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

Sponsored by:	Shire Human Genetic Therapies, Inc.
Information provided by:	Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:	NCT00633139

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: rhASA	Phase II

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

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Leukodystrophies

U.S. FDA Resources

Further study details as provided by Shire Human Genetic Therapies, Inc.:

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
Study Completion Date:	September 2008
Primary Completion Date:	September 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
rhASA: Experimental metazym	Drug: rhASA intravenous infusion 50U/kg, 100U/kg or 200U/kg every other week for 26 weeks

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

Shire Human Genetic Therapies, Inc.

Investigators

Principal Investigator:

Christine i Dali, MD

PhaseOne Trials A/S

More Information

No publications provided

Responsible Party:	Shire HGT ( Carol Cannon (US) or Steve Moloney (EU) )
Study ID Numbers:	HGT-MLD-048, rhASA-03, EudraCT number: 2007-006345-40
Study First Received:	February 29, 2008
Last Updated:	December 3, 2008
ClinicalTrials.gov Identifier:	NCT00633139 History of Changes
Health Authority:	Denmark: Danish Dataprotection Agency; Denmark: Danish Medicines Agency; Denmark: Ethics Committee

Keywords provided by Shire Human Genetic Therapies, Inc.:

Metazym
Late infantile
Metachromatic leukodystrophy
Long-term safety

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Metachromatic Leukodystrophy
Sphingolipidoses
Leukodystrophy, Metachromatic
Metabolic Diseases
Demyelinating Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases

Brain Diseases
Leukodystrophy
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Leukodystrophy, Metachromatic
Demyelinating Diseases
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Sulfatidosis

Central Nervous System Diseases
Brain Diseases
Hereditary Central Nervous System Demyelinating Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Brain Diseases, Metabolic, Inborn
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on July 02, 2009