Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Phase 1/2 Completed 13 enrolled Shire

Non-randomizedParallel-groupOpen-labelTreatment

Summary

This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561). All patients will be offered continuing treatment in this study and will in this protocol receive 13 infusions, whereby the patients total have had 27 infusions of Metazym. One infusion will be given every other week. After a total of 52 weeks of treatment the subjects will continue treatment in a compassionate use protocol. Safety (AE/SAE) will be monitored at every visit.

Timeline

Start: 2007-01-22
Primary completion: 2008-11-25
Completion: 2008-11-25

Outcome

Missed primary endpoint

paper Motor function and developmental testing scores declined during the study in all dose groups; no significant differences were observed between groups. PMID 33332761 ↗

paper There was no evidence of efficacy in preventing motor and cognitive deterioration PMID 33332761 ↗

Publications

I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Arylsulfatase A	Protein / enzyme biologic	50 iu/kg	Intravenous
Subject	Arylsulfatase A	Protein / enzyme biologic	100 iu/kg	Intravenous
Subject	Arylsulfatase A	Protein / enzyme biologic	200 iu/kg	Intravenous