Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

Phase 3

Completed

Conditions: Fabry Disease

Interventions: Biological: Pegunigalsidase alfa

Registration Number: NCT03180840

Lead Sponsor: Protalix

Brief Summary: 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 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 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 30

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pegunigalsidase alfa	Pegunigalsidase alfa	Pegunigalsidase alfa 2 mg/kg intravenous infusion every 4 weeks

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-related Adverse Events (TEAE) as Assessed by CTCAE v4.03	Month 12	Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment.

Secondary Outcome Measures

Name	Time	Method

Trial Locations

Locations (14): Azienda Ospedaliera Universitaria "Federico II"
🇮🇹
Napoli, Italy
Rigshospitalet
🇩🇰
Copenhagen, Denmark
Helse Bergen HF Haukeland Universitetssykehus
🇳🇴
Bergen, Norway
University of Iowa Hospitals and Clinics
🇺🇸
Iowa City, Iowa, United States
Infusion Associates
🇺🇸
Grand Rapids, Michigan, United States
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
🇨🇿
Praha 2, Czechia
UZ Antwerpen
🇧🇪
Edegem, Belgium
Addenbrooke's Hospital
🇬🇧
Cambridge, United Kingdom
The Royal Free Hospital
🇬🇧
London, United Kingdom
University of Utah Hospital & Clinics
🇺🇸
Salt Lake City, Utah, United States
UAB Medicine
🇺🇸
Birmingham, Alabama, United States
Emory University School of Medicine
🇺🇸
Atlanta, Georgia, United States
Institute of Metabolic Disease
🇺🇸
Dallas, Texas, United States
O & O Alpan
🇺🇸
Fairfax, Virginia, United States