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

This study has been terminated.
(Terminated prior to planned completion date due to lack of efficacy.)
Sponsor:
ClinicalTrials.gov Identifier:
NCT00681811
First Posted: May 21, 2008
Last Update Posted: August 5, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Shire
  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 (NCT00633139), 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
Intervention Model: Parallel Assignment
Masking: None (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:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Days of Exposure to HGT-1111 [ Time Frame: Baseline until end of study (Week 139) ]
    End of study was defined as until HGT-1111 was commercially available, the participant's participation was discontinued, or the study was terminated by the Sponsor.


Secondary Outcome Measures:
  • Level of Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: Baseline until end of study (Week 139) ]
    Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).

  • Level of White Matter Metabolites [ Time Frame: Baseline until end of study (Week 139) ]
    Level of white matter metabolites [N-acetyl Aspartate (NAA)] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).

  • Score of Gross Motor Function Measurement (GMFM) [ Time Frame: Baseline until end of study (Week 139) ]
    Gross motor function was measured using GMFM-88 at 6-month intervals. The GMFM-88 item scores were summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) to 264 (maximum). Decrease in GMFM score indicates disease progression.


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

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 - NCT00633139) 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 (NCT00633139).
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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)
  2. Completion of study HGT-MLD-048 (NCT00633139)
  3. The subject and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria:

  1. Spasticity so severe to inhibit transportation
  2. 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
  3. 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00681811


Locations
Denmark
Rigshospitalet
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Shire
Investigators
Principal Investigator: Christine Dali, MD Rigshospitalet, Denmark
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00681811     History of Changes
Other Study ID Numbers: HGT-MLD-049
2008-000084-41 ( EudraCT Number )
First Submitted: May 19, 2008
First Posted: May 21, 2008
Results First Submitted: September 26, 2012
Results First Posted: November 8, 2013
Last Update Posted: August 5, 2015
Last Verified: February 2014

Keywords provided by Shire:
Metazyme
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