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.
Study Type
OBSERVATIONAL
Enrollment
100
Administered via intravenous (IV) infusion under conditions of routine clinical care
University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGEmory University School of Medicine
Atlanta, Georgia, United States
RECRUITINGEstimated 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 years
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 years
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 years
High Sensitivity Troponin (hs-cTnT)
Change in levels of hs-cTnT over time
Time frame: 4 years
Safety Assessments
The endpoints will include occurrence of SAEs, infusion-related reactions (IRRs), drug-related adverse events, and proportion of participants with ADAs over time.
Time frame: 4 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
RECRUITINGUniversity of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
RECRUITINGInfusion Associates
Grand Rapids, Michigan, United States
RECRUITINGLysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
Fairfax, Virginia, United States
RECRUITINGGeneral Hospital Slovenj Gradec
Slovenj Gradec, Slovenia
RECRUITINGUniversity Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston, Birmingham, United Kingdom
RECRUITINGSalford Royal
Salford, Greater Manchester, United Kingdom
RECRUITINGThe Royal Free Hospital
London, United Kingdom
RECRUITING