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German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Sponsor: Universität Münster
Summary
Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
60
Start Date
2023-10-01
Completion Date
2027-09-30
Last Updated
2025-03-30
Healthy Volunteers
No
Conditions
Interventions
Pegunigalsidase-alfa
Standard of care
Locations (8)
Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin, Germany
Fabry disease center Cologne, Universitätsklinikum Köln
Cologne, Germany
Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg, Germany
Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover, Germany
Fabry disease center Mainz, Universitätsmedizin Mainz
Mainz, Germany
Fachinternistische Gemeinschaftspraxis, Müllheim
Müllheim, Germany
Fabry disease center Münster, Universitätsklinikum Münster
Münster, Germany
Fabry disease center Würzburg, Universitätsklinikum Würzburg
Würzburg, Germany