NCT06095713

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 60 (estimated)
• Sponsor: Universität Münster · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Pegunigalsidase-alfa, a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD), was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy compared with available products. The rationale of the current project is that disease progression of patients with FD can be stabilized comparable to patients under current ERT, leading to a validation of the clinical phase 3-studies and a transfer of these previous outcomes to a nationwide "real world" designed study in Germany.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Pegunigalsidase-alfa	Standard of care

Timeline

• Start date: 2023-10-01
• Primary completion: 2027-09-30
• Completion: 2027-09-30
• First posted: 2023-10-23
• Last updated: 2025-03-30

Locations

8 sites across 1 country: Germany