A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00140621
First received: August 30, 2005
Last updated: April 16, 2015
Last verified: April 2015
Results First Received: April 1, 2015  
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: Drug: Agalsidase beta

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was conducted at 7 centers between July 06, 2005 and August 6, 2012. One participant was treated at 2 study sites because the participant had to be transferred to another site during the 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
Agalsidase Beta (Fabrazyme [Recombinant Form]) Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.

Participant Flow:   Overall Study
    Agalsidase Beta (Fabrazyme [Recombinant Form])  
STARTED     6  
COMPLETED     6  
NOT COMPLETED     0  



  Baseline Characteristics
  Hide Baseline Characteristics

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.
Efficacy evaluable population (EEP) included all registered participants except those who had serious deviations from protocol compliance requirements, did not receive study drug, had no data available at baseline or after study drug administration, and who were found to have no condition in cardiac Fabry disease.

Reporting Groups
  Description
Agalsidase Beta Agalsidase beta 1 mg/kg intravenously once every 2 weeks up to 156 weeks.

Baseline Measures
    Agalsidase Beta  
Number of Participants  
[units: participants]
  6  
Age  
[units: years]
Mean (Standard Deviation)
  53.8  (5.7)  
Gender  
[units: participants]
 
Female     5  
Male     1  
Weight  
[units: kilograms¬†(kg)]
Mean (Standard Deviation)
  58.50  (9.24)  
Height  
[units: centimeters¬†(cm)]
Mean (Standard Deviation)
  156.63  (7.20)  
Left Ventricular Hypertrophy  
[units: participants]
 
No     0  
Yes     6  
Globotriaosylceramide (GL-3) Accumulation in the Myocardium  
[units: participants]
 
No     0  
Yes     1  
Not Measured     5  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156   [ Time Frame: Baseline to Week 156 ]

2.  Primary:   Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156   [ Time Frame: Baseline to Week 156 ]

3.  Primary:   Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156   [ Time Frame: Baseline to Week 156 ]

4.  Primary:   Change From Baseline in LVM at Week 156   [ Time Frame: Baseline to Week 156 ]

5.  Secondary:   Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test   [ Time Frame: Baseline to Week 156 ]

6.  Secondary:   Percent Change From Baseline in GL-3 Plasma Levels at Week 156   [ Time Frame: Baseline to Week 156 ]

7.  Secondary:   Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156   [ Time Frame: Baseline to Week 156 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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 text entered.


  More Information
  Hide More Information

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: Trial Transparency Team
Organization: Sanofi
e-mail: Contact-US@Sanofi.com


No publications provided


Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00140621     History of Changes
Other Study ID Numbers: AGAL03204
Study First Received: August 30, 2005
Results First Received: April 1, 2015
Last Updated: April 16, 2015
Health Authority: Japan: Ministry of Health, Labor and Welfare