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

NCT ID: NCT06095713

Last Updated: 2025-03-30

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 60 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-10-01
• Study Completion Date: 2027-09-30

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.

Conditions

Fabry Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Study Groups

Standard of Care

Patient treated with Pegunigalsidase-alfa according to standard of care

Pegunigalsidase-alfa

Intervention Type: DRUG

Standard of care

Interventions

Pegunigalsidase-alfa

Standard of care

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Chiesi GmbH

UNKNOWN

Sponsor Role: collaborator

Universität Münster

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Eva Brand, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Universitätsklinikum Münster

Locations

Fabry disease center Berlin - Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status: RECRUITING

Fabry disease center Cologne, Universitätsklinikum Köln

Cologne, , Germany

Site Status: RECRUITING

Fabry disease center Hamburg, Universitätsklinikum Hamburg

Hamburg, , Germany

Site Status: RECRUITING

Fabry disease center Hannover, Universitätsklinikum Hannover

Hanover, , Germany

Site Status: RECRUITING

Fabry disease center Mainz, Universitätsmedizin Mainz

Mainz, , Germany

Site Status: RECRUITING

Fachinternistische Gemeinschaftspraxis, Müllheim

Müllheim, , Germany

Site Status: RECRUITING

Fabry disease center Münster, Universitätsklinikum Münster

Münster, , Germany

Site Status: RECRUITING

Fabry disease center Würzburg, Universitätsklinikum Würzburg

Würzburg, , Germany

Site Status: RECRUITING

Countries

Germany

Central Contacts

Eva Brand, MD, PhD

Role: CONTACT

fabry-zentrum@ukmuenster.de

+492518347518

Facility Contacts

Sima Canaan-Kühl, MD

Role: primary

Christina Kurschat, MD, PhD

Role: primary

Nicole Muschol, MD

Role: primary

Jessica Kaufeld, MD

Role: primary

Julia B. Hennermann, MD, PhD

Role: primary

Markus Cybulla, MD

Role: primary

Eva Brand, MD,PhD

Role: primary

Peter Nordbeck, MD, PhD

Role: primary

Other Identifiers

11_0020WWU

Identifier Type: -

Identifier Source: org_study_id