A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Chiesi Farmaceutici S.p.A.
Summary
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
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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) * Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs). * Cardiac Cohort: * Evidence of Fabry…
Interventions
- DrugPegunigalsidase-alfa
Administered via intravenous (IV) infusion under conditions of routine clinical care
Locations (10)
- University of Alabama at BirminghamBirmingham, Alabama
- Emory University School of MedicineAtlanta, Georgia
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois
- University of Iowa Hospitals and ClinicsIowa City, Iowa
- Infusion AssociatesGrand Rapids, Michigan
- Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)Fairfax, Virginia