Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Status:
Completed
Trial end date:
0000-00-00
Target enrollment:
Participant gender:
Summary
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.