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

Last updated: March 25, 2025
Sponsor: Universität Münster
Overall Status: Active - Recruiting

Phase

N/A

Condition

Fabry Disease

Kidney Disease

Treatment

Pegunigalsidase-alfa

Clinical Study ID

NCT06095713
11_0020WWU
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 withpain medication or patients with GI symptoms not relieved with standard medicationor ERT switch patients (under ERT for ≥12 months).

  • Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for atleast 4 weeks before screening.

  • Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeksbefore screening.

  • Female patients must have a negative pregnancy test and use a medically acceptedform of contraception throughout the study.

Exclusion

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 5years) that in the opinion of the investigator would preclude participation in thetrial.

  • Patients with a history of organ transplantation.

Study Design

Total Participants: 60
Treatment Group(s): 1
Primary Treatment: Pegunigalsidase-alfa
Phase:
Study Start date:
October 01, 2023
Estimated Completion Date:
September 30, 2027

Study 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.

Connect with a study center

  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin

    Berlin,
    Germany

    Active - Recruiting

  • Fabry disease center Hamburg, Universitätsklinikum Hamburg

    Hamburg,
    Germany

    Active - Recruiting

  • Fabry disease center Hannover, Universitätsklinikum Hannover

    Hannover,
    Germany

    Active - Recruiting

  • Fabry disease center Cologne, Universitätsklinikum Köln

    Köln,
    Germany

    Active - Recruiting

  • Fabry disease center Mainz, Universitätsmedizin Mainz

    Mainz,
    Germany

    Active - Recruiting

  • Fachinternistische Gemeinschaftspraxis, Müllheim

    Müllheim,
    Germany

    Active - Recruiting

  • Fabry disease center Münster, Universitätsklinikum Münster

    Münster,
    Germany

    Active - Recruiting

  • Fabry disease center Würzburg, Universitätsklinikum Würzburg

    Würzburg,
    Germany

    Active - Recruiting

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