Clinical trial NCT06095713: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Official title: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fabry Disease
Keywords: Pegunigalsidase-alfa
Lead sponsor: Universität Münster
Sponsor class: OTHER
Collaborators: name: Chiesi GmbH
First posted: 2023-10-23
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Patient with Fabry Disease

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.

Eligibility criteria: 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.

Primary outcomes:
measure: eGFR
timeFrame: yearly
Secondary outcomes:
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
city: Hamburg
country: Germany
facility: Fabry disease center Hamburg, Universitätsklinikum Hamburg
city: Hannover
country: Germany
facility: Fabry disease center Hannover, Universitätsklinikum Hannover
city: Köln
country: Germany
facility: Fabry disease center Cologne, Universitätsklinikum Köln
city: Mainz
country: Germany
facility: Fabry disease center Mainz, Universitätsmedizin Mainz
city: Müllheim
country: Germany
facility: Fachinternistische Gemeinschaftspraxis, Müllheim
city: Münster
country: Germany
facility: Fabry disease center Münster, Universitätsklinikum Münster
city: Würzburg
country: Germany
facility: Fabry disease center Würzburg, Universitätsklinikum Würzburg
Study officials:
affiliation: Universitätsklinikum Münster
name: Eva Brand, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: