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Trial record 3 of 13 for:    metachromatic leukodystrophy | Completed Studies
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Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

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ClinicalTrials.gov Identifier: NCT00633139
Recruitment Status : Completed
First Posted : March 11, 2008
Results First Posted : June 15, 2012
Last Update Posted : June 14, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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Brief Summary:
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 or disease Intervention/treatment Phase
Late Infantile Metachromatic Leukodystrophy Biological: Recombinant human Arylsulfatase A (rhASA) Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Actual Study Start Date : January 22, 2007
Actual Primary Completion Date : November 25, 2008
Actual Study Completion Date : November 25, 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Leukodystrophies

Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1
Cohort 1: 50 U/kg Recombinant human Arylsulfatase A (rhASA)
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111
Experimental: Cohort 2
Cohort 2: 100 U/kg Recombinant human Arylsulfatase A (rhASA)
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111
Experimental: Cohort 3
Cohort 3: 200 U/kg Recombinant human Arylsulfatase A (rhASA)
 Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111


Outcome Measures
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Primary Outcome Measures :
  1. Relative Changes (%) in Gross Motor Function Measurement (GMFM) [ Time Frame: Baseline, 52 Weeks ]
    Change (percent change) 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 are calculated as percentage change from baseline divided by the age difference in months between first and last visit. The GMFM score decreases over time, which, indicates that the disease worsened over time. Score over time (SOT), data mentioned over mean represents the adjusted mean.

  2. Relative Change in Mullen's Scales of Early Learning [ Time Frame: Baseline, 52 Weeks ]
    Changes in Mullen's Scales of Early Learning are 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 in months between first and last visit. When Mullen's score decreases over time, it indicates the disease worsened over time. Data mentioned over mean represents the adjusted mean.


Secondary Outcome Measures :
  1. Change in Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: Baseline, 52 Weeks ]
    Changes in CSF sulfatide from baseline to end of study (Week 52). Data mentioned over mean represents the adjusted mean.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 5 Years   (Child)
Sexes Eligible for Study:   All
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
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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): NCT00633139


Locations
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Denmark
PhaseOneTrials A/S
Hvidovre, Denmark, 2650
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Takeda
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00633139    
Other Study ID Numbers: HGT-MLD-048
2007-006345-40 ( EudraCT Number )
First Posted: March 11, 2008    Key Record Dates
Results First Posted: June 15, 2012
Last Update Posted: June 14, 2021
Last Verified: May 2021
Keywords provided by Takeda ( Shire ):
Metazym
Late infantile
Metachromatic leukodystrophy
Long-term safety
Additional relevant MeSH terms:
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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