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

Purpose

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.

Condition	Intervention	Phase
Late Infantile Metachromatic Leukodystrophy	Drug: Metazym	Phase II

Genetics Home Reference related topics:

metachromatic leukodystrophy

MedlinePlus related topics:

Leukodystrophies

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Factorial Assignment, Efficacy Study

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

Further study details as provided by Zymenex A/S:

Primary Outcome Measures:

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 Measures:

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 ]

Enrollment:	12
Study Start Date:	February 2008
Estimated Study Completion Date:	December 2008
Estimated Primary Completion Date:	September 2008 (Final data collection date for primary outcome measure)

Intervention Details:

The three dose levels will be given as repeated doses. Patients in each cohort will receive one dose of enzyme every other week. Dosing will be performed as follows: Cohort 1: 50 U/kg, Cohort 2: 100 U/kg and Cohort 3: 200 U/kg. After a total of 52 weeks the subjects will continue treatment in a compassionate use protocol. Safety (AE/SAE) will be monitored at every visit. The dose will be adjusted monthly to account for changes in body weight.

Eligibility

Ages Eligible for Study:	1 Year to 5 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00633139

Locations


Denmark, Hvidovre
	PhaseOneTrials A/S
	Copenhagen, Hvidovre, Denmark, 2650

Sponsors and Collaborators

Zymenex A/S

Investigators


Principal Investigator:	Allan M Lund, MD	Rigshospitalet, Denmark

More Information

Sponsors homepage This link exits the ClinicalTrials.gov site

Responsible Party:	Zymenex ( Jens Fogh )
Study ID Numbers:	rhASA-03, rhASA-03, EudraCT number: 2007-006345-40
First Received:	February 29, 2008
Last Updated:	February 29, 2008
ClinicalTrials.gov Identifier:	NCT00633139
Health Authority:	Denmark: Danish Dataprotection Agency; Denmark: Danish Medicines Agency; Denmark: Ethics Committee

Keywords provided by Zymenex A/S:

Metazym

Late infantile

Metachromatic leukodystrophy

Long-term safety

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors

Sphingolipidoses

Metabolic Diseases

Leukodystrophy, Metachromatic

Demyelinating Diseases

Lysosomal Storage Diseases

Sphingolipidosis

Central Nervous System Diseases

Demyelinating diseases

Brain Diseases

Leukodystrophy

Metabolism, Inborn Errors

Genetic Diseases, Inborn

Infant, Newborn, Diseases

Metachromatic leukodystrophy

Brain Diseases, Metabolic, Inborn

Lipidoses

Metabolic disorder

Congenital Abnormalities

Lipid Metabolism Disorders

Additional relevant MeSH terms:

Lysosomal Storage Diseases, Nervous System

Nervous System Diseases

Sulfatidosis

Nutritional and Metabolic Diseases

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Hereditary Central Nervous System Demyelinating Diseases

ClinicalTrials.gov processed this record on May 08, 2008

Sponsored by:	Zymenex A/S
Information provided by:	Zymenex A/S
ClinicalTrials.gov Identifier:	NCT00633139