Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    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

    		 Close Top of page
    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

    		 Close Top of page
    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

    		 Close Top of page
    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

    		 Close Top of page
    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

    		 Close Top of page
    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

    		 Close Top of page
    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

    		 Close Top of page

    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.

    European Medicines Agency © 1995-Sat Apr 20 04:39:56 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA