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
This trial does not accept healthy volunteersQualifiers
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 population
The eligible population for this study includes participants who have a genetically confirmed diagnosis of Fabry disease and are being treated or plan to initiate treatment with pegunigalsidase alfa (Elfabrio®). Participants will be required to meet inclusion, not meet exclusion criteria and sign informed consent to be enrolled in the study.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Pegunigalsidase-alfa
DrugAdministered via intravenous (IV) infusion under conditions of routine clinical care
Treatment groups
100 Participants
are divided into 3 treatment groupsGroup A: Cardiac Cohort1 intervention
Group B: Naïve Cohort1 intervention
Group C: Long-Term Cohort1 intervention
Trial outcomes
Primary outcomes
1
Estimated Glomerular Filtration Rate (eGFR)
eGFR, calculated using the Chronic Kidney Disease - Epidemiology Collaboration (eGFRCKD-EPI) equation from study baseline and from pegunigalsidase alfa treatment start to end of follow-up and to pre-specified time points (annually).
Time frame
4 years2
Plasma Globotriaosylsphingosine (LysoGb3) Concentration
Levels of the Fabry disease biomarker plasma globotriaosylsphingosine (lyso-Gb3) over time, and change from study baseline and from pegunigalsidase alfa treatment start to end of follow-up.
Time frame
4 years3
Left Ventricular Mass Index (LVMI; g/m2)
Change in LVMI over time as assessed by cardiac MRI. Based on LVM indexed by height and/or body surface area.
Time frame
4 years4
High Sensitivity Troponin (hs-cTnT)
Change in levels of hs-cTnT over time
Time frame
4 yearsSponsors and contacts
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Source ClinicalTrials.gov