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

NCT00633139 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2021-06-14

Study results available
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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

BIOLOGICAL

Recombinant human Arylsulfatase A (rhASA)

intravenous infusion, every other week for 26 weeks

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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

Countries

  • Denmark

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00633139 on ClinicalTrials.gov