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

11_0020WWU

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.

2023-10-01

2026-03-31

2026-03-31

Recruiting

60

Inclusion Criteria: * 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 Criteria: * 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.

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}

2023-10-23