NCT06095713Active, not recruiting
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Source: ClinicalTrials.gov · Last updated 30 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 60 (actual)
- First submitted
- 10 Oct 2023
- Start date
- 1 Oct 2023
- Primary completion date
- 30 Sept 2027
- Lead sponsor
- Universität Münster
- Diseases
- Fabry disease
- NCT number
- NCT06095713
What you commit to
- Study type: Observational
- Participants enrolled: 60
- From 1 Oct 2023 to the primary completion date, 30 Sept 2027
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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.
Other
- Patient is unwilling to give informed consent.
- Patient is unable to comply with the clinical protocol.
- Patients on dialysis.
Who the study is looking for
- 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.
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Fabry disease center Berlin - Charité - Universitätsmedizin Berlin Coordinator of this site? Confirm its status | Berlin | Germany | Unknown status |
| Fabry disease center Cologne, Universitätsklinikum Köln Coordinator of this site? Confirm its status | Cologne | Germany | Unknown status |
| Fabry disease center Hamburg, Universitätsklinikum Hamburg Coordinator of this site? Confirm its status | Hamburg | Germany | Unknown status |
| Fabry disease center Hannover, Universitätsklinikum Hannover Coordinator of this site? Confirm its status | Hanover | Germany | Unknown status |
| Fabry disease center Mainz, Universitätsmedizin Mainz Coordinator of this site? Confirm its status | Mainz | Germany | Unknown status |
| Fachinternistische Gemeinschaftspraxis, Müllheim Coordinator of this site? Confirm its status | Müllheim | Germany | Unknown status |
| Fabry disease center Münster, Universitätsklinikum Münster Coordinator of this site? Confirm its status | Münster | Germany | Unknown status |
| Fabry disease center Würzburg, Universitätsklinikum Würzburg Coordinator of this site? Confirm its status | Würzburg | Germany | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.