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RECRUITING
NCT06095713

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

Sponsor: Universität Münster

View on ClinicalTrials.gov

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

DRUG

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