MedTrace Logo

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

NCT00418561 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2021-06-25

Study results available
· View outcomes & findings →

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.

Conditions

  • Metachromatic Leukodystrophy (MLD)

Interventions

BIOLOGICAL

rhASA - Dose Level 1

Intravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks

BIOLOGICAL

rhASA - Dose Level 2

Intravenous infusion 100 U/kg every other week for 26 weeks

BIOLOGICAL

rhASA - Dose Level 3

Intravenous infusion 200 U/kg 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
FACTORIAL

Eligibility

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

Timeline & Regulatory

Start
2007-01-22
Primary Completion
2008-03-27
Completion
2008-03-27

Countries

  • Denmark

Study Locations

More Related Trials

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 NCT00418561 on ClinicalTrials.gov