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A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

This study has been terminated.
(Study was stopped due to approved label expansion of alglucosidase alfa.)
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01597596
First received: May 10, 2012
Last updated: December 14, 2015
Last verified: December 2015
Results First Received: September 25, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Pompe Disease (Infantile-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenosis 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 between 21 August 2012 and 1 December 2014.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 5 participants were screened and 4 participants were treated.

Reporting Groups
  Description
Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase alfa (4000 L material) 20 mg/kg intravenous (IV) infusion every other week (QOW) for 52 weeks.
Alglucosidase Alfa 160 L Material (US Participants) Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.

Participant Flow:   Overall Study
    Alglucosidase Alfa 4000 L Material (Non-US Participants)     Alglucosidase Alfa 160 L Material (US Participants)  
STARTED     1     3  
COMPLETED     0     2  
NOT COMPLETED     1     1  
Study terminated by sponsor                 1                 0  
Physician Decision                 0                 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.
No text entered.

Reporting Groups
  Description
Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Alglucosidase Alfa 160 L Material (US Participants) Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Total Total of all reporting groups

Baseline Measures
    Alglucosidase Alfa 4000 L Material (Non-US Participants)     Alglucosidase Alfa 160 L Material (US Participants)     Total  
Number of Participants  
[units: participants]
  1     3     4  
Age  
[units: years]
Mean (Standard Deviation)
  0.3     0.5  (0.28)     0.4  (0.14)  
Gender  
[units: participants]
     
Female     0     0     0  
Male     1     3     4  



  Outcome Measures
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1.  Primary:   Change From Baseline in Cardiac Function at Week 52   [ Time Frame: Baseline, Week 52 ]

2.  Secondary:   Percentage of Participants With Estimated Probability of Survival   [ Time Frame: Up to Week 52 ]

3.  Secondary:   Number of Participants With Invasive Ventilator-Free Survival   [ Time Frame: Up to Week 52 ]

4.  Secondary:   Change From Baseline in Motor Development Status at Week 52   [ Time Frame: Baseline, Week 52 ]


  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



Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01597596     History of Changes
Other Study ID Numbers: AGLU07510
2011-005595-42 ( EudraCT Number )
Study First Received: May 10, 2012
Results First Received: September 25, 2015
Last Updated: December 14, 2015
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency