In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of participants with Fabry Disease. Participants will receive one Replagal infusion every other week for up to 104 weeks. They will visit the clinic every 12 to 14 weeks during treatment with a follow-up visit 2 weeks after treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
17
Participants will receive REPLAGAL 0.2 mg/kg body weight of IV infusion for 104 weeks.
Change From Baseline in Renal Function at Week 104
Renal function was planned to be assessed by estimated glomerular filtration rate (eGFR) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. The eGFR was planned to be calculated by CKD-EPI formula: eGFR = 141 x min (Serum Creatinine \[Scr\]/κ,1)\^(α) x max(Scr/κ,1)\^(-1.209) x 0.993\^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr was serum creatinine (mg/dL); κ was 0.7 for females and 0.9 for males; α was -0.329 for females and -0.411 for males; min indicated the minimum of Scr/κ or 1; max indicated the maximum of Scr /κ or 1. Change from baseline in renal function at Week 104 was planned to be reported.
Time frame: Baseline, Week 104
Change From Baseline in Cardiac Structure at Week 104
Cardiac structure was planned to be assessed by left ventricular mass index (LVMI) using cardiac magnetic resonance imaging (cMRI). Change from baseline in cardiac structure at Week 104 was planned to be reported.
Time frame: Baseline, Week 104
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) up to Week 104
Annualized rate of change in eGFR up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Annualized Rate of Change in Left Ventricular Mass Index (LVMI) up to Week 104
Annualized rate of change in LVMI up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Change From Baseline in eGFR up to Week 104
Change from baseline in eGFR up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Change From Baseline in LVMI up to Week 104
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M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Canada
Queen Elizabeth II Health Sciences Center
Halifax, Canada
Turun Yliopistollinen Keskussairaala
Turku, Finland
Vaasan Keskussairaala
Vaasa, Finland
Charité - Universitätsklinikum
Berlin, Germany
SphinCS
Höchheim, Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany
Fachinternistische Gemeinschaftspraxis
Müllheim, Germany
Universitaetsklinikum Wuerzburg
Würzburg, Germany
Laiko General Hospital of Athens
Athens, Greece
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Change from baseline in LVMI up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Change From Baseline in Proteinuria up to Week 104
Proteinuria was to be measured based on protein/creatinine ratio (PCR). Change from baseline in proteinuria up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Change From Baseline in Cardiac Fibrotic Segments up to Week 104
Change from baseline in cardiac fibrotic segments suggestive of cardiac fibrosis up to Week 104 was planned to be assessed by volume of fibrosis, measured by cMRI.
Time frame: From Baseline up to Week 104
Change From Baseline in Interventricular Septal End-Diastolic Thickness and Posterior Wall Thickness in Diastole up to Week 104
Change from baseline in interventricular septal end-diastolic thickness and posterior wall thickness in diastole up to Week 104 was planned to be measured by cMRI.
Time frame: From Baseline up to Week 104
Change From Baseline in Plasma Globotriaosylsphingosine (Lyso-Gb3) up to Week 104
Change from baseline in lyso-Gb3 up to Week 104 was planned to be reported.
Time frame: From Baseline up to Week 104
Number of Participants With Adverse Events (AEs)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
Time frame: From start of study drug administration up to follow-up visit (i.e., up to Week 106)
Number of Participants Who Will Develop Anti-drug Antibodies (ADA) to REPLAGAL
Number of participants who will develop ADA to REPLAGAL was planned to be reported.
Time frame: From Baseline up to Week 104