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Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT00633139
First received: February 29, 2008
Last updated: May 14, 2012
Last verified: May 2012
History of Changes
  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, NCT00418561). 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.


Condition Intervention Phase
Late Infantile Metachromatic Leukodystrophy
 Biological: Recombinant human Arylsulfatase A (rhASA)
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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:

MedlinePlus related topics: Leukodystrophies
U.S. FDA Resources

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

Primary Outcome Measures:
  • Relative Changes in Gross Motor Function Measurement (GMFM) [ Time Frame: 52 Weeks ] [ Designated as safety issue: No ]
    Changes in GMFM is measured from baseline to end of study (Week 52). GMFM is measured using GMFM-88. The GMFM-88 item scores can be summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score is between 0 (minimal) to 3 (maximum). The total GMFM-88 score is between 0 (minimal) to 264 (maximum). Relative changes in GMFM is calculated as percentage change from baseline divided by the age-difference between first and last visit. With GMFM score decreases over time, it indicates the disease deteriorated over time.

  • Relative Change in Mullen's Scales of Early Learning [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Changes in Mullen's Scales of Early Learning is measured from baseline to end of study (Week 52) using Mullen's Scales of Early Learning. T scores, percentile ranks, and age equivalents can be computed for the four scales separately (visual reception, fine motor, expressive language, and receptive language). Relative change is calculated as percentage change from baseline divided by the age-difference between first and last visit. With Mullen's score decreases over time, it indicates the disease deteriorated over time.


Secondary Outcome Measures:
  • Change in Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Changes in CSF sulfatide from baseline to end of study (Week 52)


Enrollment: 13
Study Start Date: August 2007
Study Completion Date: September 2008
Primary Completion Date: September 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Cohort 1
Cohort 1: 50 U/kg
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111
Cohort 2
Cohort 2: 100 U/kg
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111
Cohort 3
Cohort 3: 200 U/kg
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111

  Eligibility

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

Inclusion Criteria:

The patients from the Phase I trial must meet the following criteria to be enrolled in the study.

  • 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
PhaseOneTrials A/S
Hvidovre, Denmark, 2650
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
Principal Investigator: Allan M Lund, MD PhaseOne Trials A/S
  More Information

No publications provided

Responsible Party: Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier: NCT00633139     History of Changes
Other Study ID Numbers: HGT-MLD-048, 2007-006345-40, 2006-005341-11
Study First Received: February 29, 2008
Results First Received: October 6, 2010
Last Updated: May 14, 2012
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

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 May 19, 2013