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

NCT06095713 | Active Not Recruiting

• 1 other identifier: 11_0020WWU
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 8

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.

Conditions

Fabry Disease

Keywords

Pegunigalsidase-alfa

Outcome Measures

Primary Outcomes (1)

• eGFR

  eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.

  yearly

Study Arms (1)

Standard of Care

Patient treated with Pegunigalsidase-alfa according to standard of care

Drug: Pegunigalsidase-alfa

Interventions

Pegunigalsidase-alfa DRUG

Standard of care

Standard of Care

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patient with Fabry Disease

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Universität Münster: lead
• Chiesi GmbH: collaborator

Study Sites (8)

Fabry disease center Berlin - Charité - Universitätsmedizin Berlin

Berlin, Germany

Location

Fabry disease center Cologne, Universitätsklinikum Köln

Cologne, Germany

Location

Fabry disease center Hamburg, Universitätsklinikum Hamburg

Hamburg, Germany

Location

Fabry disease center Hannover, Universitätsklinikum Hannover

Hanover, Germany

Location

Fabry disease center Mainz, Universitätsmedizin Mainz

Mainz, Germany

Location

Fachinternistische Gemeinschaftspraxis, Müllheim

Müllheim, Germany

Location

Fabry disease center Münster, Universitätsklinikum Münster

Münster, Germany

Location

Fabry disease center Würzburg, Universitätsklinikum Würzburg

Würzburg, Germany

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Eva Brand, MD, PhD

  Universitätsklinikum Münster

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2023
• First Posted: October 23, 2023
• Study Start: October 1, 2023
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2027
• Last Updated: April 30, 2026

Record last verified: 2025-04

Locations

DE (8)