the protocol-specified criteria, patients will be able to receive their pegunigalsidase alfa infusions at a home care setup once the Investigator and Sponsor Medical Monitor agree that it is safe to do so. Safety and efficacy exploratory endpoints will be assessed throughout the 52-week study. In the case of clear clinical deterioration, the treatment may be changed to 1.0 mg/kg every 2 weeks at the Investigator's discretion and discussion with the Medical Monitor.

Clinical Study Identifier: NCT03180840

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Eric L Wallace, MD

UAB Medicine
Birmingham, AL United States
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William Wilcox, MD

Emory University School of Medicine
Atlanta, GA United States
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Bernat John, MD

University of Iowa Hospitals and Clinics
Iowa City, IA United States
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Kahn Nedd, MD

Infusion Associates
Grand Rapids, MI United States
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Robert Hopkin, MD

Cincinnati Children's Hospital Medical Center
Cincinnati, OH United States
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Raphael Schiffmann, MD, MHSc

Institute of Metabolic Disease
Dallas, TX United States
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Nicola Longo, MD, PhD

University of Utah Hospital & Clinics
Salt Lake City, UT United States
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Ozlem Goker-Alpan, MD

O & O Alpan
Fairfax, VA United States
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Francois Eyskens, MD

UZ Antwerpen
Edegem, Belgium
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Michael L West, MD

Capital District Health Authority
Halifax, NS Canada
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Ales Linhart, MD

Fakultn poliklinika V eobecn fakultn nemocnice v Praze
Praha 2, Czechia
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Mirjam Langeveld, MD

Academisch Medisch Centrum
Amsterdam, Netherlands
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Camilla Tøndel, MD

Helse Bergen HF Haukeland Universitetssykehus
Bergen, Norway
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Pilar Giraldo, MD

Hospital Quir n Floresta
Zaragoza, Spain
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Fatih Ezgu, MD

Gazi Universitesi Tip Fakultesi Cocuk Sagligi ve Hastaliklari Anabilim Dali
Ankara, Turkey
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Patrick Deegan, MD

Addenbrooke's Hospital
Cambridge, United Kingdom
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Tarekegn G Hiwot, MD

University of Birmingham, Institute of Metabolism and Systems Research
Edgbaston, United Kingdom
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Robin Lachmann, MD

University College London Hospitals
London, United Kingdom
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Derralynn Hughes, MD

The Royal Free Hospital
London, United Kingdom
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Ana Jovanovic, MD

Salford Royal
Salford, United Kingdom
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Recruitment Status: Open

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