NCT00633139

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

A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 1 Year – 5 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Late Infantile Metachromatic Leukodystrophy

Interventions

Type	Name	Description
BIOLOGICAL	Recombinant human Arylsulfatase A (rhASA)	intravenous infusion, every other week for 26 weeks

Timeline

• Start date: 2007-01-22
• Primary completion: 2008-11-25
• Completion: 2008-11-25
• First posted: 2008-03-11
• Last updated: 2021-06-14
• Results posted: 2012-06-15

Locations

1 site across 1 country: Denmark