Status:
RECRUITING
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Lead Sponsor:
Universität Münster
Collaborating Sponsors:
Chiesi GmbH
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Brief 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...
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-lif...
Eligibility Criteria
Inclusion
- Males and females, ≥18 years, diagnosed with Fabry disease.
- ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months).
- Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening.
- Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening.
- Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study.
Exclusion
- Patient is unwilling to give informed consent.
- Patient is unable to comply with the clinical protocol.
- Patients on dialysis.
- Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial.
- Patients with a history of organ transplantation.
Key Trial Info
Start Date :
October 1 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 30 2027
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT06095713
Start Date
October 1 2023
End Date
September 30 2027
Last Update
March 30 2025
Active Locations (8)
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1
Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin, Germany
2
Fabry disease center Cologne, Universitätsklinikum Köln
Cologne, Germany
3
Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg, Germany
4
Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover, Germany