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

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.

Study Overview

• Status: Recruiting
• Conditions: Fabry Disease
• Intervention / Treatment: Drug: Pegunigalsidase-alfa

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.

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

• Study Contact: Name: Eva Brand, MD, PhD; Phone Number: +492518347518; Email: fabry-zentrum@ukmuenster.de

Study Locations

• Germany
  • Berlin, Germany
    • Not yet recruiting
    • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
    • Contact: Sima Canaan-Kühl, MD
  • Hamburg, Germany
    • Not yet recruiting
    • Fabry disease center Hamburg, Universitätsklinikum Hamburg
    • Contact: Nicole Muschol, MD
  • Hannover, Germany
    • Not yet recruiting
    • Fabry disease center Hannover, Universitätsklinikum Hannover
    • Contact: Jessica Kaufeld, MD
  • Köln, Germany
    • Not yet recruiting
    • Fabry disease center Cologne, Universitätsklinikum Köln
    • Contact: Christina Kurschat, MD, PhD
  • Mainz, Germany
    • Not yet recruiting
    • Fabry disease center Mainz, Universitätsmedizin Mainz
    • Contact: Julia B. Hennermann, MD, PhD
  • Müllheim, Germany
    • Not yet recruiting
    • Fachinternistische Gemeinschaftspraxis, Müllheim
    • Contact: Markus Cybulla, MD
  • Münster, Germany
    • Recruiting
    • Fabry disease center Münster, Universitätsklinikum Münster
    • Contact: Eva Brand, MD,PhD
  • Würzburg, Germany
    • Not yet recruiting
    • Fabry disease center Würzburg, Universitätsklinikum Würzburg
    • Contact: Peter Nordbeck, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patient with Fabry Disease

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Standard of Care; Patient treated with Pegunigalsidase-alfa according to standard of care	Drug: Pegunigalsidase-alfa; Standard of care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
eGFR	eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.	yearly

Collaborators and Investigators

• Sponsor: Westfälische Wilhelms-Universität Münster
• Collaborators: Chiesi GmbH
• Investigators: Principal Investigator: Eva Brand, MD, PhD, Universitätsklinikum Münster

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2023
• Primary Completion (Estimated): March 31, 2026
• Study Completion (Estimated): March 31, 2026

Study Registration Dates

• First Submitted: October 10, 2023
• First Submitted That Met QC Criteria: October 17, 2023
• First Posted (Actual): October 23, 2023

Study Record Updates

• Last Update Posted (Actual): October 23, 2023
• Last Update Submitted That Met QC Criteria: October 17, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Pegunigalsidase-alfa
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: 11_0020WWU

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No