Long-Term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) - Tabular View

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

This study is ongoing, but not recruiting participants.
Information provided by Zymenex A/S

This Tabular View shows the required WHO registration data elements as marked by ^†

Descriptive Information Fields

Brief Title ^†

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

Official Title ^†

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

Brief 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). 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. The efficacy observed during the first 26 weeks of treatment in the phase I trial (EudraCT number: 2006-005341-11) will be studied further during totally 1 year of treatment. The myelin recovery (CNS and PNS) and functional capacity will be evaluated after this long-term treatment.

Detailed Description

Study Phase

Phase II

Study Type ^†

Interventional

Study Design ^†

Treatment, Non-Randomized, Open Label, Uncontrolled, Factorial Assignment, Efficacy Study

Primary Outcome Measure ^†

Relative change in GMFM after 52 weeks of treatment Absolute change in Mullen's Scales of Early Learning, after 52 weeks of treatment. [ Time Frame: 52 weeks of tratment ] [ Designated as safety issue: No ]

Secondary Outcome Measure ^†

Change in CSF biomarker after 52 weeks of treatment with focus on reduction of sulfatid. [ Time Frame: After 52 weeks of treatment ] [ Designated as safety issue: No ]

Condition ^†

Late Infantile Metachromatic Leukodystrophy

Intervention ^†

Drug: Metazym

MEDLINE PMIDs

Links

Sponsors homepage This link exits the ClinicalTrials.gov site

Recruitment Information Fields

Recruitment Status ^†

Active, not recruiting

Enrollment ^†

Start Date ^†

February 2008

Completion Date

December 2008

Eligibility Criteria ^†

Inclusion Criteria:

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

Gender

Both

Ages

1 Year to 5 Years

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

Denmark

Administrative Information Fields

NCT ID ^†

NCT00633139

Organization ID

rhASA-03

Secondary IDs ^††

rhASA-03, EudraCT number: 2007-006345-40

Study Sponsor ^†

Zymenex A/S

Collaborators ^††

Investigators ^†

Principal Investigator:

Allan M Lund, MD

Rigshospitalet, Denmark

Information Provided By

Zymenex A/S

Verification Date

February 2008

First Received Date ^†

February 29, 2008

Last Updated Date

February 29, 2008

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.