Compassionate Use of Metazym in Patients With Late Infantile Metachromatic Leukodystrophy - Full Text View

Sponsored by:	Shire Human Genetic Therapies, Inc.
Information provided by:	Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:	NCT00681811

Purpose

This is an open-label study of patients with late infantile MLD who have participated in clinical trials with Metazym. For ethical reasons treatment of these patients must continue after the clinical trials have ended. This group of patients will be offered ongoing treatment in this protocol. One infusion will be given every other week given no safety concerns have emerged until the product is available for sale, market or the development program is terminated (e.g. for safety reasons). Safety (vital signs, antibodies and AE/SAE) will be monitored at every visit.

Condition	Intervention	Phase
Late Infantile Metachromatic Leukodystrophy	Drug: rhASA	Phase III

Genetics Home Reference related topics: cholesteryl ester storage disease Farber lipogranulomatosis leukoencephalopathy with vanishing white matter long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy mitochondrial trifunctional protein deficiency primary carnitine deficiency

MedlinePlus related topics: Leukodystrophies

Drug Information available for: Sodium chloride Chlorides

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Open Label, Uncontrolled, Factorial Assignment, Safety/Efficacy Study
Official Title:	A Compassionate Use Open-Label Study of Metazym (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

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

Primary Outcome Measures:

The objective is to supply patients who have participated in the clinical development program of Metazym with treatment. [ Time Frame: Until the product is available for sale, market or the project is terminated ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

The secondary objective of this study objective is ongoing evaluation of disease progress and safety profile of Metazym treatment in patients with late infantile MLD. [ Time Frame: Until Metazym is available for sale, market or the project is terminated ] [ Designated as safety issue: No ]

Estimated Enrollment:	18
Study Start Date:	October 2008
Estimated Study Completion Date:	June 2010
Estimated Primary Completion Date:	March 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
rhASA: Experimental	Drug: rhASA Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients dosed with 50 U/kg will be equally randomized to treatment on 100 U/kg or 200 U/kg. The dose will be adjusted every 6-week to account for changes in body weight.The infusion length will be dependent on the dose. Infusion of 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.

Arms

Assigned Interventions

rhASA: Experimental

Drug: rhASA

Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients dosed with 50 U/kg will be equally randomized to treatment on 100 U/kg or 200 U/kg. The dose will be adjusted every 6-week to account for changes in body weight.The infusion length will be dependent on the dose. Infusion of 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.

Detailed Description:

The overall objective is to supply patients who have participated in the clinical development program of Metazym (rhASA-03 - EudraCT: 2007-006345-40 or rhASA-04 - EudraCT: 2007-007165-20) with treatment from end of the trials given no safety concerns have emerged and until the product is available for sale, market or the project is terminated.The secondary objective of this study objective is ongoing evaluation of disease progress and safety profile of Metazym treatment in patients with late infantile MLD.

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)
Previous treated with Metazym
The subject and his/her guardian(s) must have the ability to comply with the 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00681811

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

Rigshospitalet, Denmark

More Information

Responsible Party:	Shire HGT ( Carol Cannon (U.S.) or Steve Moloney (EU) )
Study ID Numbers:	rhASA-05, EudraCT # 2008-000084-41, rhASA-05
Study First Received:	May 19, 2008
Last Updated:	December 3, 2008
ClinicalTrials.gov Identifier:	NCT00681811
Health Authority:	Denmark: Danish Dataprotection Agency; Denmark: Danish Medicines Agency; Denmark: The Regional Committee on Biomedical Research Ethics

Keywords provided by Shire Human Genetic Therapies, Inc.:

Compassionate use
Metazym
Late Infantile Metachromatic Leukodystrophy

Study placed in the following topic categories:

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

Leukodystrophy
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metachromatic leukodystrophy
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lysosomal Storage Diseases, Nervous System
Nervous System Diseases
Sulfatidosis
Hereditary Central Nervous System Demyelinating Diseases

ClinicalTrials.gov processed this record on January 14, 2009