A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00196716
First received: September 12, 2005
Last updated: August 11, 2009
Last verified: April 2007
Results First Received: December 5, 2008  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy 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 Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months.

Participant Flow for 2 periods

Period 1:   1.0 mg/kg Period
    Fabrazyme  
STARTED     21  
COMPLETED     21  
NOT COMPLETED     0  

Period 2:   0.3 mg/kg Period
    Fabrazyme  
STARTED     21  
COMPLETED     21  
NOT COMPLETED     0  



  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.
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Reporting Groups
  Description
Fabrazyme Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months.

Baseline Measures
    Fabrazyme  
Number of Participants  
[units: participants]
  21  
Age  
[units: years]
Mean ± Standard Deviation
  34.0  ± 10.0  
Gender  
[units: Participants]
 
Female     0  
Male     21  
Race/Ethnicity, Customized  
[units: Participants]
 
Caucasian     21  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Globotriaosylceramide (GL-3) Clearance in Kidney Interstitial Capillary Endothelium   [ Time Frame: Throughout study; 96 weeks ]

2.  Secondary:   Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium   [ Time Frame: Throughout study ; 96 weeks ]

3.  Secondary:   Estimated Glomerular Filtration Rate (eGFR)   [ Time Frame: Throughout study; 96 weeks ]

4.  Secondary:   Plasma Globotriaosylceramide (GL-3)   [ Time Frame: Throughout study; 96 weeks ]

5.  Secondary:   Urine Globotriaosylceramide (GL-3)   [ Time Frame: Throughout study, 96 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 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 MedInfo
Organization: Genzyme Corporation
phone: 800-745-4447
e-mail: medinfo@genzyme.com


No publications provided


Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00196716     History of Changes
Other Study ID Numbers: AGAL-017-01
Study First Received: September 12, 2005
Results First Received: December 5, 2008
Last Updated: August 11, 2009
Health Authority: Estonia: The State Agency of Medicine
Czech Republic: State Institute for Drug Control
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Slovakia: State Institute for Drug Control