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    Clinical Trial Results:
    A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

    Summary
    EudraCT number
    2013-003605-26
    Trial protocol
    IT   SE   NL   BE  
    Global end of trial date
    10 Aug 2016

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

    Trial information

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    Trial identification
    Sponsor protocol code
    PRO044-CLIN-02
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02329769
    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
    30 Sep 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Aug 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the safety, tolerability and efficacy of two different doses of intravenous (IV) PRO044 and one dose of subcutaneous (SC) PRO044 in subjects with DMD after 48 weeks treatment.
    Protection of trial subjects
    Not applicable
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    31 Oct 2014
    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
    Italy: 5
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Sweden: 3
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    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)
    8
    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
    The screening visit will include confirmation of subject consent, an interview and evaluation of the inclusion and exclusion criteria and the assessments as described in the schedule of assessments. All results from assessments conducted at the screening visit must be available for review prior to the first PRO044 dose in this study.

    Period 1
    Period 1 title
    BMN044 (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    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, PS188
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Product: Active ingredient is PS188 which is the sodium salt of a 20-mer 2’-O-methyl-phosphorothioate oligoribonucleotide with a sequence optimised to skip exon 44 in the human dystrophin pre-mRNA. Formulation: Solution of the active ingredient PS188 in 20 mM phosphate buffer, pH 7 solution for injection. Strength: 200 mg/mL. Vial content: 0.7 mL or 1.0 mL in a 3 mL glass type I vial. Subject will receive 6 mg/kg weekly through IV administration.

    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, PS188
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Product: Active ingredient is PS188 which is the sodium salt of a 20-mer 2’-O-methyl-phosphorothioate oligoribonucleotide with a sequence optimised to skip exon 44 in the human dystrophin pre-mRNA. Formulation: Solution of the active ingredient PS188 in 20 mM phosphate buffer, pH 7 solution for injection. Strength: 200 mg/mL. Vial content: 0.7 mL or 1.0 mL in a 3 mL glass type I vial. Subject will receive 9 mg/kg weekly through IV administration.

    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, PS188
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Product: Active ingredient is PS188 which is the sodium salt of a 20-mer 2’-O-methyl-phosphorothioate oligoribonucleotide with a sequence optimised to skip exon 44 in the human dystrophin pre-mRNA. Formulation: Solution of the active ingredient PS188 in 20 mM phosphate buffer, pH 7 solution for injection. Strength: 200 mg/mL. Vial content: 0.7 mL or 1.0 mL in a 3 mL glass type I vial. Subject will receive 6 mg/kg weekly through SC administration.

    Number of subjects in period 1
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Started
    6
    5
    4
    Completed
    3
    2
    2
    Not completed
    3
    3
    2
         Study Terminated by Sponsor
    3
    3
    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
    6 5 4 15
    Age categorical
    Units: Subjects
        9-19
    6 5 4 15
    Age continuous
    Units: Years
        arithmetic mean (standard deviation)
    13 ( 3.63 ) 12.4 ( 1.82 ) 12.8 ( 3.77 ) -
    Gender categorical
    Units: Subjects
        Female
    0 0 0 0
        Male
    6 5 4 15
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    1 0 0 1
        Not Hispanic or Latino
    5 5 4 14
        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
    6 5 4 15
        Other
    0 0 0 0
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    52 ( 20.94 ) 52.1 ( 13.43 ) 35.7 ( 17.37 ) -
    Length
    Units: cm
        arithmetic mean (standard deviation)
    145.5 ( 13.52 ) 140.6 ( 19.33 ) 131.9 ( 19 ) -

    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: Not applicable

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    End point title
    Not applicable [1]
    End point description
    Due to a company decision to stop development of exon-skipping DMD therapy, efficacy data were not summarised.
    End point type
    Primary
    End point timeframe
    Not applicable
    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 a company decision to stop development of exon-skipping DMD therapy, efficacy data were not summarised.
    End point values
    6 mg/kg/week IV 9 mg/kg/week IV 6 mg/kg/week SC
    Number of subjects analysed
    0 [2]
    0 [3]
    0 [4]
    Units: not applicable
        number (not applicable)
    Notes
    [2] - Not applicable
    [3] - Not applicable
    [4] - Not applicable
    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/week IV
    Reporting group description
    -

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

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

    Serious adverse events
    6 mg/kg/week IV 6 mg/kg/week SC 9 mg/kg/week IV
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Cardiac disorders
    Atrial tachycardia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Supraventricular tachycardia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         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/week IV 6 mg/kg/week SC 9 mg/kg/week IV
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    4 / 4 (100.00%)
    5 / 5 (100.00%)
    Surgical and medical procedures
    Suture insertion
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Fatigue
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    0
    1
    Inflammation
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Influenza like illness
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    1
    0
    6
    Infusion site pruritus
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    2 / 6 (33.33%)
    2 / 4 (50.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    2
    0
    Vaccination site erythema
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Xerosis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 6 (50.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    1
    0
    Dyspnoea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Epistaxis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    3 / 6 (50.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    2
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    0
    1
    Alpha 1 microglobulin increased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    3
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Complement factor C3 decreased
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 4 (25.00%)
    2 / 5 (40.00%)
         occurrences all number
    1
    1
    5
    Crystal urine present
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Cystatin C increased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Ejection fraction decreased
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Glutamate dehydrogenase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    1
    Inflammatory marker increased
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Investigation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Platelet count decreased
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    0
    Protein urine present
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    1
    0
    Red blood cells urine
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    0
    Weight decreased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    White blood cell count decreased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Hand fracture
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Laceration
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Ligament sprain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Procedural pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    1
    Thermal burn
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Cardiac disorders
    Left ventricular dysfunction
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Tachycardia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 4 (0.00%)
    4 / 5 (80.00%)
         occurrences all number
    3
    0
    17
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Abdominal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    4
    0
    3
    Constipation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    2
    1
    1
    Dyspepsia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    0
    Gastritis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Haemorrhoids
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Nausea
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    4
    0
    1
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    2
    0
    1
    Skin and subcutaneous tissue disorders
    Alopecia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Ecchymosis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Erythema
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Keloid scar
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Pruritus
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Rash
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    0
    0
    3
    Rash pruritic
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Skin lesion
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    2
    Back pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Groin pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Musculoskeletal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Pain in extremity
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    2
    1
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Ear infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    1
    0
    Impetigo
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    4 / 6 (66.67%)
    3 / 4 (75.00%)
    0 / 5 (0.00%)
         occurrences all number
    7
    6
    0
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 4 (0.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    0
    Sinusitis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Tinea versicolour
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    0 / 5 (0.00%)
         occurrences all number
    0
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 4 (25.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    2
    1
    Wound infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 4 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Dec 2014
    Amendment 1
    12 Oct 2015
    Amendment 2

    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
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