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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-01-22

Study Completion Date

2008-11-25

Brief Summary

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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.

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Detailed Description

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Conditions

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Late Infantile Metachromatic Leukodystrophy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Cohort 1: 50 U/kg Recombinant human Arylsulfatase A (rhASA)

Group Type EXPERIMENTAL

Recombinant human Arylsulfatase A (rhASA)

Intervention Type BIOLOGICAL

intravenous infusion, every other week for 26 weeks

Cohort 2

Cohort 2: 100 U/kg Recombinant human Arylsulfatase A (rhASA)

Group Type EXPERIMENTAL

Recombinant human Arylsulfatase A (rhASA)

Intervention Type BIOLOGICAL

intravenous infusion, every other week for 26 weeks

Cohort 3

Cohort 3: 200 U/kg Recombinant human Arylsulfatase A (rhASA)

Group Type EXPERIMENTAL

Recombinant human Arylsulfatase A (rhASA)

Intervention Type BIOLOGICAL

intravenous infusion, every other week for 26 weeks

Interventions

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Recombinant human Arylsulfatase A (rhASA)

intravenous infusion, every other week for 26 weeks

Intervention Type BIOLOGICAL

Other Intervention Names

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Metazym HGT-1111

Eligibility Criteria

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Inclusion Criteria

The patients from the Phase I trial must meet the following criteria to be enrolled in the study.

* Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
* The subject and his/her guardian(s) must have the ability to comply with the clinical protocol

Exclusion Criteria

* Spasticity so severe to inhibit transportation
* Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
* Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
* Use of any investigational product other than rhASA within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations

Minimum Eligible Age

1 Year

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No