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

Chiesi

Status: Not yet enrolling

Conditions: Fabry Disease

Treatments: Drug: pegunigalsidase alfa

Study type: Observational

Funder types: Industry

Identifiers

NCT07109375

CHIT-2401

Details and patient eligibility

About

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.

Enrollment: 75 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

75 participants in 1 patient group

Fabry patients

Description:

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:

Drug: pegunigalsidase alfa