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    Clinical Trial Results:
    An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

    Summary
    EudraCT number
    2015-001955-54
    Trial protocol
    NL   DE   BE   ES  
    Global end of trial date
    07 Sep 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
    BMN-051-302
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02636686
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    Duchenne Muscular Dystrophy: DMD
    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, clinicaltrials@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?
    No
    Results analysis stage
    Analysis stage
    Interim
    Date of interim/final analysis
    17 Oct 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Sep 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study.
    Protection of trial subjects
    Not applicable
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    15 Sep 2015
    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: 4
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Netherlands: 8
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    United States: 2
    Worldwide total number of subjects
    19
    EEA total number of subjects
    17
    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)
    5
    Adolescents (12-17 years)
    11
    Adults (18-64 years)
    3
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    All subjects entering the treatment phase are required to provide written informed consent/assent (along with parental/guardian consent if applicable) and have confirmation that the inclusion and exclusion criteria have been evaluated by a medically qualified designee, including medical history and concurrent medication review.

    Period 1
    Period 1 title
    BMN051-302 (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
    6 mg/kg/wk SC
    Arm description
    6 mg/kg/wk SC
    Arm type
    Experimental

    Investigational medicinal product name
    drisapersen
    Investigational medicinal product code
    BMN051
    Other name
    GSK2402968, PRO051
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV dosing of 3mg/kg/week over a 1 hour infusion time

    Arm title
    3 mg/kg/wk IV
    Arm description
    3 mg/kg/wk IV
    Arm type
    Experimental

    Investigational medicinal product name
    drisapersen
    Investigational medicinal product code
    BMN051
    Other name
    GSK2402968, PRO051
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV dosing of 3mg/kg/week over a 1 hour infusion time

    Number of subjects in period 1
    6 mg/kg/wk SC 3 mg/kg/wk IV
    Started
    11
    8
    Completed
    0
    0
    Not completed
    11
    8
         Consent withdrawn by subject
    1
    -
         Death
    1
    -
         Study Terminated by Sponsor
    9
    7
         Missing
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    6 mg/kg/wk SC
    Reporting group description
    6 mg/kg/wk SC

    Reporting group title
    3 mg/kg/wk IV
    Reporting group description
    3 mg/kg/wk IV

    Reporting group values
    6 mg/kg/wk SC 3 mg/kg/wk IV Total
    Number of subjects
    11 8 19
    Age categorical
    Units: Subjects
        9 - 11
    4 1 5
        12 - 17
    4 7 11
        >= 18
    3 0 3
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    13.8 ± 4.31 13.3 ± 1.98 -
    Gender categorical
    Units: Subjects
        Female
    0 0 0
        Male
    11 8 19
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    0 1 1
        Not Hispanic or Latino
    10 7 17
        Unknown or Not Reported
    1 0 1

    End points

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    End points reporting groups
    Reporting group title
    6 mg/kg/wk SC
    Reporting group description
    6 mg/kg/wk SC

    Reporting group title
    3 mg/kg/wk IV
    Reporting group description
    3 mg/kg/wk IV

    Primary: Safety

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    End point title
    Safety [1]
    End point description
    To evaluate the long-term safety and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study.
    End point type
    Primary
    End point timeframe
    Long term
    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: Study is terminated early and only safety data is presented.
    End point values
    6 mg/kg/wk SC 3 mg/kg/wk IV
    Number of subjects analysed
    11
    8
    Units: Safety
    0
    0
    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
    Reporting groups
    Reporting group title
    3 mg/kg/wk IV
    Reporting group description
    -

    Reporting group title
    6 mg/kg/wk SC
    Reporting group description
    -

    Serious adverse events
    3 mg/kg/wk IV 6 mg/kg/wk SC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    Congenital, familial and genetic disorders
    Duchenne muscular dystrophy
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         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: 5%
    Non-serious adverse events
    3 mg/kg/wk IV 6 mg/kg/wk SC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 8 (75.00%)
    11 / 11 (100.00%)
    Investigations
    Complement factor C3 decreased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 11 (9.09%)
         occurrences all number
    4
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    4
    Lower limb fracture
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Tibia fracture
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Cardiac disorders
    Arrhythmia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Cyanosis
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Headache
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    General disorders and administration site conditions
    Application site hypersensitivity
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Chills
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Influenza like illness
         subjects affected / exposed
    1 / 8 (12.50%)
    2 / 11 (18.18%)
         occurrences all number
    2
    2
    Injection site discolouration
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    3
    Injection site dryness
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    4
    Injection site erythema
         subjects affected / exposed
    0 / 8 (0.00%)
    7 / 11 (63.64%)
         occurrences all number
    0
    35
    Injection site haematoma
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    Injection site haemorrhage
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Injection site hypoaesthesia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Injection site induration
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    5
    Injection site vesicles
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    5
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Eye disorders
    Eye irritation
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Nausea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Hypoventilation
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Rash erythematous
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Skin hyperpigmentation
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    3 / 8 (37.50%)
    1 / 11 (9.09%)
         occurrences all number
    6
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Joint swelling
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Muscle contracture
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    Myalgia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Pain in extremity
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 11 (9.09%)
         occurrences all number
    2
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    Rhinitis
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Jul 2015
    Protocol Amendment 1

    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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