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

This study has been completed.
Sponsor:
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00081497
First received: April 14, 2004
Last updated: August 18, 2010
Last verified: August 2010
Results First Received: December 17, 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: 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
Only those patients who successfully completed the AGAL-008-00 (NCT00074984) trial were eligible for participation in the AGAL02503 (NCT00081497) extension study.

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
Placebo/Fabrazyme Patients who had been randomized to placebo during AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry to AGAL02503 (NCT00081497).
Fabrazyme/Fabrazyme Patients who had been randomized to Fabrazyme in AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry into AGAL02503 (NCT00081497).

Participant Flow:   Overall Study
    Placebo/Fabrazyme     Fabrazyme/Fabrazyme  
STARTED     28 [1]   39 [2]
COMPLETED     25     37  
NOT COMPLETED     3     2  
Adverse Event                 2                 0  
Death                 0                 1  
Withdrawal by Subject                 1                 0  
Non-compliance                 0                 1  
[1] Patients who were randomized to placebo in AGAL-008-00 (NCT00074984).
[2] Patients who were randomized to Fabrazyme in AGAL-008-00 (NCT00074984).



  Baseline Characteristics
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Reporting Groups
  Description
Placebo/Fabrazyme Patients who had been randomized to placebo during AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry to AGAL02503 (NCT00081497).
Fabrazyme/Fabrazyme Patients who had been randomized to Fabrazyme in AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry into AGAL02503 (NCT00081497).
Total Total of all reporting groups

Baseline Measures
    Placebo/Fabrazyme     Fabrazyme/Fabrazyme     Total  
Number of Participants  
[units: participants]
  28     39     67  
Age, Customized  
[units: participants]
     
<40 years     8     7     15  
≥40 years     20     32     52  
Age  
[units: years]
Mean ± Standard Deviation
  43.5  ± 9.31     46.6  ± 9.72     45.3  ± 9.60  
Gender  
[units: participants]
     
Female     4     5     9  
Male     24     34     58  
Race/Ethnicity  
[units: participants]
     
Caucasian     24     35     59  
Black     0     0     0  
Hispanic     2     3     5  
Asian     1     1     2  
Other     1     0     1  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods   [ Time Frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) ]

2.  Secondary:   Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months   [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]

3.  Secondary:   Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months   [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]

4.  Secondary:   Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months   [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]

5.  Secondary:   Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months   [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]

6.  Post-Hoc:   Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2.   [ Time Frame: Throughout study; 18 months ]


  Serious Adverse Events


  Other Adverse Events


  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.


Limitations and Caveats
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
No valid conclusions can be made from the predefined primary efficacy analysis. The sponsor believes that it is more statistically appropriate to compare placebo patients with Fabrazyme patients as they were randomized in the original Phase 4 trial.  


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


No publications provided


Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00081497     History of Changes
Other Study ID Numbers: AGAL02503
Study First Received: April 14, 2004
Results First Received: December 17, 2008
Last Updated: August 18, 2010
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Hungary: National Institute of Pharmacy
Czech Republic: State Institute for Drug Control
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United Kingdom: Medicines and Healthcare Products Regulatory Agency