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    Clinical Trial Results:
    A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy

    Summary
    EudraCT number
    2011-005042-35
    Trial protocol
    GB   BE   IT   NL   FR  
    Global end of trial date
    31 Aug 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    23 Mar 2017
    First version publication date
    23 Mar 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PRO053-CLIN-01
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01957059
    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, BioMarin Pharmaceutical Inc., clinicaltrials@bmrn.com
    Scientific contact
    Clinical Trials, BioMarin Pharmaceutical Inc., clinicaltrials@bmrn.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-001374-PIP01-12
    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
    10 Oct 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Aug 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the efficacy of PRO053 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.
    Protection of trial subjects
    Not applicable
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    10 Jul 2013
    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
    Belgium: 1
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Netherlands: 4
    Country: Number of subjects enrolled
    United Kingdom: 2
    Worldwide total number of subjects
    9
    EEA total number of subjects
    9
    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)
    8
    Adolescents (12-17 years)
    1
    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
    -

    Pre-assignment
    Screening details
    Visit S1 will include confirmation of consent and evaluation of the full DNA diagnostic report and the inclusion and exclusion criteria. Visit S2 should not be conducted until enrolment into any given dose group is agreed. S2 will be conducted within 2 weeks prior to first dose. S2 will include evaluation of the inclusion and exclusion criteria.

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

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Cohort 1
    Arm description
    BMN 053
    Arm type
    Experimental

    Investigational medicinal product name
    BMN053
    Investigational medicinal product code
    BMN053
    Other name
    PRO053
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    In the dose-escalation phase, two cohorts of three subjects each receive two single doses of BMN 053 in two study periods (i.e., four single doses in total per subject). In each study period they will receive BMN 053 by IV infusion and by SC injection (separated by one week). Cohort 1: 1 mg/kg SC followed by 1 mg/kg IV And then 6 mg/kg SC followed by 6 mg/kg IV

    Arm title
    Cohort 2
    Arm description
    BMN 053
    Arm type
    Experimental

    Investigational medicinal product name
    BMN053
    Investigational medicinal product code
    BMN053
    Other name
    PRO053
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    In the dose-escalation phase, two cohorts of three subjects each receive two single doses of BMN 053 in two study periods (i.e., four single doses in total per subject). In each study period they will receive BMN 053 by IV infusion and by SC injection (separated by one week). Cohort 2: 3 mg/kg SC followed by 3 mg/kg IV And then 9 mg/kg SC followed by 9 mg/kg IV

    Number of subjects in period 1
    Cohort 1 Cohort 2
    Started
    9
    3
    Completed
    0
    3
    Not completed
    9
    0
         Study Subject Withdrawal by Parent or Guardian
    2
    -
         Study Terminated by Sponsor
    7
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    dose escalation
    Reporting group description
    -

    Reporting group values
    dose escalation Total
    Number of subjects
    9 9
    Age categorical
    Units: Subjects
        6 - 14
    9 9
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    8.6 ( 2.83 ) -
    Gender categorical
    Units: Subjects
        Female
    0 0
        Male
    9 9

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    BMN 053

    Reporting group title
    Cohort 2
    Reporting group description
    BMN 053

    Primary: Efficacy

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    End point title
    Efficacy [1]
    End point description
    To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks of dosing in ambulant subjects with Duchenne muscular dystrophy.
    End point type
    Primary
    End point timeframe
    48 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: Due to the early termination of this study, the treatment phase of the study was not initiated. For the dose escalation/regimen selection phase no formal statistical analysis was performed.
    End point values
    Cohort 1 Cohort 2
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: meter
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [2] - Due to the early termination of this study, the treatment phase of the study was not initiated.
    [3] - Due to the early termination of this study, the treatment phase of the study was not initiated.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Study Period
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18.1/19
    Reporting groups
    Reporting group title
    BMN 053
    Reporting group description
    -

    Serious adverse events
    BMN 053
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 9 (11.11%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Nervous system disorders
    Myoclonus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    BMN 053
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 9 (100.00%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Haematoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Inflammation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Pyrexia
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    4
    Injection site erythema
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    3
    Cough
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    4
    Rhinorrhoea
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Activated partial thromboplastin time shortened
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    4
    Alpha 1 microglobulin increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    C-reactive protein increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Complement factor c3 decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Glutamate dehydrogenase increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    3
    International normalised ratio increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Platelet count decreased
         subjects affected / exposed
    5 / 9 (55.56%)
         occurrences all number
    16
    Protein urine
         subjects affected / exposed
    7 / 9 (77.78%)
         occurrences all number
    54
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    4
    White blood cell count decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Fall
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    4
    Infusion related reaction
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Joint injury
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Post procedural haematoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Procedural pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Wound
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nervous system disorders
    Myoclonus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    26
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Eye disorders
    Conjunctival hyperaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Eye inflammation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Ocular hyperaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    6
    Abdominal pain upper
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Anal fissure
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Constipation
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Diarrhoea
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    9
    Dental caries
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Dyspepsia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    8
    Mouth ulceration
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Toothache
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    3
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Erythema
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Granuloma skin
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Pigmentation disorder
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Renal and urinary disorders
    Chromaturia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Haematuria
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Pain in extremity
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Myalgia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Back pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Conjunctivitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    4
    Laryngitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    3
    Ear infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    5 / 9 (55.56%)
         occurrences all number
    11
    Onychomycosis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Post procedural infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    6
    Tonsillitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Rhinitis
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    9

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Dec 2013
    Prot Amt 1
    17 Apr 2014
    Prot Amt 2
    11 May 2015
    Prot Amt 3
    13 Jan 2016
    Prot Amt 4

    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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