A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01650779
First received: July 24, 2012
Last updated: June 9, 2014
Last verified: June 2014
Results First Received: June 9, 2014  
Study Type: Interventional
Study Design: 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
The study was conducted at 6 centers in the United States of America between April 30, 2012 and March 15, 2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 16 participants were screened of which 1 participant was screen failure. A total of 15 participants were enrolled in this study.

Reporting Groups
  Description
Agalsidase Beta Commercially available agalsidase beta (Fabrazyme ®) 1.0 milligram per kilogram (mg/kg) administered as an intravenous infusion every 2 weeks (q2w) up to Month 6.

Participant Flow:   Overall Study
    Agalsidase Beta  
STARTED     15  
COMPLETED     14  
NOT COMPLETED     1  
Withdrawal by Subject                 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.
All enrolled participants were included in the analysis.

Reporting Groups
  Description
Agalsidase Beta Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.

Baseline Measures
    Agalsidase Beta  
Number of Participants  
[units: participants]
  15  
Age  
[units: years]
Mean ± Standard Deviation
  28.5  ± 16.11  
Gender  
[units: participants]
 
Female     0  
Male     15  
Race/Ethnicity, Customized  
[units: participants]
 
Caucasian     13  
Hispanic     1  
Other     1  
Duration of Fabry Disease [1]
[units: years]
Median ( Full Range )
  9.3  
  ( 2 to 19 )  
Method of Diagnosis of Fabry Disease [2]
[units: participants]
 
Genotype     14  
Leukocyte alfa-galactosidase A (GAL) activity     11  
Plasma alfa-GAL activity     9  
[1] Duration of Fabry disease was calculated from date of initial diagnosis of Fabry disease to date of the first study infusion. Number of participants analyzed for this baseline measure were 14, as one participant did not have information on date of initial diagnosis of Fabry disease.
[2] Number of participants with each method (genotype, leukocyte alfa-galactosidase A [GAL] activity, and plasma alfa-GAL activity) are reported. Some participants were diagnosed by more than 1 method, hence reported under more than 1 category.



  Outcome Measures
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1.  Primary:   Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6   [ Time Frame: Baseline, Month 2, 4, 6 ]

2.  Secondary:   Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6   [ Time Frame: Baseline, Month 2, 4, 6 ]

3.  Secondary:   Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6   [ Time Frame: Baseline, Month 2, 4, 6 ]

4.  Secondary:   Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6   [ Time Frame: Baseline, Month 2, 4, 6 ]


  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
This is considered to be an exploratory study for the following reasons: it was based on a small number of participants and has been designed as an open-label, single-arm study as opposed to a two-arm crossover design.


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


No publications provided


Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01650779     History of Changes
Other Study ID Numbers: AGAL19412
Study First Received: July 24, 2012
Results First Received: June 9, 2014
Last Updated: June 9, 2014
Health Authority: United States: Food and Drug Administration