At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
In Brief
A Phase 1 clinical trial evaluating rhASA - Dose Level 1, rhASA - Dose Level 2, and 1 other intervention for Metachromatic Leukodystrophy (MLD). Completed, enrolled 13 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
Intravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks
Intravenous infusion 100 U/kg every other week for 26 weeks
Intravenous infusion 200 U/kg every other week for 26 weeks