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    Clinical Trial Results:
    An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

    Summary
    EudraCT number
    2015-000582-31
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    21 Jul 2010

    Results information
    Results version number
    v1(current)
    This version publication date
    23 May 2016
    First version publication date
    28 Jun 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AGLU03306
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00483379
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Genzyme Corporation
    Sponsor organisation address
    500 Kendall Street, Cambridge, United States, 02142
    Public contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    22 Oct 2010
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Jul 2010
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in subjects with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.
    Protection of trial subjects
    Paediatric subjects: The study was conducted by investigators experienced in the treatment of pediatric subjects. The parent(s) or guardian(s) as well as the children were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time. In addition to the consent form for the parent(s)/guardian(s), an assent form in child-appropriate language was provided and explained to the child. Repeated invasive procedures were minimized. The number of blood samples as well as the amount of blood drawn were adjusted according to age and weight. A topical anesthesia may have been used to minimize distress and discomfort. Adult Subjects: Subjects were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time in language and terms appropriate for the subject and considering the local culture. During the course of the trial, subjects were provided with individual subject cards indicating the nature of the trial the subject is participating, contact details and any information needed in the event of a medical emergency. Collected personal data and human biological samples were processed in compliance with the Sanofi-Aventis Group Personal Data Protection Charter ensuring that the Group abides by the laws governing personal data protection in force in all countries in which it operates.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    08 May 2007
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 11
    Country: Number of subjects enrolled
    Australia: 1
    Country: Number of subjects enrolled
    Canada: 1
    Worldwide total number of subjects
    13
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    1
    Children (2-11 years)
    5
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    4
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Fourteen subjects were screened and enrolled; however, one withdrew before receiving any study infusions due to the burden of weekly trips to the medical centre.

    Period 1
    Period 1 title
    Treatment Period
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Alglucosidase Alfa 20 mg/kg Every Week
    Arm description
    Subjects were treated with alglucosidase alfa every week for 52 weeks. This was the 'frequent dose' arm.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase alfa
    Investigational medicinal product code
    Other name
    Recombinant human acid glucosidase, Myozyme®
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    20 mg/kg/week

    Arm title
    Alglucosidase Alfa 40 mg/kg Every Other Week
    Arm description
    Subjects were treated with alglucosidase alfa every other week for 52 weeks. This was the 'high dose' arm.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase alfa
    Investigational medicinal product code
    Other name
    Recombinant human acid glucosidase, Myozyme®
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    40 mg/kg

    Number of subjects in period 1
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Started
    6
    7
    Completed
    4
    7
    Not completed
    2
    0
         Withdrawal by Subject
    1
    -
         'Adverse Event '
    1
    -
    Period 2
    Period 2 title
    Extension Period
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Alglucosidase Alfa 20 mg/kg Every Week
    Arm description
    Alglucosidase Alfa every Week until commercial supply became available.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase alfa
    Investigational medicinal product code
    Other name
    Recombinant human acid glucosidase, Myozyme
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    20 mg/kg

    Arm title
    Alglucosidase Alfa 40 mg/kg Every Other Week
    Arm description
    Alglucosidase Alfa every week until commercial supply became available.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase alfa
    Investigational medicinal product code
    Other name
    Recombinant human acid glucosidase, Myozyme®
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    40 mg/kg

    Number of subjects in period 2 [1]
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Started
    1
    2
    Completed
    1
    2
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Three subjects participated into the extension period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Alglucosidase Alfa 20 mg/kg Every Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every week for 52 weeks. This was the 'frequent dose' arm.

    Reporting group title
    Alglucosidase Alfa 40 mg/kg Every Other Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every other week for 52 weeks. This was the 'high dose' arm.

    Reporting group values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week Total
    Number of subjects
    6 7 13
    Age categorical
    Units: Subjects
        <18 years
    4 5 9
        >= 18 and <=65 years
    2 2 4
        >65 years
    0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    23.3 ± 27.75 16.8 ± 15.56 -
    Gender categorical
    Units: Subjects
        Female
    2 3 5
        Male
    4 4 8
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0
        Asian
    0 1 1
        Native Hawaiian or Other Pacific Islander
    0 0 0
        Black or African American
    0 0 0
        White
    6 6 12
        More than one race
    0 0 0
        Unknown or Not Reported
    0 0 0
    Life-stage of Disease Onset
    Units: Subjects
        Infantile-onset Pompe Disease
    4 5 9
        Late-onset Pompe Disease
    2 2 4
    Parameter in Clinical Decline
    Subject counts of the parameter in clinical decline (cardiac, respiratory or motor skills as compared to their condition prior to the beginning alglucosidase alfa treatment) for which subjects were included in the study.
    Units: Subjects
        Cardiac
    0 0 0
        Respiratory
    1 1 2
        Motor Skills
    5 6 11
    Cross-Reactive Immunologic Material (CRIM) Assay Result
    Units: Subjects
        Positive
    0 3 3
        Negative
    1 0 1
        Unknown
    5 4 9

    End points

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    End points reporting groups
    Reporting group title
    Alglucosidase Alfa 20 mg/kg Every Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every week for 52 weeks. This was the 'frequent dose' arm.

    Reporting group title
    Alglucosidase Alfa 40 mg/kg Every Other Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every other week for 52 weeks. This was the 'high dose' arm.
    Reporting group title
    Alglucosidase Alfa 20 mg/kg Every Week
    Reporting group description
    Alglucosidase Alfa every Week until commercial supply became available.

    Reporting group title
    Alglucosidase Alfa 40 mg/kg Every Other Week
    Reporting group description
    Alglucosidase Alfa every week until commercial supply became available.

    Primary: Subjects' Efficacy Response During the Treatment Period as Compared to Baseline for Subjects With Respiratory Decline on Standard Treatment

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    End point title
    Subjects' Efficacy Response During the Treatment Period as Compared to Baseline for Subjects With Respiratory Decline on Standard Treatment [1]
    End point description
    Subjects were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each subject was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each subject served as his or her own control. All subjects who enrolled due to decline in respiratory function while on standard treatment.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the analysis was descriptive, no statistical analysis is provided.
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    1
    1
    Units: Subjects
        Improved
    0
    0
        No change (on invasive ventilator for 24 hrs)
    0
    1
        Worsened
    0
    0
        Not evaluated
    1
    0
    No statistical analyses for this end point

    Primary: Subjects' Efficacy Response During the Treatment Period as Compared to Baseline for Subjects With Motor Function Decline on Standard Treatment

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    End point title
    Subjects' Efficacy Response During the Treatment Period as Compared to Baseline for Subjects With Motor Function Decline on Standard Treatment [2]
    End point description
    Subjects were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each subject was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Subjects could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each subject served as his or her own control. All subjects who enrolled due to decline in motor function while on standard treatment.
    End point type
    Primary
    End point timeframe
    Baseline, Week 52
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the analysis was descriptive, no statistical analysis is provided.
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    5
    6
    Units: subjects
        Gained gross or fine motor skills
    2
    4
        No change
    1
    2
        Continued motor loss
    1
    0
        Not evaluated
    1
    0
    No statistical analyses for this end point

    Primary: Summary of Subjects Reporting Treatment-Emergent Adverse Events During the Treatment Period

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    End point title
    Summary of Subjects Reporting Treatment-Emergent Adverse Events During the Treatment Period [3]
    End point description
    Overall safety summary of subjects 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. Safety population comprised of all subjects who received intervention.
    End point type
    Primary
    End point timeframe
    Day 1 up to Week 52
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the analysis was descriptive, no statistical analysis is provided.
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    6
    7
    Units: subjects
        Subjects with AEs
    6
    7
        Related AEs
    0
    2
        Not related AEs
    6
    7
        Mild AEs
    6
    6
        Moderate AEs
    3
    2
        Severe AEs
    2
    0
        AEs leading to discontinuation from study
    1
    0
        Deaths
    1
    0
        Infusion Associated Reactions
    0
    2
        Serious AEs
    2
    1
    No statistical analyses for this end point

    Secondary: Baseline Values for Left Ventricular Mass (LVM) Z-Scores

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    End point title
    Baseline Values for Left Ventricular Mass (LVM) Z-Scores
    End point description
    Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children’s Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist. Full analysis population of subjects with LVM data.
    End point type
    Secondary
    End point timeframe
    Day 0
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    5
    5
    Units: Z-score
        median (full range (min-max))
    0.3 (-1.2 to 6.3)
    -0.3 (-1.2 to 2.2)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52

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    End point title
    Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
    End point description
    Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children’s Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist. Full analysis population of subjects with LVM data at both timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    3
    3
    Units: Z-score
        median (full range (min-max))
    0.3 (-0.8 to 1.2)
    0.4 (-0.1 to 0.5)
    No statistical analyses for this end point

    Secondary: Baseline Values for Left Ventricular Mass Index (LVMI)

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    End point title
    Baseline Values for Left Ventricular Mass Index (LVMI)
    End point description
    Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the subject's body surface area in the calculation of LVMI. Full analysis population of subjects with LVMI data.
    End point type
    Secondary
    End point timeframe
    Day 0
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    5
    5
    Units: g/m^2
        median (full range (min-max))
    62.1 (49 to 187.3)
    56.5 (45.4 to 73.1)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52

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    End point title
    Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
    End point description
    Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the subject's body surface area in the calculation of LVMI. Full analysis population of subjects with LVMI data at both timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    3
    3
    Units: g/m^2
        median (full range (min-max))
    12.5 (-10.9 to 15.5)
    4 (-5.3 to 5.8)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)

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    End point title
    Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
    End point description
    The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support. Full analysis population. The subject in the worsened category died after week 52.
    End point type
    Secondary
    End point timeframe
    Baseline, approximately Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    6
    7
    Units: subjects
        Improved
    0
    0
        No change
    2
    3
        Worsened
    1
    0
        Did not use ventilator
    3
    4
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52

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    End point title
    Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
    End point description
    Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength. Full analysis population of subjects >= 8 years old. Due to the age restriction and small study population, the number of subjects analyzed is too small for results to be meaningful.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: subjects
    Notes
    [4] - Reason is provided in outcome description.
    [5] - Reason is provided in outcome description.
    No statistical analyses for this end point

    Secondary: Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results

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    End point title
    Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
    End point description
    The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions. Full analysis population.
    End point type
    Secondary
    End point timeframe
    Day 0
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    6
    6
    Units: units on a scale
        arithmetic mean (standard deviation)
    65 ± 60.52
    82.8 ± 84
    No statistical analyses for this end point

    Secondary: Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52

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    End point title
    Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
    End point description
    The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions. Full analysis population.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    4
    6
    Units: units on a scale
        arithmetic mean (standard deviation)
    6 ± 8.49
    6.7 ± 6.12
    No statistical analyses for this end point

    Secondary: Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)

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    End point title
    Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
    End point description
    The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability. Full analysis population.
    End point type
    Secondary
    End point timeframe
    Day 0
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    6
    7
    Units: units on a scale
        arithmetic mean (standard deviation)
    38.3 ± 20.94
    46.8 ± 21.26
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52

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    End point title
    Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
    End point description
    The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near “0” reflecting low capability and scores near “100” reflecting high capability. Full analysis population.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    4
    7
    Units: units on a scale
        arithmetic mean (standard deviation)
    0.6 ± 4.28
    3.5 ± 3.84
    No statistical analyses for this end point

    Secondary: Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)

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    End point title
    Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
    End point description
    Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for subjects ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life. Full analysis population of subjects >= 14 years old.
    End point type
    Secondary
    End point timeframe
    Day 0
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    2
    3
    Units: units on a scale
        arithmetic mean (standard deviation)
    31.3 ± 2.84
    36 ± 9.33
    No statistical analyses for this end point

    Secondary: Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52

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    End point title
    Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
    End point description
    Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for subjects ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life. Full analysis population of subjects >= 14 years old.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Alglucosidase Alfa 20 mg/kg Every Week Alglucosidase Alfa 40 mg/kg Every Other Week
    Number of subjects analysed
    1
    3
    Units: units on a scale
        arithmetic mean (standard deviation)
    2.5 ± 0
    4.4 ± 11.24
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment period AEs were collected up to week 52. Extension period AEs were collected following completion of the treatment period until the product was commercially available (up to week 118).
    Adverse event reporting additional description
    In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the subjects has been included in the numerator ("number of affected participants") of both adverse event tables. Events are listed independent of relationship to treatment reported.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    13.0
    Reporting groups
    Reporting group title
    Extension: Alglucosidase Alfa 20 mg/kg Every Week
    Reporting group description
    Alglucosidase Alfa every Week until commercial supply became available.

    Reporting group title
    Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every other week for 52 weeks. This was the 'high dose' arm.

    Reporting group title
    Treatment: Alglucosidase Alfa 20 mg/kg Every Week
    Reporting group description
    Subjects were treated with alglucosidase alfa every week for 52 weeks. This was the 'frequent dose' arm.

    Reporting group title
    Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
    Reporting group description
    Alglucosidase Alfa every week until commercial supply became available.

    Serious adverse events
    Extension: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Treatment: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 1 (100.00%)
    1 / 7 (14.29%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
    0
    1
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Fibula Fracture
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Weight Decreased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Supraventricular Tachycardia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Respiratory Failure
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    Gastrointestinal disorders
    Dysphagia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Device Related Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Extension: Alglucosidase Alfa 20 mg/kg Every Week Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week Treatment: Alglucosidase Alfa 20 mg/kg Every Week Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 1 (100.00%)
    7 / 7 (100.00%)
    6 / 6 (100.00%)
    1 / 2 (50.00%)
    Vascular disorders
    Blood Pressure Fluctuation
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypotension
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Adverse Drug Reaction
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Fatigue
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Asthenia
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Generalised Oedema
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 1 (0.00%)
    2 / 7 (28.57%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Injury, poisoning and procedural complications
    Arthropod Bite
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Contusion
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Epicondylitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Excoriation
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Fall
         subjects affected / exposed
    1 / 1 (100.00%)
    1 / 7 (14.29%)
    1 / 6 (16.67%)
    1 / 2 (50.00%)
         occurrences all number
    1
    2
    2
    1
    Muscle Strain
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vaccination Complication
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Investigations
    Aspiration Tracheal Abnormal
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood Calcium Increased
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Breath Sounds Abnormal
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Heart Rate Irregular
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Prostatic Specific Antigen Increased
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    White Blood Cells Urine Positive
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Protein Urine Present
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cardiac disorders
    Right Ventricular Hypertrophy
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tachycardia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Atelectasis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cough
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Dyspnoea
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    1 / 6 (16.67%)
    1 / 2 (50.00%)
         occurrences all number
    0
    3
    1
    1
    Increased Bronchial Secretion
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oropharyngeal Pain
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vasomotor Rhinitis
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Lymphadenitis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Lymphadenopathy
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Hypotonia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Headache
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    3
    0
    0
    Presyncope
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Restless Legs Syndrome
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Somnolence
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Tremor
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eye disorders
    Dry Eye
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Eyelid Ptosis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Ear and labyrinth disorders
    Hypoacusis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastrointestinal disorders
    Abdominal Discomfort
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Amalgam Tattoo
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Abdominal Pain Upper
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Anal Fissure
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Constipation
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Aphthous Stomatitis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    0 / 1 (0.00%)
    2 / 7 (28.57%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Haematochezia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nausea
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Renal and urinary disorders
    Pollakiuria
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Dermatitis Diaper
         subjects affected / exposed
    0 / 1 (0.00%)
    2 / 7 (28.57%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Eczema
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Erythema
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Rash
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Rash Erythematous
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    3
    0
    0
    Rash Macular
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Muscle Spasms
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Back Pain
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    2
    0
    Myalgia
         subjects affected / exposed
    0 / 1 (0.00%)
    2 / 7 (28.57%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Pain In Extremity
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Gout
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypocalcaemia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypokalaemia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hypomagnesaemia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Infections and infestations
    Abscess Limb
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Acute Sinusitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cellulitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Candidiasis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ear Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Device Related Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Fungal Skin Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastroenteritis Viral
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Gastrointestinal Viral Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hordeolum
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Influenza
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Otitis Media Acute
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Otitis Media
         subjects affected / exposed
    0 / 1 (0.00%)
    2 / 7 (28.57%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    3
    0
    0
    Pneumococcal Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    4
    0
    Sinusitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Respiratory Tract Infection Viral
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory Tract Infection Bacterial
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Rash Pustular
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Tonsillitis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Tooth Abscess
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Subcutaneous Abscess
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 7 (0.00%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    3 / 7 (42.86%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    4
    3
    0
    Streptococcal Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Urinary Tract Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    1 / 6 (16.67%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Varicella
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 7 (14.29%)
    0 / 6 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Viral Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 7 (0.00%)
    2 / 6 (33.33%)
    0 / 2 (0.00%)
         occurrences all number
    0
    0
    2
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Jan 2007
    Changed age stratification for randomization from 16 years to 18 years. Added assessment of the Pompe PEDI for all patients and the MOS SF-36 for subjects ≥14 years of age. Scope of echocardiogram (ECHO) assessments was reduced to focus on key measurements, including LVM, LVMI, left posterior wall thickness, shortening fraction, and ejection fraction.
    02 Oct 2007
    Exclude subjects negative for Cross-Reactive Immunologic Material (CRIM), to remove the analysis of results by CRIM status, and to increase the enrollment limit from 12 to 14 because 1 of the subjects enrolled prior to the amendment was found to be CRIM. Physician’s global assessment of the subject’s clinical status by organ system was removed. GMFM-88 was replaced by the GMFM-66. Risk section of introduction was updated. Revised text to emphasize that written informed consent must be obtained before randomization. Added screening/baseline assessment of retrospective, related AEs that occurred during previous clinical study and/or commercial Myozyme treatment.
    11 Oct 2008
    The pre-specified end date for enrollment was removed.
    28 Apr 2009
    Added an extension period to the study after the 52-week study period to allow late-onset subjects access to treatment until commercial alglucosidase alfa became available.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    This small exploratory study lacked a parallel control arm at the standard dose for a longer period; decline in respiratory or motor function prior to study was not collected systematically, thus change from baseline observations are inconclusive.
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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