This is an open label switch over study to assess the safety and efficacy of PRX-102 (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a stable dose (\>80% labelled dose/kg) for at least 6 months. Patients will be screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, the patient will be enrolled and switched from their agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25% of treated patients will be female.
Dosage and administration details: pegunigalsidase alfa individual dose for each patient was prepared according to the patient's weight. Pegunigalsidase alfa administrated at 1 mg/kg, intravenously over 3 hours, every 2 weeks. After the first 2 months of treatment with pegunigalsidase alfa, infusion time may be reduced gradually to 1.5 hours pending patient tolerability.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
PRX-102 1 mg/kg every 2 weeks
Royal Melbourne Hospital
Parkville, Victoria, Australia
Capital Health
Halifax, Nova Scotia, Canada
Vseobecna fakultni nemocnice v Praze
Prague, Czechia
Academisch Medisch Centrum
Amsterdam, Netherlands
Helse Bergen HF Haukeland Universitetssykehus
Bergen, Norway
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia
Queen Elizabeth Hospital, Department of Neurology,
Edgbaston, Birmingham, United Kingdom
The Royal Free Hospital
London, United Kingdom
Salford Royal NHS Foundation Trust
Salford, United Kingdom
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment
Time frame: 12 months
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