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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00633139
First received: February 29, 2008
Last updated: July 14, 2015
Last verified: February 2014

February 29, 2008
July 14, 2015
August 2007
September 2008   (final data collection date for primary outcome measure)
  • Relative Changes (%) in Gross Motor Function Measurement (GMFM) [ Time Frame: Baseline, 52 Weeks ] [ Designated as safety issue: No ]
    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.
  • Relative Change in Mullen's Scales of Early Learning [ Time Frame: Baseline, 52 Weeks ] [ Designated as safety issue: No ]
    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.
Relative change in GMFM after 52 weeks of treatment Absolute change in Mullen's Scales of Early Learning, after 52 weeks of treatment. [ Time Frame: 52 weeks of tratment ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00633139 on ClinicalTrials.gov Archive Site
Change in Cerebrospinal Fluid (CSF) Sulfatide [ Time Frame: Baseline, 52 Weeks ] [ Designated as safety issue: No ]
Changes in CSF sulfatide from baseline to end of study (Week 52). Data mentioned over mean represents the adjusted mean.
Change in CSF biomarker after 52 weeks of treatment with focus on reduction of sulfatid. [ Time Frame: After 52 weeks of treatment ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
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)

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.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Late Infantile Metachromatic Leukodystrophy
Biological: Recombinant human Arylsulfatase A (rhASA)
intravenous infusion, every other week for 26 weeks
Other Names:
  • Metazym
  • HGT-1111
  • Experimental: Cohort 1
    Cohort 1: 50 U/kg Recombinant human Arylsulfatase A (rhASA)
    Intervention: Biological: Recombinant human Arylsulfatase A (rhASA)
  • Experimental: Cohort 2
    Cohort 2: 100 U/kg Recombinant human Arylsulfatase A (rhASA)
    Intervention: Biological: Recombinant human Arylsulfatase A (rhASA)
  • Experimental: Cohort 3
    Cohort 3: 200 U/kg Recombinant human Arylsulfatase A (rhASA)
    Intervention: Biological: Recombinant human Arylsulfatase A (rhASA)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
13
September 2008
September 2008   (final data collection date for primary outcome measure)

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
Both
1 Year to 5 Years
No
Contact information is only displayed when the study is recruiting subjects
Denmark
 
NCT00633139
HGT-MLD-048, 2007-006345-40, 2006-005341-11
Yes
Shire
Shire
Not Provided
Principal Investigator: Allan M Lund, MD PhaseOne Trials A/S
Shire
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP