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    Clinical Trial Results:
    A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients with Infantile-Onset Pompe Disease

    Summary
    EudraCT number
    2011-005595-42
    Trial protocol
    DE  
    Global end of trial date
    01 Dec 2014

    Results information
    Results version number
    v1
    This version publication date
    04 Jul 2016
    First version publication date
    12 Jun 2015
    Other versions
    v2

    Trial information

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    Trial identification
    Sponsor protocol code
    AGLU07510/EFC12722
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01597596
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Genzyme Corporation
    Sponsor organisation address
    500 Kendall Street, Cambridge, MA, 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
    26 Jan 2015
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Dec 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective was to demonstrate the noninferiority of alglucosidase alfa produced at the 4000 L scale to the 160 L scale product in terms of the change from baseline of the left ventricular mass Z-score (LVM-Z) after 52 weeks of treatment.
    Protection of trial 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.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    21 Aug 2012
    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
    Germany: 1
    Country: Number of subjects enrolled
    United States: 3
    Worldwide total number of subjects
    4
    EEA total number of subjects
    1
    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)
    4
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted between 21 August 2012 and 1 December 2014.

    Pre-assignment
    Screening details
    A total of 5 subjects were screened and 4 subjects were randomized and treated.

    Period 1
    Period 1 title
    Overall Study (Overall Period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Algucosidase Alfa 4000 L material (Non-US subjects)
    Arm description
    Alglucosidase alfa 4000 L material for 52 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase Alfa
    Investigational medicinal product code
    Other name
    Lumizyme
    Pharmaceutical forms
    Powder for concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    20 mg/kg body weight every other week (qow).

    Arm title
    Algucosidase Alfa 160 L material (US subjects)
    Arm description
    Alglucosidase alfa 160 L material for 52 weeks.
    Arm type
    Active comparator

    Investigational medicinal product name
    Alglucosidase Alfa
    Investigational medicinal product code
    Other name
    Myozyme
    Pharmaceutical forms
    Powder for concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    20 mg/kg body weight qow.

    Number of subjects in period 1
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects)
    Started
    1
    3
    Treated
    1
    3
    Completed
    0
    2
    Not completed
    1
    1
         Study Terminated by Sponsor
    1
    -
         Physician decision
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Algucosidase Alfa 4000 L material (Non-US subjects)
    Reporting group description
    Alglucosidase alfa 4000 L material for 52 weeks.

    Reporting group title
    Algucosidase Alfa 160 L material (US subjects)
    Reporting group description
    Alglucosidase alfa 160 L material for 52 weeks.

    Reporting group values
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects) Total
    Number of subjects
    1 3 4
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    0.3 ± 0 0.5 ± 0.28 -
    Gender categorical
    Units: Subjects
        Female
    0 0 0
        Male
    1 3 4

    End points

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    End points reporting groups
    Reporting group title
    Algucosidase Alfa 4000 L material (Non-US subjects)
    Reporting group description
    Alglucosidase alfa 4000 L material for 52 weeks.

    Reporting group title
    Algucosidase Alfa 160 L material (US subjects)
    Reporting group description
    Alglucosidase alfa 160 L material for 52 weeks.

    Primary: Change From Baseline in Cardiac Function at Week 52

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    End point title
    Change From Baseline in Cardiac Function at Week 52 [1]
    End point description
    Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). 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 indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. Analysis was carried out on full analysis population defined as all subjects who receive at least 1 infusion of alglucosidase alfa.
    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: Since the data for this endpoint is available for only 1 arm, no statistical analysis could be performed for between arm comparison.
    End point values
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects)
    Number of subjects analysed
    0 [2]
    2
    Units: Units on a scale
        arithmetic mean (standard deviation)
    ±
    -5.06 ± 1.103
    Notes
    [2] - For this endpoint no subjects were analyzed in this arm at Baseline and Week 52
    No statistical analyses for this end point

    Secondary: Estimated Probability of Survival

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    End point title
    Estimated Probability of Survival
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects)
    Number of subjects analysed
    0 [3]
    0 [4]
    Units: weeks
        number (not applicable)
    Notes
    [3] - No death occurred during the study.
    [4] - No death occurred during the study.
    No statistical analyses for this end point

    Secondary: Probability of Invasive Ventilator-Free Survival

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    End point title
    Probability of Invasive Ventilator-Free Survival
    End point description
    Invasive ventilator-free survival is defined as the time during which the subject is alive and not invasively ventilated. Analysis was carried out on full analysis population.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects)
    Number of subjects analysed
    1
    3
    Units: weeks
        number (confidence interval 95%)
    0 (0 to 0)
    0 (0 to 0)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Motor Development Status at Week 52

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    End point title
    Change From Baseline in Motor Development Status at Week 52
    End point description
    Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. Analysis was carried out on full analysis population.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    Algucosidase Alfa 4000 L material (Non-US subjects) Algucosidase Alfa 160 L material (US subjects)
    Number of subjects analysed
    0 [5]
    2
    Units: percentage of maximum total score
        arithmetic mean (standard deviation)
    ±
    48.65 ± 17.183
    Notes
    [5] - For this endpoint no subjects were analyzed in this arm at Week 52.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 52) regardless of seriousness or relationship to investigational product.
    Adverse event reporting additional description
    Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period' (the period from the first infusion date to the date that the last data were collected). Analysis was carried out on full analysis population.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Alglucosidase Alfa 160 L material (US subjects)
    Reporting group description
    Alglucosidase alfa 160 L material for 52 weeks.

    Reporting group title
    Algucosidase Alfa 4000 L material (Non-US subjects)
    Reporting group description
    Alglucosidase alfa 4000 L material for 52 weeks.

    Serious adverse events
    Alglucosidase Alfa 160 L material (US subjects) Algucosidase Alfa 4000 L material (Non-US subjects)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Cardiac disorders
    Cardiac Failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary Oedema
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory Failure
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Hypotonia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Syncope
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Urticaria
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Adenovirus Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lobar Pneumonia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Otitis Media Acute
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory Syncytial Virus Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Alglucosidase Alfa 160 L material (US subjects) Algucosidase Alfa 4000 L material (Non-US subjects)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 1 (100.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    General disorders and administration site conditions
    Device Occlusion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Infusion Site Erythema
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 1 (100.00%)
         occurrences all number
    9
    3
    Injury, poisoning and procedural complications
    Arthropod Bite
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Oxygen Saturation Decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Cardiac Failure
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Cardiomyopathy
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Cardiac Failure Congestive
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nodal Rhythm
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Aspiration
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Asthma
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Bronchial Secretion Retention
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Cough
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nasal Congestion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Hypoxia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Pleural Effusion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Rhinorrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Respiratory Disorder
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Nystagmus
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Middle Ear Effusion
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    4
    0
    Skin and subcutaneous tissue disorders
    Dermatitis Contact
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Dermatitis Diaper
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Rash Erythematous
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Rash Pruritic
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Urticaria
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Muscle Contracture
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Croup Infectious
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Eye Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Fungal Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Pneumonia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Urinary Tract Infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Jan 2012
    This protocol amendment includes 2 major changes that substantially affect the inclusion criteria for participation in this study. The upper limit of age at the time of the first study infusion was increased from 6 months to 12 months. In addition, the requirement that a subject must have an left ventricular mass index Z-score (LVMI-Z) of greater than 2 was removed. Protocol Synopsis and Abbreviations: Updated to reflect overall changes in protocol. Number of Subjects: expanded the subject population to include subjects without cardiac hypertrophy who met the inclusion criteria. Revised secondary objectives to reflect addition of Alberta Infant Motor Scale (AIMS) for evaluation of changes in motor function in response to enzyme replacement therapy (ERT) over time. Revised tertiary efficacy endpoints to include AIMS as the most appropriate endpoint to assess subjects without cardiac hypertrophy. Assessment was moved from Demographic and Screening Assessments to Efficacy Assessments. Respiratory Function: Updated for clarification. If the subject required mechanical ventilatory support during the study, the start date, stop date, and reasons for each episode of continuous ventilator support would be recorded on the electronic case report form (eCRF) by authorized site personnel. Determination of Sample Size: Updated for clarity. Secondary Efficacy Analyses: Updated for clarity. Schedule of Study Events: Added assessment of AIMS at Week 12, Week 26, and Week 52 as AIMS was the most appropriate endpoint to assess subjects without cardiac hypertrophy.
    11 Oct 2012
    This protocol amendment includes 1 major change to the planned analysis of this study. The primary endpoint of this study was change from baseline in cardiac function as measured by left ventricular mass Z-score (LVM-Z) assessed at the end of the 52-week treatment period. - Changed protocol descriptor and protocol title. - Added date of Protocol Amendment 2. - Added Genzyme Europe BV as Sponsor. - Updated Medical Monitor information. - Changed “Global Patient Safety and Risk Management” to “Global Pharmacovigilance and Epidemiology” to reflect recent department name change.- Changed primary objective to indicate that the primary efficacy endpoint was change in left ventricular mass Z-score (LVM-Z) from baseline to Week 52.- Changed all instances of "LVMI-Z" to "LVM-Z" to reflect change in primary efficacy endpoint. - Updated Abbreviations section to add "confidence interval", "Global Pharmacovigilance and Epidemiology" and "noninferiority" and to remove "Global Patient Safety and Risk Management".- Added text to describe Phase 3 or Phase 4 protocol dependent upon country registration status.- Added text to indicate blood testing for cross-reactive immunologic material (CRIM) status was optional and did not had to be repeated if written results were available for testing performed prior to study enrollment.- Amended text to indicate R voltage V6 and S voltage V1 were not required assessments.- Added Sections to provide definitions for "Anaphylactic / Hypersensitivity Reactions" and "Immune Mediated Reactions", respectively. - Revised statistical parameters to correspond with LVM values from Genzyme study AGLU01602/2403.- Revised text to indicate change in analysis planned for clinical laboratory assessments.- Removed stipulation that Data Monitoring Committee be comprised of 4 physicians. Removed Sponsor contact information for Europe as all product handling is now provided by North America office.

    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.
    None reported
    The status of studies in GB is no longer updated from 1.1.2021
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