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Recruiting NCT07109375

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

— Observational Fabry Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: pegunigalsidase alfa.
Who it may be relevant to
Registry conditions: Fabry Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Italy
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Observational Prospective Cohort Study on Long-term Effective and Safe Use of Pegunigalsidase Alfa in Adult Fabry Patients Under "Real-world" Setting

Overview

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.

Interventions

  • Drug pegunigalsidase alfa
    Pegunigalsidase alfa is 2 mg/mL concentrate for solution and is administered via intravenous infusion every two weeks.

Primary outcome measures

  • Assessment of structural abnormalities of the left ventricle [Time frame: 24 months]
  • Assessment of left ventricular diastolic function [Time frame: 12 and 24 months]
  • Assessment of renal function [Time frame: 6, 12, 18 and 24 months]
Secondary outcome measures (12)
  • Assessment of peak oxygen uptake (pVO2) [Time frame: 24 months]
  • Assessment of carbon dioxide production (VCO2) [Time frame: 24 months]
  • Assessment of NT-pro-BNP and high sensitivity cardiac troponin levels [Time frame: 6, 12,18 and 24 months]
  • Assessment of Cardiovascular Magnetic Resonance (CMR) [Time frame: 12 and 24 months]
  • Assessment of proteinuria and microalbuminuria [Time frame: 6, 12, 18 and 24 months]
  • Assessment of Globotriaosylsphingosine (lyso-Gb3) [Time frame: 3, 6, 12 and 24 months]
  • Assessment of QoL outcomes using the Short Form Health Survey 36 (SF-36) [Time frame: 12 and 24 months]
  • Assessment of QoL outcomes using the Kansas City Cardiomyopathy Questionnaire (KCCQ) [Time frame: 12 and 24 months]
  • Assessment of severity of neuropathic pain using Chronic Pain Grade (CPG) [Time frame: 12 and 24 months]
  • Assessment of severity of neuropathic pain using Short Form of the Brief Pain Inventory (BPI-SF) [Time frame: 12 and 24 months]
  • Assessment of gastrointestinal symptoms using the Gastrointestinal Symptoms Rating Scale (GSRS) [Time frame: 12 and 24 months]
  • Assessment of specific Fabry disease ocular findings [Time frame: Baseline, 12 and 24 months]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Italy · 7 centers
  • AUSL Toscana Sud-Est - Ospedale San Donato — Arezzo
  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari — Bari
  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola — Bologna
  • A.O. Sant'Anna e San Sebastiano — Caserta
  • U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo — Monza
  • AOU Federico II, Dipartimento di Nefrologia — Naples
  • A.O.U. Policlinico "Giaccone" — Palermo

Identifiers

NCT: NCT07109375 · CHIT-2401

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗