Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
ConditionFabry Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorChiesi Italia
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
Adults patients who have a genetically confirmed diagnosis of Fabry disease and are being treated or plan to initiate treatment with pegunigalsidase alfa as per clinical practice.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
pegunigalsidase alfa
DrugPegunigalsidase alfa is 2 mg/mL concentrate for solution and is administered via intravenous infusion every two weeks.
Treatment groups
75 Participants
are divided into 1 treatment groupGroup A: Fabry patients1 intervention
Trial outcomes
Primary outcomes
1
Assessment of structural abnormalities of the left ventricle
Changes from baseline in structural abnormalities of the left ventricle assessed by echocardiography and defined by the presence of Left Ventricular Hypertrophy.
Time frame
24 months2
Assessment of left ventricular diastolic function
Changes from baseline in left ventricular diastolic function by echocardiography. Diastolic dysfunction will be assessed using early to late diastolic trans-mitral flow velocity.
Time frame
12 and 24 months3
Assessment of renal function
Changes from baseline in renal function by estimated Glomerular Filtration Rate.
Time frame
6, 12, 18 and 24 monthsSecondary outcomes
1
Assessment of peak oxygen uptake (pVO2)
Changes from baseline in peak oxygen uptake (pVO2) using cardiopulmonary exercise testing (CPET).
Time frame
24 months2
Assessment of carbon dioxide production (VCO2)
Changes from baseline in carbon dioxide production (VCO2) using cardiopulmonary exercise testing (CPET).
Time frame
24 months3
Assessment of NT-pro-BNP and high sensitivity cardiac troponin levels
Changes from baseline in NT-pro-BNP and high sensitivity cardiac troponin levels.
Time frame
6, 12,18 and 24 months4
Assessment of Cardiovascular Magnetic Resonance (CMR)
Changes from baseline in CMR assessed using T1 mapping to achieve myocardial tissue characterization.
Time frame
12 and 24 monthsSponsors and contacts
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Source ClinicalTrials.gov