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

Launched by UNIVERSITÄT MÜNSTER · Oct 17, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called pegunigalsidase-alfa for patients with Fabry disease, a genetic condition that affects the body’s ability to break down certain fats. The aim of the study is to gather long-term information about how this treatment works in real-life situations. Researchers will be observing 60 patients who are either starting treatment for the first time or switching from another type of enzyme replacement therapy. The study will take place over two years at eight specialized centers in Germany.

To participate in the trial, individuals must be at least 18 years old and diagnosed with Fabry disease. They should either have symptoms affecting their organs or be switching from another enzyme replacement therapy after at least 12 months. Participants will receive regular monitoring and support from the expert centers throughout the study. It’s important to note that individuals who are on dialysis, have serious organ diseases, or have had organ transplants cannot take part in this trial. Overall, this study aims to provide valuable insights into the effectiveness and safety of pegunigalsidase-alfa for people living with Fabry disease.

Gender

ALL

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.