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A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

This study has been completed.
Sponsor:
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00158600
First received: September 8, 2005
Last updated: April 6, 2015
Last verified: April 2015
Results First Received: June 24, 2010  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Pompe Disease (Late-onset)
Glycogen Storage Disease Type II (GSD-II)
Acid Maltase Deficiency Disease
Glycogenosis 2
Interventions: Biological: alglucosidase alfa
Drug: Placebo

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
One hundred patients screened and 90 enrolled.

Reporting Groups
  Description
Alglucosidase Alfa Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Participant Flow:   Overall Study
    Alglucosidase Alfa   Placebo
STARTED   60   30 
COMPLETED   55   26 
NOT COMPLETED   5   4 
unable to commit time to study                0                1 
Adverse Event                2                1 
Death                1                0 
Withdrawal by Subject                2                2 



  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 Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.
Total Total of all reporting groups

Baseline Measures
   Alglucosidase Alfa   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 60   30   90 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 45.3  (12.37)   42.6  (11.63)   44.4  (12.14) 
[1] Age at First Infusion
Gender 
[Units: Participants]
     
Female   26   19   45 
Male   34   11   45 
Race/Ethnicity, Customized 
[Units: Participants]
     
Hispanic   1   1   2 
Asian   1   1   2 
Black or African American   0   0   0 
White   57   27   84 
Unknown or not reported   1   1   2 


  Outcome Measures
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1.  Primary:   Summary of Patients Reporting Treatment-Emergent Adverse Events   [ Time Frame: weeks 0-78 ]
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Measure Type Primary
Measure Title Summary of Patients Reporting Treatment-Emergent Adverse Events
Measure Description Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo.
Time Frame weeks 0-78  
Safety Issue Yes  

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 patients who received any amount of study treatment comprise the safety population. Patients were considered, for safety analysis, to be in the treatment group of the treatment they actually received.

Missing or invalid safety or resource utilization data were not replaced.


Reporting Groups
  Description
Alglucosidase Alfa Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Measured Values
   Alglucosidase Alfa   Placebo 
Participants Analyzed 
[Units: Participants]
 60   30 
Summary of Patients Reporting Treatment-Emergent Adverse Events 
[Units: Participants]
   
Patients with Any AEs   60   30 
Patients with Treatment-Related AEs   32   17 
Patients with Infusion-Associated Reactions   17   7 
Patients with SAEs   13   6 
Patients with Severe AEs   14   10 
Patients who Discontinued Due to AEs (incl death)   3   1 
Patients who Died   1   0 

No statistical analysis provided for Summary of Patients Reporting Treatment-Emergent Adverse Events



2.  Primary:   Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline   [ Time Frame: weeks 0, 78 ]

3.  Primary:   Percent of Predicted Forced Vital Capacity (FVC)   [ Time Frame: weeks 0, 78 ]

4.  Primary:   Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC)   [ Time Frame: weeks 0, 12 and 52 ]

5.  Primary:   Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax)   [ Time Frame: weeks 0, 12, 52 ]

6.  Primary:   Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax)   [ Time Frame: weeks 0, 12, 52 ]

7.  Secondary:   Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT)   [ Time Frame: weeks 0, 78 ]

8.  Secondary:   Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey   [ Time Frame: weeks 0, 78 ]


  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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