Observational study of Fabry disease patients receiving Pegunigalsidase-alfa treatment
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
This study is testing how well a new treatment called Pegunigalsidase-alfa works for people with Fabry disease over two years, gathering information on its safety and effectiveness in real-life settings.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 60 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Universität Münster Academic / other |
| Locations | 8 sites (Berlin and 7 other locations) |
| Trial ID | NCT06095713 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather long-term data on the effects of Pegunigalsidase-alfa, a new enzyme replacement therapy for Fabry disease, in a real-world setting. It will involve 60 patients from eight specialized Fabry centers across Germany, including both therapy-naïve individuals and those previously treated with other enzyme replacement therapies. The study will document the treatment's efficacy and safety over a two-year period, focusing on its pharmacokinetics and immunogenicity compared to existing therapies.
Who should consider this trial
Good fit: Ideal candidates include adults diagnosed with Fabry disease, either therapy-naïve or those switching from other enzyme replacement therapies.
Not a fit: Patients who are on dialysis or unable to comply with the clinical protocol may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term effectiveness and safety of Pegunigalsidase-alfa, potentially improving treatment options for Fabry disease patients.
How similar studies have performed: Previous studies on enzyme replacement therapies for Fabry disease have shown promising results, suggesting that this approach may also yield beneficial outcomes.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Berlin and 7 other locations
- Fabry disease center Berlin - Charité - Universitätsmedizin Berlin — Berlin, Germany (Recruiting)
- Fabry disease center Hamburg, Universitätsklinikum Hamburg — Hamburg, Germany (Recruiting)
- Fabry disease center Hannover, Universitätsklinikum Hannover — Hannover, Germany (Recruiting)
- Fabry disease center Cologne, Universitätsklinikum Köln — Köln, Germany (Recruiting)
- Fabry disease center Mainz, Universitätsmedizin Mainz — Mainz, Germany (Recruiting)
- Fachinternistische Gemeinschaftspraxis, Müllheim — Müllheim, Germany (Recruiting)
- Fabry disease center Münster, Universitätsklinikum Münster — Münster, Germany (Recruiting)
- Fabry disease center Würzburg, Universitätsklinikum Würzburg — Würzburg, Germany (Recruiting)
Study contacts
- Principal investigator: Eva Brand, MD, PhD — Universitätsklinikum Münster
- Study coordinator: Eva Brand, MD, PhD
- Email: fabry-zentrum@ukmuenster.de
- Phone: +492518347518
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.