Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD. Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timeframe: From study drug administration up to Week 28
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26
Timeframe: Baseline, Week 26
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26
Timeframe: Baseline, Week 26
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine
Timeframe: Baseline up to Week 26
Change From Baseline in Mullen's Scales of Early Learning at Week 26
Timeframe: Baseline, Week 26
Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA)
Timeframe: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8
Arylsulfatase A (ASA) Activity in Leukocytes
Timeframe: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26