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:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00681811
First received: May 19, 2008
Last updated: August 4, 2015
Last verified: February 2014
Results First Received: September 26, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Drug: HGT-1111

  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
Children with an established diagnosis of late metachromatic leukodystrophy (MLD) due to arylsulfatase A (ASA) deficiency were recruited.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All participants who completed Study HGT-MLD-048 (NCT00633139) participated in this study.

Reporting Groups
  Description
100 U/kg HGT-1111 Participants received 100 units per kilogram (U/kg) of HGT1111 intravenous (IV) infusion every other week.
200 U/kg HGT-1111 Participants received 200 U/kg of HGT1111 IV infusion every other week.

Participant Flow:   Overall Study
    100 U/kg HGT-1111     200 U/kg HGT-1111  
STARTED     6     5  
COMPLETED     3     1  
NOT COMPLETED     3     4  
Adverse Event                 1                 1  
Withdrawal by Subject                 2                 2  
Non-compliance                 0                 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
  Description
100 U/kg HGT1111 Participants received 100 U/kg of HGT1111 IV infusion every other week.
200 U/kg Participants received 200 U/kg of HGT1111 IV infusion every other week.
Total Total of all reporting groups

Baseline Measures
    100 U/kg HGT1111     200 U/kg     Total  
Number of Participants  
[units: participants]
  6     5     11  
Age  
[units: participants]
     
<=18 years     6     5     11  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age [1]
[units: months]
Mean (Standard Deviation)
  53.14  (10.526)     49.99  (8.752)     51.71  (9.420)  
Gender  
[units: participants]
     
Female     3     3     6  
Male     3     2     5  
Region of Enrollment  
[units: participants]
     
Denmark     6     5     11  
[1] Age at Week 54 or Week 56 in HGT-MLD-049 (NCT00681811)



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Days of Exposure to HGT-1111   [ Time Frame: Baseline until end of study (Week 139) ]

2.  Secondary:   Level of Cerebrospinal Fluid (CSF) Sulfatide   [ Time Frame: Baseline until end of study (Week 139) ]

3.  Secondary:   Level of White Matter Metabolites   [ Time Frame: Baseline until end of study (Week 139) ]

4.  Secondary:   Score of Gross Motor Function Measurement (GMFM)   [ Time Frame: Baseline until end of study (Week 139) ]


  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
Early termination due to lack of efficacy.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI 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: NCT00681811     History of Changes
Other Study ID Numbers: HGT-MLD-049, 2008-000084-41
Study First Received: May 19, 2008
Results First Received: September 26, 2012
Last Updated: August 4, 2015
Health Authority: Denmark: Danish Dataprotection Agency
Denmark: Danish Medicines Agency
Denmark: The Regional Committee on Biomedical Research Ethics
United States: Food and Drug Administration