Pegunigalsidase Alfa in Fabry Disease: Real‑World Use

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study collects prospective data on adults with Fabry disease who are treated with pegunigalsidase alfa or are planning to start it, capturing safety and effectiveness in real-world settings. No experimental interventions beyond routine treatment are required. The study aims to describe how the therapy performs outside controlled trials, including long-term outcomes and any adverse events. Enrollment is limited to adults able to give informed consent, with sites in Italy and collaboration with local healthcare facilities. The decision to treat with pegunigalsidase alfa remains at the physician’s discretion and is independent of participation in the study.

Why it matters

Who can join: the full rules

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.

Where it's running

• Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari
  Bari, Italy Recruiting
• Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola
  Bologna, Italy Recruiting
• AUSL Toscana Sud-Est - Ospedale San Donato
  Arezzo, Italy Not yet 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

Study contacts

• Principal investigator: Antonio Pisani, MD — AOU Federico II, Dipartimento di Nefrologia, Napoli, Italy
• Study coordinator: Chiesi Clinical Trial info
• Email: clinicaltrials_info@chiesi.com
• Phone: +3905212791

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.