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

This study has been completed.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: February 29, 2008
Last updated: February 19, 2014
Last verified: February 2014
Results First Received: October 6, 2010  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Biological: Recombinant human Arylsulfatase A (rhASA)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
After written approval from the Independent Ethics Committee (IEC) and competent authority has been obtained, the Investigator obtained informed consent from the subject’s legally acceptable representative.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Not applicable.

Reporting Groups
Cohort 1 Cohort 1: 25 U/kg as a single dose - thereafter 50 U/kg
Cohort 2 Cohort 2: 100 U/kg
Cohort 3 Cohort 3: 200 U/kg

Participant Flow:   Overall Study
    Cohort 1     Cohort 2     Cohort 3  
STARTED     4     5     4  
COMPLETED     4     4     3  
NOT COMPLETED     0     1     1  

  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
Cohort 1 Cohort 1: 25 U/kg as a single dose - thereafter 50 U/kg
Cohort 2 Cohort 2: 100 U/kg
Cohort 3 Cohort 3: 200 U/kg
Total Total of all reporting groups

Baseline Measures
    Cohort 1     Cohort 2     Cohort 3     Total  
Number of Participants  
[units: participants]
  4     5     4     13  
[units: participants]
<=18 years     4     5     4     13  
Between 18 and 65 years     0     0     0     0  
>=65 years     0     0     0     0  
[units: months]
Mean (Standard Deviation)
  36.25  (9.32)     41.80  (10.13)     30.75  (7.27)     36.69  (9.59)  
[units: participants]
Female     2     3     3     8  
Male     2     2     1     5  
Region of Enrollment  
[units: participants]
Denmark     4     5     4     13  

  Outcome Measures
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1.  Primary:   Relative Changes (%) in Gross Motor Function Measurement (GMFM)   [ Time Frame: baseline to 52 Weeks ]

2.  Primary:   Relative Change in Mullen's Scales of Early Learning   [ Time Frame: 52 weeks ]

3.  Secondary:   Change in Cerebrospinal Fluid (CSF) Sulfatide   [ Time Frame: 52 weeks ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact:  
Name/Title: Norman Barton
Organization: Shire Human Genetic Therapies, Inc
phone: 781-482-9297
e-mail: nbarton@shire.com

No publications provided

Responsible Party: Shire
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: February 19, 2014
Health Authority: Denmark: Danish Dataprotection Agency
Denmark: Danish Medicines Agency
Denmark: Ethics Committee