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    Clinical Trial Results:
    A Phase 4, Multi-Center, Multi-National, Open-Label, Randomized, Two Dose Level Study of Naglazyme® (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI)

    Summary
    EudraCT number
    		 2005-003512-30
    Trial protocol
    		 FR   PT  
    Global end of trial date
    30 Apr 2009

    Results information
    Results version number
    		 v1(current)
    This version publication date
    18 Dec 2019
    First version publication date
    18 Dec 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ASB-008
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00299000
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    BioMarin Pharmaceutical Inc.
    Sponsor organisation address
    105 Digital Drive, Novato, United States, CA 94949
    Public contact
    Clinical Trials Information, BioMarin Pharmaceutical Inc., clinicaltrials@bmrn.com
    Scientific contact
    Clinical Trials Information, BioMarin Pharmaceutical Inc., 800 983-4587, medinfo@bmrn.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
    14 Jan 2010
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Apr 2009
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Apr 2009
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
    Protection of trial subjects
    This clinical study was designed, conducted, recorded, and reported in compliance with the principles of Good Clinical Practice (GCP) guidelines. These guidelines are stated in U.S. federal regulations as well as “Guidance for Good Clinical Practice,” International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use.
    Background therapy
    		 Not applicable
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    08 May 2006
    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: 2
    Country: Number of subjects enrolled
    Portugal: 1
    Country: Number of subjects enrolled
    France: 1
    Worldwide total number of subjects
    4
    EEA total number of subjects
    2
    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
    		 This study was conducted at 3 primary study centers.

    Pre-assignment
    Screening details
    		 Subjects enrolled were 4 and all subjects completed the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Naglazyme, 1.0 mg/kg
    Arm description
    		 Weekly infusions for minimum of 52 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Naglazyme, 1.0 mg/kg
    Investigational medicinal product code
    Other name
    Recombinant human N-acetylgalactosamine 4-sulfatase, rh-arylsulfatase B, rhASB
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Naglazyme, 1.0 mg/kg/week was administered as an intravenous infusion over no less than a 4-hour period per infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution

    Arm title
    		 Naglazyme, 2.0 mg/kg
    Arm description
    		 Weekly infusions for minimum of 52 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    Naglazyme, 2.0 mg/kg
    Investigational medicinal product code
    Other name
    Recombinant human N-acetylgalactosamine 4-sulfatase, rh-arylsulfatase B, rhASB
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Naglazyme, 2.0 mg/kg/week was administered as an intravenous infusion over no less than a 4-hour period per infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution

    Number of subjects in period 1
    		Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Started
    2
    2
    Completed
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Naglazyme, 1.0 mg/kg
    Reporting group description
    		 Weekly infusions for minimum of 52 weeks

    Reporting group title
    Naglazyme, 2.0 mg/kg
    Reporting group description
    		 Weekly infusions for minimum of 52 weeks

    Reporting group values
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg Total
    Number of subjects
    		 2 2 4
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 2 2 4
    Age continuous
    Units: months
        arithmetic mean (standard deviation)
    		 6.05 ( 3.89 ) 12.4 ( 0.42 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 2 2 4

    End points

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    End points reporting groups
    Reporting group title
    Naglazyme, 1.0 mg/kg
    Reporting group description
    		 Weekly infusions for minimum of 52 weeks

    Reporting group title
    Naglazyme, 2.0 mg/kg
    Reporting group description
    		 Weekly infusions for minimum of 52 weeks

    Primary: Change in Height

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    End point title
    		 Change in Height [1]
    End point description
    End point type
    Primary
    End point timeframe
    52 weeks
    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: Statistical testing was conducted, but no formal statistical inference was planned for the study because of the small number of subjects planned to enroll into the study.
    End point values
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Number of subjects analysed
    2
    2
    Units: centimeters
    arithmetic mean (standard deviation)
        Height at Baseline
    67.3 ( 5.1 )
    80.3 ( 1.8 )
        Height at week 52
    81.9 ( 1.8 )
    91.3 ( 5.2 )
        Change in Height
    14.6 ( 6.9 )
    11.0 ( 7.0 )
    No statistical analyses for this end point

    Primary: Change in Weight

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    End point title
    		 Change in Weight [2]
    End point description
    End point type
    Primary
    End point timeframe
    52 weeks
    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: Statistical testing was conducted, but no formal statistical inference was planned for the study because of the small number of subjects planned to enroll into the study.
    End point values
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Number of subjects analysed
    2
    2
    Units: Kilograms
    arithmetic mean (standard deviation)
        Weight at Baseline
    7.1 ( 0.3 )
    10.2 ( 0.6 )
        Weight at week 52
    11.3 ( 0.3 )
    13.3 ( 1.0 )
        Change in weight
    4.2 ( 0.6 )
    3.1 ( 1.6 )
    No statistical analyses for this end point

    Primary: Change in Head Circumference

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    End point title
    		 Change in Head Circumference [3]
    End point description
    End point type
    Primary
    End point timeframe
    52 weeks
    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: Statistical testing was conducted, but no formal statistical inference was planned for the study because of the small number of subjects planned to enroll into the study.
    End point values
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Number of subjects analysed
    2
    2
    Units: Centimeter
    arithmetic mean (standard deviation)
        Head circumference at Baseline
    43.5 ( 3.0 )
    49.1 ( 0.5 )
        Head circumference at 52 weeks
    48.5 ( 1.0 )
    51.8 ( 0.8 )
        Change in Head circumference
    5.0 ( 4.0 )
    2.7 ( 1.3 )
    No statistical analyses for this end point

    Secondary: Change in Urinary Glycosaminoglycan Levels

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    End point title
    		 Change in Urinary Glycosaminoglycan Levels
    End point description
    Change in urinary GAG levels was calculated from baseline to week 52 of treatment
    End point type
    Secondary
    End point timeframe
    52 weeks
    End point values
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Number of subjects analysed
    2
    2
    Units: ug/mg
    arithmetic mean (standard deviation)
        Baseline
    1041.87 ( 86.32 )
    698.95 ( 142.34 )
        Week 52
    261.15 ( 54.38 )
    178.10 ( 42.43 )
        Change from Baseline to Week 52
    -780.72 ( 140.69 )
    -520.85 ( 99.91 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    Naglazyme, 1.0 mg/kg
    Reporting group description
    		 -

    Reporting group title
    Naglazyme, 2.0 mg/kg
    Reporting group description
    		 -

    Serious adverse events
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Vascular disorders
    Poor venous access
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Surgical and medical procedures
    Cast application
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Febrile convulsion
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Umbilical hernia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Adenoidal hypertrophy
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory distress
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Scoliosis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tonsillitis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Naglazyme, 1.0 mg/kg Naglazyme, 2.0 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    8
    31
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
         occurrences all number
    4
    3
    Umbilical hernia
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    3
    1
    Vomiting
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 2 (100.00%)
         occurrences all number
    2
    3
    Respiratory, thoracic and mediastinal disorders
    Nasal congestion
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    15
    1
    Infections and infestations
    Ear infection
         subjects affected / exposed
    2 / 2 (100.00%)
    2 / 2 (100.00%)
         occurrences all number
    2
    4
    Rhinitis
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 2 (100.00%)
         occurrences all number
    3
    3
    Upper respiratory tract infections
         subjects affected / exposed
    2 / 2 (100.00%)
    1 / 2 (50.00%)
         occurrences all number
    9
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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