Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD - Full Text View

Purpose

This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048, defined as the completion of all Week 52 procedures. This group of patients will be offered ongoing treatment with HGT-1111 in this protocol. One infusion will be given every other week until the product is commercially available, the patient discontinues, or the study is terminated by the Sponsor, provided no safety issues have emerged.

Condition	Intervention	Phase
Late Infantile Metachromatic Leukodystrophy	Drug: HGT-1111	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

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 Schindler disease 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:

The objective is to supply patients who have participated in the clinical development program of HGT-1111 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 HGT-1111 treatment in patients with late infantile MLD. [ Time Frame: Until the product is available for sale, market or the project is terminated ] [ Designated as safety issue: No ]

Enrollment:	11
Study Start Date:	February 2008
Study Completion Date:	October 2010
Primary Completion Date:	October 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: HGT-1111 100 U/kg	Drug: HGT-1111 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. Other Name: rhASA, Metazym
Experimental: HGT-1111 200 U/kg	Drug: HGT-1111 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. Other Name: rhASA, Metazym

Arms

Assigned Interventions

Experimental: HGT-1111 100 U/kg

Drug: HGT-1111

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.

Other Name: rhASA, Metazym

Experimental: HGT-1111 200 U/kg

Drug: HGT-1111

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.

Other Name: rhASA, Metazym

Detailed Description:

The primary objective of this study is to provide ongoing treatment of HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03 - EudraCT: 2007-006345-40) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged. The secondary objective of this study is to monitor disease progression and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048.

Eligibility

Ages Eligible for Study:	3 Years to 6 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)
Completion of study HGT-MLD-048
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
Rigshospitalet
Copenhagen, Hvidovre, Denmark, 2650

Sponsors and Collaborators

Shire Human Genetic Therapies, Inc.

Investigators

Principal Investigator:

Christine Dali, MD

Rigshospitalet, Denmark

More Information

No publications provided

Responsible Party:	Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:	NCT00681811 History of Changes
Other Study ID Numbers:	HGT-MLD-049, 2008-000084-41
Study First Received:	May 19, 2008
Last Updated:	March 16, 2012
Health Authority:	Denmark: Danish Dataprotection Agency Denmark: Danish Medicines Agency Denmark: The Regional Committee on Biomedical Research Ethics United States: Food and Drug Administration

Keywords provided by Shire Human Genetic Therapies, Inc.:

Metazym
Late Infantile Metachromatic Leukodystrophy

Additional relevant MeSH terms:

Leukodystrophy, Metachromatic
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Sulfatidosis
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System

Leukoencephalopathies
Demyelinating Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 18, 2013