Clinical Trials in Italy / NCT07109375
Recruiting
Observational
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
NCT07109375 · tracked via the Priya Life Science Italy tracker
Condition(s) studied
Fabry Disease
Investigational drug(s) / intervention(s)
pegunigalsidase alfa: Pegunigalsidase alfa is 2 mg/mL concentrate for solution and is administered via intravenous infusion every two weeks.
Study summary
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.
Eligibility
Inclusion Criteria:
1. Male and female adults (≥ 18 years).
2. Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene.
3. Patients either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. The treatment decision must be made independently from participation in this study.
4. Written informed consent to participate in the study and for the processing of personal data.
Exclusion Criteria:
1. History of hypersensitivity reaction to pegunigalsidase alfa.
2. Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
3. Female subjects who are pregnant or planning to become pregnant during the study.
Primary outcome measure(s)
- Assessment of structural abnormalities of the left ventricle — 24 months
Changes from baseline in structural abnormalities of the left ventricle assessed by echocardiography and defined by the presence of Left Ventricular Hypertrophy. - Assessment of left ventricular diastolic function — 12 and 24 months
Changes from baseline in left ventricular diastolic function by echocardiography. Diastolic dysfunction will be assessed using early to late diastolic trans-mitral flow velocity. - Assessment of renal function — 6, 12, 18 and 24 months
Changes from baseline in renal function by estimated Glomerular Filtration Rate.
Trial sites (7)
| Facility | City | Region | Status |
|---|---|---|---|
| AUSL Toscana Sud-Est - Ospedale San Donato | Arezzo | Italy | Not Yet Recruiting |
| Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari | Bari | Italy | Recruiting |
| Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola | Bologna | Italy | Recruiting |
| A.O. Sant'Anna e San Sebastiano | Caserta | Italy | Not Yet Recruiting |
| U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo | Monza | Italy | Not Yet Recruiting |
| AOU Federico II, Dipartimento di Nefrologia | Naples | Italy | Not Yet Recruiting |
| A.O.U. Policlinico "Giaccone" | Palermo | Italy | Not Yet Recruiting |
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07109375 on ClinicalTrials.gov ↗ ← All trials in Italy