A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
ConditionFabry Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorChiesi Farmaceutici S.p.A.
About this trial
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
Eligibility criteria
Qualifiers
Male or female aged > 18 years of age at the time of consent.
Genetically confirmed diagnosis of Fabry disease.
Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease.
No contraindications for cardiac magnetic resonance imaging (cMRI)
Disqualifiers
Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication.
Pregnant at the time of enrolment.
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.
Active participation in any interventional study for Fabry disease
Trial design
Treatments tested in this trial
- Pegunigalsidase-alfa
Treatment groups
100 Participants
are divided into 3 treatment groupsLocations
10Slovenia
General Hospital Slovenj Gradec2380, Slovenj Gradec Slovenia
United Kingdom
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth HospitalB152TH, EdgbastonBirmingham, United Kingdom
Salford RoyalM6 8HD, SalfordGreater Manchester, United Kingdom
The Royal Free HospitalNW3 2QG, London United Kingdom
Sponsors and collaborators
Chiesi Farmaceutici S.p.A.
Lead sponsor
Source ClinicalTrials.gov