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          Last Updated: 10/31/2025

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          3 clinical trials found

            Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

            Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

            Enrollment Status: Recruiting
            Publish Date: September 19, 2025
            Intervention Type: Drug
            Study Phase: Phase 2/Phase 3

            Summary: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

            A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)

            A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)

            Enrollment Status: Recruiting
            Publish Date: April 04, 2025
            Intervention Type: Drug
            Study Phase: Phase 2/Phase 3

            Summary: The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 18-20 male and female Fabry disease patients between the ages of 13 and 60 years to be part of the study. The study is conducted in Japan.

            A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

            A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

            Enrollment Status: Recruiting
            Publish Date: November 18, 2024
            Intervention Type: Drug

            Summary: A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

            Showing 1-3 of 3

            Last Updated: 10/31/2025