Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01288027
First received: January 27, 2011
Last updated: December 4, 2014
Last verified: December 2014
Results First Received: November 24, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Pompe Disease (Late-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: Alglucosidase Alfa

  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 11 centers between July 06, 2011 and December 19, 2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Alglucosidase Alfa Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.

Participant Flow:   Overall Study
    Alglucosidase Alfa  
STARTED     16  
Full Analysis Set (FAS)     16 [1]
COMPLETED     16  
NOT COMPLETED     0  
[1] All participants who received at least 1 complete infusion of alglucosidase alfa.



  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.
FAS population included all participants who received at least one complete infusion of alglucosidase alfa.

Reporting Groups
  Description
Alglucosidase Alfa Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.

Baseline Measures
    Alglucosidase Alfa  
Number of Participants  
[units: participants]
  16  
Age  
[units: years]
Mean (Standard Deviation)
  51.6  (13.69)  
Gender  
[units: participants]
 
Female     9  
Male     7  



  Outcome Measures
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1.  Primary:   Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26   [ Time Frame: Baseline, Week 26 ]

2.  Secondary:   Glycogen Distribution   [ Time Frame: Baseline, Week 26 ]

3.  Secondary:   Muscle Fiber Morphology   [ Time Frame: Baseline, Week 26 ]

4.  Secondary:   Lysosomal Inclusions   [ Time Frame: Baseline, Week 26 ]

5.  Secondary:   Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26   [ Time Frame: Baseline, Week 26 ]

6.  Secondary:   Percent Change From Baseline in Degree of Fatty Infiltration Using 3-Point 3-Dimensional (3D) Dixon at Week 26   [ Time Frame: Baseline, Week 26 ]

7.  Secondary:   Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26   [ Time Frame: Baseline, Week 26 ]


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


No publications provided


Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01288027     History of Changes
Other Study ID Numbers: AGLU07310, 2010-020611-36, MSC12823
Study First Received: January 27, 2011
Results First Received: November 24, 2014
Last Updated: December 4, 2014
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency