Not Yet RecruitingNot Applicable

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

Italy75 participantsStarted 2026-04

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Assessment of structural abnormalities of the left ventricle

Timeframe: 24 months

Assessment of left ventricular diastolic function

Timeframe: 12 and 24 months

Assessment of renal function

Timeframe: 6, 12, 18 and 24 months

Trial details

NCT IDNCT07109375

SponsorChiesi Italia

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2028-12

Contact for this trial

Chiesi Clinical Trial info

+3905212791 clinicaltrials_info@chiesi.com

View on ClinicalTrials.gov More Fabry Disease trials