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    Clinical Trial Results:
    A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

    Summary
    EudraCT number
    2015-003681-87
    Trial protocol
    BE   IT  
    Global end of trial date
    12 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-044-201
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02958202
    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., 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
    Final
    Date of interim/final analysis
    03 Nov 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Sep 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety of BMN 044 in subjects with DMD correctable by BMN 044 induced DMD exon 44 skipping who have previously participated in an eligible study with BMN 044
    Protection of trial subjects
    Not applicable.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    19 Apr 2016
    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
    Sweden: 3
    Worldwide total number of subjects
    7
    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)
    2
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    For subjects enrolling directly after completion of an existing BMN044 study, assessments from their last visit will be used as the baseline assessment. For subjects who have not received any BMN044 treatment for more than 28 days, these assessments will be performed: Physical examination, ECG, echocardiography, urinalysis hematology/biochemistry.

    Period 1
    Period 1 title
    044201 (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/week IV
    Arm description
    6 mg/kg/week IV
    Arm type
    Experimental

    Investigational medicinal product name
    BMN-044
    Investigational medicinal product code
    BMN-044
    Other name
    PRO-044
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects will receive 6 mg/kg weekly through IV administration. BMN 044 is presented in a 2R glass vial (Type 1) containing a solution of the active ingredient dissolved in 20 mM aqueous phosphate buffer, pH 7 solution for injection, 100 and 200 mg/mL. Each vial of BMN 044 contains 1.0 mL (0.8 mL extractable amount) of active ingredient. The solution is colorless to slightly yellow in appearance.

    Arm title
    9 mg/kg/week IV
    Arm description
    9 mg/kg/week IV
    Arm type
    Experimental

    Investigational medicinal product name
    BMN-044
    Investigational medicinal product code
    BMN-044
    Other name
    PRO-044
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects will receive 9 mg/kg weekly through IV administration. BMN 044 is presented in a 2R glass vial (Type 1) containing a solution of the active ingredient dissolved in 20 mM aqueous phosphate buffer, pH 7 solution for injection, 100 and 200 mg/mL. Each vial of BMN 044 contains 1.0 mL (0.8 mL extractable amount) of active ingredient. The solution is colorless to slightly yellow in appearance.

    Arm title
    6 mg/kg/week SC
    Arm description
    6 mg/kg/week SC
    Arm type
    Experimental

    Investigational medicinal product name
    BMN-044
    Investigational medicinal product code
    BMN-044
    Other name
    PRO-044
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects will receive 6 mg/kg weekly through SC administration. BMN 044 is presented in a 2R glass vial (Type 1) containing a solution of the active ingredient dissolved in 20 mM aqueous phosphate buffer, pH 7 solution for injection, 100 and 200 mg/mL. Each vial of BMN 044 contains 1.0 mL (0.8 mL extractable amount) of active ingredient. The solution is colorless to slightly yellow in appearance.

    Number of subjects in period 1
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Started
    3
    2
    2
    Completed
    0
    0
    0
    Not completed
    3
    2
    2
         Consent withdrawn by subject
    1
    -
    -
         Study Terminated by Sponsor
    2
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    6 mg/kg/week IV
    Reporting group description
    6 mg/kg/week IV

    Reporting group title
    9 mg/kg/week IV
    Reporting group description
    9 mg/kg/week IV

    Reporting group title
    6 mg/kg/week SC
    Reporting group description
    6 mg/kg/week SC

    Reporting group values
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC Total
    Number of subjects
    3 2 2 7
    Age categorical
    Units: Subjects
        10 - 21
    3 2 2 7
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    14.7 ± 5.69 13.5 ± 0.71 15 ± 5.66 -
    Gender categorical
    Units: Subjects
        Female
    0 0 0 0
        Male
    3 2 2 7
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    0 0 0 0
        Not Hispanic or Latino
    3 2 2 7
        Unknown or Not Reported
    0 0 0 0
    Race
    Units: Subjects
        American Indian or Alaska Native
    0 0 0 0
        Asian
    0 0 0 0
        Native Hawaiian or Other Pacific Islander
    0 0 0 0
        Black or African American
    0 0 0 0
        White
    3 2 2 7
        Other
    0 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    6 mg/kg/week IV
    Reporting group description
    6 mg/kg/week IV

    Reporting group title
    9 mg/kg/week IV
    Reporting group description
    9 mg/kg/week IV

    Reporting group title
    6 mg/kg/week SC
    Reporting group description
    6 mg/kg/week SC

    Primary: Safety

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    End point title
    Safety [1]
    End point description
    The primary (safety) analysis will be conducted on final completion of the study and will include the entirety of available safety data from both the source studies and this study.
    End point type
    Primary
    End point timeframe
    Long term extension
    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: Safety data presented elsewhere
    End point values
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Number of subjects analysed
    3
    2
    2
    Units: Totality of safety data
    3
    2
    2
    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
    19.0
    Reporting groups
    Reporting group title
    6 mg/kg/week IV
    Reporting group description
    -

    Reporting group title
    9 mg/kg/week IV
    Reporting group description
    -

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

    Serious adverse events
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    2 / 2 (100.00%)
    2 / 2 (100.00%)
    Investigations
    Complement factor C3 decreased
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    1
    0
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Sunburn
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    2
    Traumatic haematoma
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Injection site atrophy
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    1
    Injection site discolouration
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    8
    Injection site erythema
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    4
    Injection site haematoma
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    2 / 2 (100.00%)
         occurrences all number
    0
    0
    9
    Malaise
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    0
    Pyrexia
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    3
    0
    0
    Vessel puncture site haematoma
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    2
    0
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    2
    0
    Musculoskeletal and connective tissue disorders
    Neck pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Pain in extremity
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    2
    0
    1
    Infections and infestations
    Gastroenteritis viral
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Streptococcal infection
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Jan 2016
    The rationale for this amendment is to improve the understanding of the required assessments to be completed and to address feedback from Regulatory and Ethics Groups. 1. Clarification of how subjects will transition from parent studies to this study depending on whether they are still taking BMN 044 or have been off treatment for more than 28 days. 2. Clarification of assessments required during the 2 phases of the study, including revising the schedule of assessment tables. 3. Confirm monitoring requirements for intravenous infusions. 4. Making the laboratory stopping criteria consistent with the BioMarin AON programme. 5. Amending the statistical section to account for handling transitioning subjects and their data. 6. Administrative changes to improve the readability of the document and ensure consistency throughout.

    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.
    BioMarin terminated the study early due to a company decision to stop the development of exon-skipping DMD therapies. This decision was based on the marketing authorisation filing in the US and EU for a related compound, drisapersen, for which regula
    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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