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    Clinical Trial Results:
    An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    		 2011-001266-17
    Trial protocol
    		 BE   FR   DE   GB   NL   ES   IT   Outside EU/EEA   BG   HU   DK   CZ  
    Global end of trial date
    18 Feb 2014

    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
    DMD114349
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01480245
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    BioMarin Pharmaceutical Inc.
    Sponsor organisation address
    105 Digital Drive, Novato, United States, CA94949
    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)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000746-PIP01-09
    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
    24 Jul 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Feb 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the long term safety, tolerability and efficacy of subcutaneous 6mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044.
    Protection of trial subjects
    Not applicable
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    23 Aug 2011
    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
    Argentina: 6
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Brazil: 16
    Country: Number of subjects enrolled
    Bulgaria: 2
    Country: Number of subjects enrolled
    Canada: 21
    Country: Number of subjects enrolled
    Chile: 7
    Country: Number of subjects enrolled
    Czech Republic: 7
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    France: 19
    Country: Number of subjects enrolled
    Germany: 29
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Italy: 25
    Country: Number of subjects enrolled
    Japan: 14
    Country: Number of subjects enrolled
    Korea, Republic of: 6
    Country: Number of subjects enrolled
    Netherlands: 10
    Country: Number of subjects enrolled
    Norway: 2
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Russian Federation: 8
    Country: Number of subjects enrolled
    Spain: 16
    Country: Number of subjects enrolled
    Taiwan: 3
    Country: Number of subjects enrolled
    Turkey: 12
    Country: Number of subjects enrolled
    United Kingdom: 10
    Worldwide total number of subjects
    233
    EEA total number of subjects
    132
    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)
    207
    Adolescents (12-17 years)
    26
    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
    		 This study will include all eligible subjects who participated in studies DMD114117 or DMD114044, and choose to enter this open label study.

    Period 1
    Period 1 title
    114349 (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
    		 6 mg/kg Drisapersen Continuous
    Arm description
    		 6 mg/kg Drisapersen Continuous
    Arm type
    		 Experimental

    Investigational medicinal product name
    drisapersen
    Investigational medicinal product code
    BMN-051
    Other name
    PRO-051, GSK2402968
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Eligible subjects will receive drisapersen 6mg/kg/weekly. Drisapersen will be supplied as 3 ml vials containing 1ml or 0.7ml sterile solution for subcutaneous injection. The strength of drisapersen solution will be 200 mg/ml.

    Arm title
    		 6 mg/kg Drisapersen Intermittent
    Arm description
    		 6 mg/kg Drisapersen Intermittent
    Arm type
    		 Experimental

    Investigational medicinal product name
    drisapersen
    Investigational medicinal product code
    BMN-051
    Other name
    PRO-051, GSK2402968
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Eligible subjects will receive drisapersen 6mg/kg/week for 8 weeks followed by 4 weeks off of drug. Drisapersen will be supplied as 3 ml vials containing 1ml or 0.7ml sterile solution for subcutaneous injection. The strength of drisapersen solution will be 200 mg/ml.

    Arm title
    		 Natural History
    Arm description
    		 Natural History
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		6 mg/kg Drisapersen Continuous 6 mg/kg Drisapersen Intermittent Natural History
    Started
    228
    12
    17
    Completed
    0
    0
    0
    Not completed
    228
    12
    17
         Consent withdrawn by subject
    9
    -
    1
         Adverse event
    1
    -
    -
         Study Terminated by Sponsor
    216
    12
    15
         Lack of efficacy
    2
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    6 mg/kg Drisapersen Continuous
    Reporting group description
    		 6 mg/kg Drisapersen Continuous

    Reporting group title
    6 mg/kg Drisapersen Intermittent
    Reporting group description
    		 6 mg/kg Drisapersen Intermittent

    Reporting group title
    Natural History
    Reporting group description
    		 Natural History

    Reporting group values
    		 6 mg/kg Drisapersen Continuous 6 mg/kg Drisapersen Intermittent Natural History Total
    Number of subjects
    		 228 12 17 233
    Age categorical
    Units: Subjects
        0-85 years and over
    		 228 12 17 233
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 8.9 ( 2.12 ) 9.9 ( 1.73 ) 8.7 ( 1.69 ) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0 0
        Male
    		 228 12 17 233

    End points

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    End points reporting groups
    Reporting group title
    6 mg/kg Drisapersen Continuous
    Reporting group description
    		 6 mg/kg Drisapersen Continuous

    Reporting group title
    6 mg/kg Drisapersen Intermittent
    Reporting group description
    		 6 mg/kg Drisapersen Intermittent

    Reporting group title
    Natural History
    Reporting group description
    		 Natural History

    Primary: change from baseline in muscle function using the 6MWD at week 104

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    End point title
    		 change from baseline in muscle function using the 6MWD at week 104 [1]
    End point description
    The primary efficacy endpoint for this study was the difference between baseline and Week 104 in 6MWD for subjects on the continuous drisapersen treatment group for the Modified Ambulant ITT population. However, only 4 subjects in the continuous drisapersen group and 1 subject in the natural history arm had efficacy data at Week 104. Therefore, the efficacy results presented in this section focus on data up through Week 72.
    End point type
    Primary
    End point timeframe
    Week 72
    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 has been terminated early and no full SAP has been used.
    End point values
    		 6 mg/kg Drisapersen Continuous 6 mg/kg Drisapersen Intermittent Natural History
    Number of subjects analysed
    56
    3
    2
    Units: Meter
        arithmetic mean (standard deviation)
    -90.81 ( 99.732 )
    -88.57 ( 115.366 )
    -41 ( 137.179 )
    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
    6 mg/kg Drisapersen Continuous
    Reporting group description
    		 -

    Reporting group title
    Natural History
    Reporting group description
    		 -

    Reporting group title
    6 mg/kg Drisapersen Intermittent
    Reporting group description
    		 -

    Serious adverse events
    		 6 mg/kg Drisapersen Continuous Natural History 6 mg/kg Drisapersen Intermittent
    Total subjects affected by serious adverse events
         subjects affected / exposed
    27 / 228 (11.84%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    International normalised ratio increased
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Ankle fracture
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Femur fracture
         subjects affected / exposed
    3 / 228 (1.32%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cyanosis
         subjects affected / exposed
    2 / 228 (0.88%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Hypotonia
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Haemolytic anaemia
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    8 / 228 (3.51%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    7 / 8
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Injection site oedema
         subjects affected / exposed
    2 / 228 (0.88%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Optic disc disorder
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Small intestinal obstruction
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatocellular injury
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatotoxicity
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal impairment
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    2 / 228 (0.88%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 6 mg/kg Drisapersen Continuous Natural History 6 mg/kg Drisapersen Intermittent
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    208 / 228 (91.23%)
    6 / 17 (35.29%)
    9 / 12 (75.00%)
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    6 / 228 (2.63%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    7
    0
    1
    Complement factor C3 decreased
         subjects affected / exposed
    12 / 228 (5.26%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    16
    0
    0
    Cystatin C increased
         subjects affected / exposed
    16 / 228 (7.02%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    33
    0
    0
    Fibrin D dimer increased
         subjects affected / exposed
    5 / 228 (2.19%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    6
    1
    0
    International normalised ratio increased
         subjects affected / exposed
    7 / 228 (3.07%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    11
    0
    1
    Protein urine present
         subjects affected / exposed
    30 / 228 (13.16%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    115
    0
    0
    Red blood cells urine
         subjects affected / exposed
    16 / 228 (7.02%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    37
    0
    0
    Red blood cells urine positive
         subjects affected / exposed
    25 / 228 (10.96%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    53
    0
    0
    Urine analysis abnormal
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    3
    0
    2
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    20 / 228 (8.77%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    40
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    22 / 228 (9.65%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    35
    1
    0
    Fall
         subjects affected / exposed
    51 / 228 (22.37%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    134
    0
    0
    Femur fracture
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    Injection related reaction
         subjects affected / exposed
    8 / 228 (3.51%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    9
    0
    1
    Ligament sprain
         subjects affected / exposed
    13 / 228 (5.70%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    18
    1
    0
    Nervous system disorders
    Aphonia
         subjects affected / exposed
    0 / 228 (0.00%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Burning sensation
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    Headache
         subjects affected / exposed
    63 / 228 (27.63%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    230
    0
    4
    Sensory disturbance
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    15 / 228 (6.58%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    21
    0
    0
    Hyperthermia
         subjects affected / exposed
    0 / 228 (0.00%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Injection site atrophy
         subjects affected / exposed
    24 / 228 (10.53%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    38
    0
    2
    Injection site bruising
         subjects affected / exposed
    19 / 228 (8.33%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    132
    0
    0
    Injection site discolouration
         subjects affected / exposed
    85 / 228 (37.28%)
    0 / 17 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    307
    0
    6
    Injection site erythema
         subjects affected / exposed
    73 / 228 (32.02%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    346
    0
    4
    Injection site haematoma
         subjects affected / exposed
    17 / 228 (7.46%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    38
    0
    3
    Injection site haemorrhage
         subjects affected / exposed
    3 / 228 (1.32%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    5
    0
    1
    Injection site induration
         subjects affected / exposed
    55 / 228 (24.12%)
    1 / 17 (5.88%)
    1 / 12 (8.33%)
         occurrences all number
    149
    1
    1
    Injection site pain
         subjects affected / exposed
    23 / 228 (10.09%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    81
    0
    1
    Injection site pruritus
         subjects affected / exposed
    22 / 228 (9.65%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    47
    0
    1
    Injection site reaction
         subjects affected / exposed
    29 / 228 (12.72%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    145
    0
    0
    Injection site swelling
         subjects affected / exposed
    17 / 228 (7.46%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    25
    0
    0
    Pyrexia
         subjects affected / exposed
    49 / 228 (21.49%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    86
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    31 / 228 (13.60%)
    1 / 17 (5.88%)
    1 / 12 (8.33%)
         occurrences all number
    74
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    18 / 228 (7.89%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    27
    0
    1
    Diarrhoea
         subjects affected / exposed
    43 / 228 (18.86%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    96
    0
    1
    Flatulence
         subjects affected / exposed
    1 / 228 (0.44%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    1
    0
    1
    Nausea
         subjects affected / exposed
    14 / 228 (6.14%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    19
    0
    0
    Toothache
         subjects affected / exposed
    2 / 228 (0.88%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    6
    1
    0
    Vomiting
         subjects affected / exposed
    59 / 228 (25.88%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    118
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    34 / 228 (14.91%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    40
    0
    0
    Epistaxis
         subjects affected / exposed
    24 / 228 (10.53%)
    1 / 17 (5.88%)
    1 / 12 (8.33%)
         occurrences all number
    36
    1
    1
    Oropharyngeal pain
         subjects affected / exposed
    16 / 228 (7.02%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    23
    0
    0
    Skin and subcutaneous tissue disorders
    Macule
         subjects affected / exposed
    0 / 228 (0.00%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Miliaria
         subjects affected / exposed
    0 / 228 (0.00%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Rash
         subjects affected / exposed
    13 / 228 (5.70%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    23
    0
    0
    Renal and urinary disorders
    Albuminuria
         subjects affected / exposed
    3 / 228 (1.32%)
    0 / 17 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    4
    0
    2
    Haematuria
         subjects affected / exposed
    25 / 228 (10.96%)
    0 / 17 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    63
    0
    3
    Proteinuria
         subjects affected / exposed
    75 / 228 (32.89%)
    0 / 17 (0.00%)
    3 / 12 (25.00%)
         occurrences all number
    216
    0
    11
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    19 / 228 (8.33%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    36
    0
    0
    Pain in extremity
         subjects affected / exposed
    30 / 228 (13.16%)
    1 / 17 (5.88%)
    3 / 12 (25.00%)
         occurrences all number
    50
    1
    3
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    18 / 228 (7.89%)
    0 / 17 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    21
    0
    2
    Influenza
         subjects affected / exposed
    13 / 228 (5.70%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    16
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    60 / 228 (26.32%)
    1 / 17 (5.88%)
    1 / 12 (8.33%)
         occurrences all number
    116
    1
    2
    Rhinitis
         subjects affected / exposed
    20 / 228 (8.77%)
    1 / 17 (5.88%)
    0 / 12 (0.00%)
         occurrences all number
    33
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    26 / 228 (11.40%)
    0 / 17 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    33
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Jul 2013
    Update to renal criteria timing; update to additional vial fill volume, length of study, removal of all dried bloodspot assessments and analysis, removal of several biomarker tests, minor corrections and clarifications added;
    09 Oct 2013
    Instructions for investigators for subject management during the time period while dosing is on hold per drisapersen Dear Investigator Letter

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    09 Oct 2013
    Dosing hold after results of study DMD114044 were analysed. (see amendmends)
    -

    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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