Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alpha (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease

Conditions

Fabry disease (α-galactosidase A deficiency)

Brief summary

Safety. Changes from baseline in: • Clinical laboratory tests. • Physical examination. • Assessment of the injection site. • Electrocardiography • Brain MRI. • Treatment-emergent adverse events • The ability to taper off infusion’s pre-medication at the start of the study. • Requirement for the use of premedication overall to manage infusion reactions. • Treatment-induced anti-pegunigalsidase alfa antibodies., Efficacy. • Estimated glomerular filtration rate (eGFRCKD-EPI). • Left Ventricular Mass Index (g/m2) by echocardiogram. • Plasma Lyso-Gb3 concentration. • Plasma Gb3 concentration • Protein/Creatinine ratio, spot urine test • Frequency of pain medication use. • Exercise tolerance (Stress Test). • Short Form Brief Pain Inventory (BPI). • Mainz Severity Score Index (MSSI). • Quality of life (EQ-5D-5L).

Related papers

No related papers found yet.

Interventions

None listed

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Safety. Changes from baseline in: • Clinical laboratory tests. • Physical examination. • Assessment of the injection site. • Electrocardiography • Brain MRI. • Treatment-emergent adverse events • The ability to taper off infusion’s pre-medication at the start of the study. • Requirement for the use of premedication overall to manage infusion reactions. • Treatment-induced anti-pegunigalsidase alfa antibodies., Efficacy. • Estimated glomerular filtration rate (eGFRCKD-EPI). • Left Ventricular Mass Index (g/m2) by echocardiogram. • Plasma Lyso-Gb3 concentration. • Plasma Gb3 concentration • Protein/Creatinine ratio, spot urine test • Frequency of pain medication use. • Exercise tolerance (Stress Test). • Short Form Brief Pain Inventory (BPI). • Mainz Severity Score Index (MSSI). • Quality of life (EQ-5D-5L).	—

Measure

Time frame

Safety. Changes from baseline in: • Clinical laboratory tests. • Physical examination. • Assessment of the injection site. • Electrocardiography • Brain MRI. • Treatment-emergent adverse events • The ability to taper off infusion’s pre-medication at the start of the study. • Requirement for the use of premedication overall to manage infusion reactions. • Treatment-induced anti-pegunigalsidase alfa antibodies., Efficacy. • Estimated glomerular filtration rate (eGFRCKD-EPI). • Left Ventricular Mass Index (g/m2) by echocardiogram. • Plasma Lyso-Gb3 concentration. • Plasma Gb3 concentration • Protein/Creatinine ratio, spot urine test • Frequency of pain medication use. • Exercise tolerance (Stress Test). • Short Form Brief Pain Inventory (BPI). • Mainz Severity Score Index (MSSI). • Quality of life (EQ-5D-5L).

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Countries

Belgium, Czechia, Denmark, Italy, Norway

Outcome results