The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
31
Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks
Powder for concentrate for solution for infusion 0.5 mg/kg/2 weeks
Unnamed facility
Decatur, Georgia, United States
Unnamed facility
Cincinnati, Ohio, United States
Unnamed facility
Seattle, Washington, United States
Unnamed facility
Buenos Aires, Argentina
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium
Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
Time frame: Baseline, Week 52, Week 156 and Week 260
Percent Change From Baseline in GL-3 Clearance From Plasma
Plasma samples were assayed for GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal plasma GL-3 level of 7.0 μg/mL. Number of participants analyzed=participants with both baseline and post-baseline GL-3 plasma clearance assessment. Here 'n' signifies number of participants with available data for specified category.
Time frame: Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260
Percent Change From Baseline in GL-3 Clearance From Urine
Plasma samples were assayed for total urine GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal of \<0.030 mg/mmoL of creatinine. Number of participants analyzed=participants with both baseline and post-baseline GL-3 urine clearance assessment. Here 'n' signifies number of participants with available data for specified category.
Time frame: Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260
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Unnamed facility
Passo Fundo, Rio Grande do Sul, Brazil
Unnamed facility
Vancouver, British Columbia, Canada
Unnamed facility
Montreal, QC, Canada
Unnamed facility
Prague, Czechia
Unnamed facility
Amsterdam, Netherlands
Unnamed facility
Bergen, Norway
...and 2 more locations