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

Fabry Disease Clinical Trial

— GoPEG  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06095713
• Other study ID #: 11_0020WWU
• Status: Recruiting
• Start date: October 1, 2023
• Est. completion date: March 31, 2026

Study information

• Verified date: October 2023
• Source: Westfälische Wilhelms-Universität Münster
• Contact: Eva Brand, MD, PhD
• Phone: +492518347518
• Email: fabry-zentrum[@]ukmuenster.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: March 31, 2026
• Est. primary completion date: March 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Pegunigalsidase-alfa
Standard of care

Locations

Country	Name	City	State
Germany	Fabry disease center Berlin - Charité - Universitätsmedizin Berlin	Berlin
Germany	Fabry disease center Hamburg, Universitätsklinikum Hamburg	Hamburg
Germany	Fabry disease center Hannover, Universitätsklinikum Hannover	Hannover
Germany	Fabry disease center Cologne, Universitätsklinikum Köln	Köln
Germany	Fabry disease center Mainz, Universitätsmedizin Mainz	Mainz
Germany	Fachinternistische Gemeinschaftspraxis, Müllheim	Müllheim
Germany	Fabry disease center Münster, Universitätsklinikum Münster	Münster
Germany	Fabry disease center Würzburg, Universitätsklinikum Würzburg	Würzburg

Sponsors (2)

Lead Sponsor	Collaborator
Westfälische Wilhelms-Universität Münster	Chiesi GmbH

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	eGFR	eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.	yearly