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    Clinical Trial Results:
    A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

    Summary
    EudraCT number
    2010-020611-36
    Trial protocol
    GB   DE   NL  
    Global end of trial date
    19 Dec 2013

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Apr 2016
    First version publication date
    09 Apr 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AGLU07310
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01288027
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    Secondary identifier: MSC12823
    Sponsors
    Sponsor organisation name
    Genzyme Corporation
    Sponsor organisation address
    500 Kendall Street, Cambridge, MA, United States, 02142
    Public contact
    Sanofi aventis recherche & développement, Trial Transparency Team, Contact-US@Sanofi.com
    Scientific contact
    Sanofi aventis recherche & développement, Trial Transparency Team, 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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    18 Mar 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Dec 2013
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in subjects with Late-Onset Pompe disease.
    Protection of trial 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
    06 Jul 2011
    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 Kingdom: 1
    Country: Number of subjects enrolled
    United States: 9
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Netherlands: 3
    Worldwide total number of subjects
    16
    EEA total number of subjects
    7
    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)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    13
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted at 11 centres between July 06, 2011 and December 19, 2013.

    Pre-assignment
    Screening details
    A total of 20 subjects were screened and 16 subjects were enrolled.

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

    Arms
    Arm title
    Alglucosidase Alfa
    Arm description
    Alglucosidase alfa every other week for 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Alglucosidase Alfa
    Investigational medicinal product code
    GZ419829
    Other name
    Myozyme®, Lumizyme®
    Pharmaceutical forms
    Powder for concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Alglucosidase alfa 20 mg/kg.

    Number of subjects in period 1
    Alglucosidase Alfa
    Started
    16
    Full Analysis Set (FAS)
    16
    Completed
    16

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Alglucosidase Alfa
    Reporting group description
    Alglucosidase alfa every other week for 24 weeks.

    Reporting group values
    Alglucosidase Alfa Total
    Number of subjects
    16 16
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    51.6 ± 13.69 -
    Gender categorical
    Units: Subjects
        Female
    9 9
        Male
    7 7

    End points

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    End points reporting groups
    Reporting group title
    Alglucosidase Alfa
    Reporting group description
    Alglucosidase alfa every other week for 24 weeks.

    Primary: Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26

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    End point title
    Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 [1]
    End point description
    Tissue glycogen content was measured by quadriceps biopsies as ‘percent area of tissue occupied by glycogen'. Analysis was carried out on full analysis set (FAS) population defined as all subjects who received at least one complete infusion of alglucosidase alfa. Here, n = number of subjects with both Baseline and Week 26 assessment of tissue glycogen content.
    End point type
    Primary
    End point timeframe
    Baseline, Week 26
    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: Due to EudraCT format constraint, the statistical analysis could not be provided for single arm study.
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    16
    Units: percent area occupied by glycogen
    arithmetic mean (standard deviation)
        Baseline (n=14)
    5.3 ± 4.59
        Change at Week 26 (n=13)
    -1.6 ± 4.11
    No statistical analyses for this end point

    Secondary: Glycogen Distribution

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    End point title
    Glycogen Distribution
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    0 [2]
    Units: subjects
    Notes
    [2] - No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
    No statistical analyses for this end point

    Secondary: Muscle Fiber Morphology

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    End point title
    Muscle Fiber Morphology
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    0 [3]
    Units: subjects
    Notes
    [3] - No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
    No statistical analyses for this end point

    Secondary: Lysosomal Inclusions

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    End point title
    Lysosomal Inclusions
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    0 [4]
    Units: subjects
    Notes
    [4] - No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26

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    End point title
    Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26
    End point description
    Muscle involvement was assessed by T1-weighted magnetic resonance imaging (MRI). T1-weighted MRI data was analyzed using the Mercuri scoring in both legs (Total score = 1-4; where 1=Normal appearance, 2=Mild involvement, 3=Moderate involvement, and 4=Severe involvement). For each subjects, the average for each the upper (thigh) and lower leg was computed for Mercuri grading. Analysis was carried out on FAS population defined as all subjects who received at least one complete infusion of alglucosidase alfa. Here, Number of subjects analyzed= subjects with both Baseline and Week 26 assessment of muscle involvement, n= number of subjects with both Baseline and Week 26 assessment of muscle involvement for specified category.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    14
    Units: percent change
    arithmetic mean (standard deviation)
        Mercuri Scoring - Upper Leg (n=13)
    2.6 ± 9.24
        Mercuri Scoring - Lower Leg (n=14)
    0 ± 0
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Degree of Fatty Infiltration Using 3-Point 3-Dimensional (3D) Dixon at Week 26

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    End point title
    Percent Change From Baseline in Degree of Fatty Infiltration Using 3-Point 3-Dimensional (3D) Dixon at Week 26
    End point description
    Degree of Fatty Infiltration was assessed by 3-point 3D Dixon acquisition using skeletal muscle MRI in a subset of subjects. Analysis was carried out on FAS population defined as all subjects who received at least one complete infusion of alglucosidase alfa. Here, number of subjects analyzed = number of subjects with both Baseline and Week 26 assessment of degree of fatty infiltration.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    3
    Units: percent change
        arithmetic mean (standard deviation)
    2 ± 12.83
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26

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    End point title
    Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26
    End point description
    Disease activity (inflammation and/or water content within muscles) was quantitatively assessed by T2 MRI values in a subset of subjects. A T2 MRI value of greater than (>) 39 millisecond (ms) was defined as abnormal. T2 estimation normally requires an additional acquisition for computing the B1 spatial deviation however, can still be estimated if this acquisition is missing. Analysis was carried out on FAS population defined as all subjects who received at least one complete infusion of alglucosidase alfa. Here Number of subjects analyzed = number of subjects with both baseline and Week 26 assessment of disease activity.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    Alglucosidase Alfa
    Number of subjects analysed
    6
    Units: percent change
    arithmetic mean (standard deviation)
        T2 with B1
    8.1 ± 14.19
        T2 without B1
    7.2 ± 13.58
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From signature of the informed consent form up to the final visit (Week 26)
    Adverse event reporting additional description
    In the event a single subject has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all subjects who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    Aglucosidase Alfa
    Reporting group description
    Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.

    Serious adverse events
    Aglucosidase Alfa
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 16 (6.25%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Eye disorders
    Retinal Vascular Thrombosis
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Aglucosidase Alfa
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 16 (93.75%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Flushing
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Peripheral Coldness
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Thrombosis
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Melanocytic Naevus
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    General disorders and administration site conditions
    Adverse Drug Reaction
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Feeling Cold
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Influenza Like Illness
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Injection Site Extravasation
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Malaise
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Reproductive system and breast disorders
    Epididymitis
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Arthropod Bite
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Ligament Sprain
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Procedural Pain
         subjects affected / exposed
    3 / 16 (18.75%)
         occurrences all number
    3
    Investigations
    Heart Rate Increased
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Nasal Congestion
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Oropharyngeal Pain
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Pharyngeal Oedema
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Tachypnoea
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 16 (18.75%)
         occurrences all number
    3
    Headache
         subjects affected / exposed
    4 / 16 (25.00%)
         occurrences all number
    4
    Muscle Contractions Involuntary
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Paraesthesia
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Sciatica
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Eye disorders
    Ocular Hyperaemia
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Tympanic Membrane Perforation
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Abdominal Discomfort
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Dry Skin
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Urticaria
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Back Pain
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Muscle Spasms
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Myalgia
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Nasopharyngitis
         subjects affected / exposed
    5 / 16 (31.25%)
         occurrences all number
    5
    Ear Infection
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Subcutaneous Abscess
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Upper Respiratory Tract Infection
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Oct 2010
    - Six-minute Walk Test added to Study - Post-treatment assessment visit added after Week 24 - Screening and baseline visit take place on separate days - Metabolism and proteomics data to be reported separately from CSR - Hand-held dynamometry changed from break technique to make technique
    11 Mar 2011
    - Removed 65 year-old limit for inclusion in Study - Removed testing for cardiac hypertrophy - Contraception requirement added - Multiple techniques for muscle biopsy allowed - Allow dose modification in case of AE
    30 Mar 2012
    Added electrical impedance myography (EIM) as an additional exploratory assessment at Screening/Baseline and Visits 6, 9, 12, and 15 at a selected subset of sites.

    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
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