Fabry Disease
Conditions
Keywords
Pegunigalsidase-alfa
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 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.
Interventions
Standard of care
Sponsors
Universität Münster
Chiesi GmbH
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| eGFR | yearly | eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch. |
Countries
Germany
Contacts
PRINCIPAL_INVESTIGATOREva Brand, MD, PhD
Universitätsklinikum Münster
Outcome results
None listed