A Study of Fabrazyme in Pediatric Patients With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00074958
First received: December 24, 2003
Last updated: March 13, 2015
Last verified: March 2015
Results First Received: March 3, 2009  
Study Type: Interventional
Study Design: Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fabry Disease
Intervention: Biological: Fabrazyme (agalsidase beta)

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Fabrazyme 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.

Participant Flow:   Overall Study
    Fabrazyme  
STARTED     16  
COMPLETED     15  
NOT COMPLETED     1  
Adverse Event                 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
Fabrazyme 1.0 mg/kg of Fabrazyme given to the patients every 2 weeks.

Baseline Measures
    Fabrazyme  
Number of Participants  
[units: participants]
  16  
Age  
[units: years]
Mean (Standard Deviation)
  12.1  (2.52)  
Gender  
[units: participants]
 
Female     2  
Male     14  



  Outcome Measures
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1.  Primary:   Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin   [ Time Frame: Baseline, Week 24 and Week 48 ]

2.  Secondary:   Plasma GL-3   [ Time Frame: Baseline, Week 24 and Week 48 ]


  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
The trial was not powered to demonstrate clinical benefit.


  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 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 more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00074958     History of Changes
Other Study ID Numbers: AGAL-016-01
Study First Received: December 24, 2003
Results First Received: March 3, 2009
Last Updated: March 13, 2015
Health Authority: United States: Food and Drug Administration