Observes long-term pegunigalsidase alfa use in adults with Fabry disease
Follows adults with Fabry disease who take pegunigalsidase alfa in everyday care.
Key facts
- Who runs it
- Chiesi Italia
- Where
- Arezzo, Bari, Bologna, Caserta, Monza, Naples, and Palermo, ItalySee the 7 sites
- Trial code
- Registry title
- Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
- Status
- Recruiting
- People taking part
- 75 people, planned
- Started
- 15 Jun 2026
- Expected to end
- Feb 2029
- Type of study
- Observational
- Treatments tested
- pegunigalsidase alfa
- Record updated
- 8 July 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Fabry disease who are taking, or plan to take, pegunigalsidase alfa. The treatment decision is made separately from the study.
- Age
- 18 years or older
- Sex
- Men and women can join.
- Disease stage
- Fabry disease confirmed by an α-Gal A assay and a mutation found in the α-Gal A gene.
- Earlier treatment
- People must be taking pegunigalsidase alfa or planning to take it. The decision to treat must be made independently of this study.
- Biomarker or test result
- Fabry disease confirmed by an α-Gal A assay and a mutation in the α-Gal A gene.
Main reasons for not taking part
- Past hypersensitivity reaction to pegunigalsidase alfa
- Any medical, emotional, behavioural or psychological condition that the physician judges could interfere with taking part
- Women who are pregnant or planning to become pregnant during the study
The registry criteria, word for word
Show all 7 criteriaShow less
Who can take part
- Male and female adults (≥ 18 years)
- Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene
- 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
- Written informed consent to participate in the study and for the processing of personal data
Who cannot take part
- History of hypersensitivity reaction to pegunigalsidase alfa
- 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
- Female subjects who are pregnant or planning to become pregnant during the study
Where in Italy
- AUSL Toscana Sud-Est - Ospedale San DonatoArezzo, ItalyContact: Maurizio Pieroni, MDNot yet recruiting
- Azienda Ospedaliera Universitaria Consorziale Policlinico di BariBari, ItalyContact: GesualdoTaking people
- IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'OrsolaBologna, ItalyContact: La MannaTaking people
- A.O. Sant'Anna e San SebastianoCaserta, ItalyContact: Paolo CalabròNot yet recruiting
- U.O.C. Nefrologia ASST Monza - Ospedale San GerardoMonza, ItalyContact: Federico PieruzziNot yet recruiting
- AOU Federico II, Dipartimento di NefrologiaNaples, ItalyContact: Antonio Pisani, MDNot yet recruiting
- A.O.U. Policlinico GiacconePalermo, ItalyContact: Antonino Tuttolomondo, MDNot yet recruiting
About the trial
What the trial wants to find out
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.
The group in the trial
- Fabry patients
Adult patients whit genetically confirmed diagnosis of Fabry disease being treated or planning to start treatment with Pegunigalsidase alfa according to clinical practice. Participants will be required to meet the inclusion and exclusion criteria and sign informed consent to be enrolled in the study.
Treatment: pegunigalsidase alfa
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Observational Prospective Cohort Study on Long-term Effective and Safe Use of Pegunigalsidase Alfa in Adult Fabry Patients Under "Real-world" Setting
- Plain-language title
- Observes long-term pegunigalsidase alfa use in adults with Fabry diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 8 July 2026