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    Clinical Trial Results:
    A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy

    Summary
    EudraCT number
    2017-004139-35
    Trial protocol
    ES   BG   IT  
    Global end of trial date
    25 Feb 2020

    Results information
    Results version number
    v2(current)
    This version publication date
    18 Feb 2021
    First version publication date
    06 Sep 2020
    Other versions
    v1
    Version creation reason
    • Correction of full data set
    Correction of typing error: For non-serious adverse events, 5% reporting threshold is corrected to 3% reporting threshold

    Trial information

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    Trial identification
    Sponsor protocol code
    MNK14112096
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03400852
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Mallinckrodt ARD LLC
    Sponsor organisation address
    1425 U.S. Route 206, Bedminster, NJ, United States, 07921
    Public contact
    Medical Information Call Center, Mallinckrodt ARD LLC, 1 1 800-844-2830 Ext 5, clinicaltrials@mnk.com
    Scientific contact
    Medical Information Call Center, Mallinckrodt ARD LLC, 1 1 800-844-2830 Ext 5, clinicaltrials@mnk.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 May 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Feb 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main purpose of this study is to determine the effect of MNK-1411 on motor function in subjects with Duchenne Muscular Dystrophy (DMD). Information has been collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI). The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.
    Protection of trial subjects
    The study was conducted in full compliance with applicable international, national and local regulatory requirements; US Food and Drug Administration (FDA) regulations including 21 CFR 314.106 and 312.120, (where applicable); and ICH guidelines for GCP and in accordance with the ethical principles that have their origins in the Declaration of Helsinki.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    27 Jul 2018
    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
    Spain: 6
    Country: Number of subjects enrolled
    Bulgaria: 2
    Country: Number of subjects enrolled
    Israel: 1
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    United States: 10
    Country: Number of subjects enrolled
    Mexico: 18
    Country: Number of subjects enrolled
    Serbia: 2
    Country: Number of subjects enrolled
    Turkey: 3
    Worldwide total number of subjects
    44
    EEA total number of subjects
    10
    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)
    44
    Adolescents (12-17 years)
    0
    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
    After screening, 44 patients were randomized into blinded treatment groups at 16 sites in 8 countries.

    Period 1
    Period 1 title
    Blinded Treatment Period
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer
    Blinding implementation details
    Patients were randomized 2:1 to receive MNK-1411:placebo at the dose appropriate for their weight

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    MNK-1411
    Arm description
    All patients who received any dose of MNK-1411 in Period 1
    Arm type
    Experimental

    Investigational medicinal product name
    Cosyntropin acetate
    Investigational medicinal product code
    MNK-1411
    Other name
    Tetracosactide Hexaacetate
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    MNK-1411 suspension for subcutaneous injection

    Arm title
    Placebo
    Arm description
    All patients who received placebo in Period 1
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Matching placebo
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Matching placebo suspension for subcutaneous administration

    Number of subjects in period 1
    MNK-1411 Placebo
    Started
    29
    15
    Completed
    20
    9
    Not completed
    9
    6
         Physician decision
    -
    1
         Adverse event, non-fatal
    1
    1
         Study terminated by sponsor
    8
    4
    Period 2
    Period 2 title
    Open Label Period
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    All patients entering open label period received MNK-1411

    Arms
    Arm title
    MNK-1411
    Arm description
    All patients who entered the open label extension period receive MNK-1411
    Arm type
    Experimental

    Investigational medicinal product name
    Cosyntropin acetate
    Investigational medicinal product code
    MNK-1411
    Other name
    Tetracosactide Hexaacetate
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    MNK-1411 1 mg/ml [milligram(s)/milliliter] suspension for subcutaneous injection

    Number of subjects in period 2 [1]
    MNK-1411
    Started
    24
    Completed
    2
    Not completed
    22
         Physician decision
    1
         Study terminated by sponsor
    21
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Patients who chose to discontinue double-blind period prior to Week 24, entered OLE period. Patients who did not enter OLE Period were followed-up to Week 28.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    MNK-1411
    Reporting group description
    All patients who received any dose of MNK-1411 in Period 1

    Reporting group title
    Placebo
    Reporting group description
    All patients who received placebo in Period 1

    Reporting group values
    MNK-1411 Placebo Total
    Number of subjects
    29 15 44
    Age categorical
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0
        Children (2-11 years)
    29 15 44
        Adolescents (12-17 years)
    0 0 0
        Adults (18-64 years)
    0 0 0
        From 65-84 years
    0 0 0
        85 years and over
    0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    5.7 ( 1.49 ) 6.3 ( 1.18 ) -
    Gender categorical
    Units: Subjects
        Female
    0 0 0
        Male
    29 15 44

    End points

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    End points reporting groups
    Reporting group title
    MNK-1411
    Reporting group description
    All patients who received any dose of MNK-1411 in Period 1

    Reporting group title
    Placebo
    Reporting group description
    All patients who received placebo in Period 1
    Reporting group title
    MNK-1411
    Reporting group description
    All patients who entered the open label extension period receive MNK-1411

    Primary: Time to Complete 10 Meter Walk/Run

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    End point title
    Time to Complete 10 Meter Walk/Run [1]
    End point description
    10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
    End point type
    Primary
    End point timeframe
    Baseline, Week 24
    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: No statistical analyses were performed to arrive at these descriptive statistics
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    29
    15
    Units: Seconds
    median (full range (min-max))
        at Baseline
    5.9 (4.7 to 22.3)
    7.8 (3.9 to 13.0)
        at Week 24 (n=16,9)
    5.4 (4.1 to 8.9)
    8.7 (3.3 to 18.3)
    No statistical analyses for this end point

    Secondary: North Star Ambulatory Assessment (NSAA) Score

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    End point title
    North Star Ambulatory Assessment (NSAA) Score
    End point description
    The NSAA is comprised of 17 items, each of which is graded using the standard scorecard. Each assessment is rated as 0 - unable to achieve independently, 1 - modified method but achieves goal independent of physical assistance from another, or 2 - normal with no obvious modification of activity. The subscale scores are summed for a total score ranging from 0 to 34. The higher the total score, the better the outcome.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    29
    15
    Units: score on a scale
    arithmetic mean (standard deviation)
        at Baseline
    17.9 ( 6.80 )
    17.1 ( 6.40 )
        at Week 24 (n=17,9)
    20.5 ( 7.94 )
    16.6 ( 8.82 )
    No statistical analyses for this end point

    Secondary: Time to Climb 4 Standardized Stairs

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    End point title
    Time to Climb 4 Standardized Stairs
    End point description
    Time to climb 4 standardized stairs is a motor performance test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    29
    15
    Units: Seconds
    arithmetic mean (standard deviation)
        at Baseline
    8.528 ( 8.8832 )
    8.467 ( 4.3353 )
        at Week 24 (n=15,9)
    4.711 ( 2.4547 )
    15.087 ( 13.8414 )
    No statistical analyses for this end point

    Secondary: Time to Stand from a Supine Position

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    End point title
    Time to Stand from a Supine Position
    End point description
    Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    23
    13
    Units: Seconds
    arithmetic mean (standard deviation)
        at Baseline
    11.141 ( 9.0756 )
    15.031 ( 12.4518 )
        at Week 24 (n=16,9)
    7.645 ( 4.9939 )
    24.889 ( 26.4763 )
    No statistical analyses for this end point

    Secondary: Quantitative Muscle Testing Scores at Baseline

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    End point title
    Quantitative Muscle Testing Scores at Baseline
    End point description
    Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer.
    End point type
    Secondary
    End point timeframe
    at Baseline
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    25
    15
    Units: Newton
    arithmetic mean (standard deviation)
        Knee flexion
    26.610 ( 14.1697 )
    29.870 ( 14.1697 )
        Knee extension
    28.987 ( 16.2362 )
    26.643 ( 15.2748 )
    No statistical analyses for this end point

    Secondary: Quantitative Muscle Testing Scores at Week 24

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    End point title
    Quantitative Muscle Testing Scores at Week 24
    End point description
    Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    18
    9
    Units: Newtons
    arithmetic mean (standard deviation)
        Knee flexion
    33.636 ( 15.7832 )
    25.267 ( 13.3471 )
        Knee extension
    26.610 ( 14.1697 )
    29.870 ( 15.1330 )
    No statistical analyses for this end point

    Secondary: Summary of Adverse Events in the Blinded Treatment Period

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    End point title
    Summary of Adverse Events in the Blinded Treatment Period
    End point description
    Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
    End point type
    Secondary
    End point timeframe
    within 28 weeks
    End point values
    MNK-1411 Placebo
    Number of subjects analysed
    29
    15
    Units: Patients
        Number of patients exposed
    29
    15
        Number affected by serious adverse events
    0
    1
        Total number affected by non-serious AEs
    22
    15
        Total number of deaths (all causes)
    0
    0
        Number of deaths due to AEs
    0
    0
    No statistical analyses for this end point

    Secondary: Summary of AEs in the Open Label Extension Period

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    End point title
    Summary of AEs in the Open Label Extension Period
    End point description
    Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as AEs
    End point type
    Secondary
    End point timeframe
    within 28 weeks
    End point values
    MNK-1411
    Number of subjects analysed
    24
    Units: Patients
        Number of patients exposed
    24
        Number affected by SAEs
    2
        Total number affected by non-serious AEs
    11
        Total number of deaths (all causes)
    0
        Number of deaths due to AEs
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    within 28 weeks
    Adverse event reporting additional description
    Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessment were reported as AEs
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    MNK-1411 (Blinded Treatment Period)
    Reporting group description
    All patients who received MNK-1411 during the blinded treatment period

    Reporting group title
    Placebo (Blinded Treatment Period)
    Reporting group description
    Patients who received placebo during the blinded treatment period

    Reporting group title
    MNK-1411 (Open Label Extension)
    Reporting group description
    Patients who received MNK-1411 during the open label extension period

    Serious adverse events
    MNK-1411 (Blinded Treatment Period) Placebo (Blinded Treatment Period) MNK-1411 (Open Label Extension)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    2 / 24 (8.33%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Muscle disorder
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rhabdomyolysis
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         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: 3%
    Non-serious adverse events
    MNK-1411 (Blinded Treatment Period) Placebo (Blinded Treatment Period) MNK-1411 (Open Label Extension)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    22 / 29 (75.86%)
    15 / 15 (100.00%)
    11 / 24 (45.83%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Hypertension
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    2
    General disorders and administration site conditions
    Face oedema
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 15 (13.33%)
    0 / 24 (0.00%)
         occurrences all number
    1
    2
    0
    Fatigue
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Injection site bruising
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    1
    1
    0
    Injection site erythema
         subjects affected / exposed
    4 / 29 (13.79%)
    1 / 15 (6.67%)
    1 / 24 (4.17%)
         occurrences all number
    4
    1
    1
    Injection site haematoma
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Injection site haemorrhage
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Injection site induration
         subjects affected / exposed
    4 / 29 (13.79%)
    1 / 15 (6.67%)
    2 / 24 (8.33%)
         occurrences all number
    4
    1
    2
    Injection site irritation
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Injection site mass
         subjects affected / exposed
    1 / 29 (3.45%)
    3 / 15 (20.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    3
    1
    Injection site pain
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    2
    1
    0
    Injection site swelling
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 15 (13.33%)
    1 / 24 (4.17%)
         occurrences all number
    1
    2
    1
    Swelling face
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Allergy to arthropod bite
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    1 / 24 (4.17%)
         occurrences all number
    0
    2
    2
    Respiratory, thoracic and mediastinal disorders
    Bronchial hyperreactivity
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Cough
         subjects affected / exposed
    2 / 29 (6.90%)
    2 / 15 (13.33%)
    0 / 24 (0.00%)
         occurrences all number
    3
    2
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Asthma
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Affect lability
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Depression
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    1
    1
    0
    Mood swings
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Separation anxiety disorder
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    1 / 24 (4.17%)
         occurrences all number
    0
    1
    1
    Investigations
    Weight increased
         subjects affected / exposed
    7 / 29 (24.14%)
    0 / 15 (0.00%)
    3 / 24 (12.50%)
         occurrences all number
    9
    0
    3
    Nerve stimulation test abnormal
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Lower limb fracture
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    Congestive cardiomyopathy
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Sinus tachycardia
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Motor dysfunction
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Psychomotor hyperactivity
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Blood and lymphatic system disorders
    Leukocytosis
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Abdominal pain
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Aphthous ulcer
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Diarrhoea
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    2
    0
    1
    Food poisoning
         subjects affected / exposed
    1 / 29 (3.45%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    1
    1
    0
    Irritable bowel syndrome
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Vomiting
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Erythema
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Hirsutism
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Hypertrichosis
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Seborrhoeic dermatitis
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Swelling face
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Lipohypertrophy
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Glycosuria
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    3
    Endocrine disorders
    Cushing's syndrome
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Cushingoid
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Ear infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 29 (0.00%)
    3 / 15 (20.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    4
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 29 (6.90%)
    1 / 15 (6.67%)
    1 / 24 (4.17%)
         occurrences all number
    2
    1
    1
    Gastroenteritis viral
         subjects affected / exposed
    2 / 29 (6.90%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    2
    0
    0
    Influenza
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Enterobiasis
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Otitis media acute
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Pharyngitis
         subjects affected / exposed
    0 / 29 (0.00%)
    2 / 15 (13.33%)
    0 / 24 (0.00%)
         occurrences all number
    0
    2
    0
    Pharyngotonsillitis
         subjects affected / exposed
    3 / 29 (10.34%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    3
    0
    0
    Scarlet fever
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Tonsillitis bacterial
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Tooth abscess
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 29 (3.45%)
    2 / 15 (13.33%)
    1 / 24 (4.17%)
         occurrences all number
    1
    2
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Viral infection
         subjects affected / exposed
    0 / 29 (0.00%)
    1 / 15 (6.67%)
    0 / 24 (0.00%)
         occurrences all number
    0
    1
    0
    Viral pharyngitis
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Oral herpes
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Upper respiratory tract infection bacterial
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    0
    0
    1
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Hyperphagia
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    0 / 24 (0.00%)
         occurrences all number
    1
    0
    0
    Increased appetite
         subjects affected / exposed
    1 / 29 (3.45%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         occurrences all number
    1
    0
    1
    Hypokalaemia
         subjects affected / exposed
    0 / 29 (0.00%)
    0 / 15 (0.00%)
    1 / 24 (4.17%)
         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
    16 Nov 2017
    Protocol Amendment 1 was developed to address issues raised by the central institutional review board including adding a study drug taper followed by a cosyntropin stimulation test and clarifying parent and/or legal guardian consent. Additional minor changes that do not impact study conduct or subject safety were also made.
    22 Aug 2018
    Protocol Amendment 2 was developed to address the following considerations:  - In a randomized, double-blind, placebo controlled crossover study in patients ages 5 to 8 with Duchenne muscular dystrophy Beenakker et al, 2005 utilized a 2 month washout period between treatment periods with prednisone and placebo and this washout period appeared to be effective. Subsequently a Medical Advisory Board evaluated the current study design and based on their clinical experience recommended allowing the enrollment of subjects who have not received a therapeutic dose of corticosteroids within 2 months prior to the start of the study. - Subjects with asthma are excluded from the study. Therefore, the concomitant use of inhaled corticosteroids (whose approved indication is for asthma) should not be permitted.
    23 May 2019
    To accommodate subject needs, Protocol Amendment 3 was developed primarily to extend the Open Label Extension Period beyond Week 52 (to continue until the subject chooses to discontinue treatment, the investigator feels that treatment is no longer indicated, MNK-1411 is approved and marketed, or the sponsor ceases development of this compound for Duchenne Muscular Dystrophy [DMD]). Open label visits will continue every 12 weeks, with dispensing of study drug every 12 weeks. Along with this change, text has been added stating that if a subject requires a switch in dose during the Open Label Extension Period (eg, switch to low dose due to being unable to tolerate high dose, or switch to high dose based on increased weight [eg, as subject grows with age]), the investigator should consult with the medical monitor. Since subjects will be participating in the study longer, measurements of height have been added to all study visits. Additional major protocol changes are summarized below: -To accommodate the needs of study sites in Israel, the combination of Sunday and Wednesday has been added as possible visit and/or dosage administration days. -Text has been added specifying that subjects who have a hypersensitivity reaction to the study drug are not required to undergo the 2-week taper, or standard cosyntropin stimulation test (250 μg) at the follow-up visit. -Varicella zoster testing has been added along with an inclusion criterion (Inclusion criterion 6) requiring subjects to test positive prior to study entry (since such infections may be serious in patients who are immunosuppressed). -Pharmacokinetic analysis text has been corrected. -Additional guidance on the administration of motor tests has been provided, along with modification of Exclusion criterion 7 to require that subjects be able to complete the 10 Meter Walk/Run test in 10 seconds or less at the Screening and Baseline Visits.

    Interruptions (globally)

    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    25 Feb 2020
    The trial was permanently discontinued because of slow enrollment.
    -

    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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