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    Clinical Trial Results:
    A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD).

    Summary
    EudraCT number
    		 2019-002076-13
    Trial protocol
    		 GB   SE   ES   NL   GR   IT   NO  
    Global end of trial date
    19 Oct 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Nov 2024
    First version publication date
    16 Nov 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    NS-065/NCNP-01-301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04060199
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 US IND: 127474
    Sponsors
    Sponsor organisation name
    NS Pharma, Inc.
    Sponsor organisation address
    140 East Ridgewood Avenue Suite 280s, Paramus, United States, 07652-3914
    Public contact
    Legal team, NS Pharma, Inc. , +1 2019863860, legal@nspharma.com
    Scientific contact
    Tial information team, NS Pharma, Inc. , +1 8666774276, trialinfo@nspharma.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
    06 Dec 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Oct 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Oct 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To compare the efficacy of viltolarsen administered intravenously at a weekly dose of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using Time to Stand Test from supine (TTSTAND) as a measure of strength and function.
    Protection of trial subjects
    The study was conducted in accordance with the Declaration of Helsinki and with all applicable laws and regulations of the locale and country where the study was conducted, and in compliance with Good Clinical Practice Guidelines.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    14 Apr 2020
    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
    Ukraine: 1
    Country: Number of subjects enrolled
    Türkiye: 11
    Country: Number of subjects enrolled
    Korea, Republic of: 6
    Country: Number of subjects enrolled
    Russian Federation: 10
    Country: Number of subjects enrolled
    New Zealand: 1
    Country: Number of subjects enrolled
    Mexico: 2
    Country: Number of subjects enrolled
    Japan: 1
    Country: Number of subjects enrolled
    China: 12
    Country: Number of subjects enrolled
    Chile: 3
    Country: Number of subjects enrolled
    Canada: 2
    Country: Number of subjects enrolled
    Australia: 1
    Country: Number of subjects enrolled
    Netherlands: 10
    Country: Number of subjects enrolled
    Norway: 2
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 5
    Country: Number of subjects enrolled
    Greece: 3
    Country: Number of subjects enrolled
    Italy: 4
    Worldwide total number of subjects
    77
    EEA total number of subjects
    22
    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)
    77
    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
    		 Screening includes assessments to confirm eligibility (review of inclusion/exclusion criteria and review to confirm the DMD diagnosis and appropriate mutations).

    Period 1
    Period 1 title
    Treatment and follow up (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Viltolarsen
    Arm description
    		 All randomized subjects who received 80 mg/kg Viltolarsen injection once weekly over a 48-week period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Viltolarsen
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received 80 mg/kg Viltolarsen intravenous infusions once weekly over a 48-week period.

    Arm title
    		 Placebo
    Arm description
    		 All randomized subjects who received saline solution as intravenous infusions administered once weekly over a 48-week period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects received saline solution as intravenous infusions administered once weekly over a 48-week period.

    Number of subjects in period 1
    		Viltolarsen Placebo
    Started
    38
    39
    Completed
    36
    38
    Not completed
    2
    1
         Patient Relocation
    1
    -
         Protocol deviation
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Viltolarsen
    Reporting group description
    		 All randomized subjects who received 80 mg/kg Viltolarsen injection once weekly over a 48-week period.

    Reporting group title
    Placebo
    Reporting group description
    		 All randomized subjects who received saline solution as intravenous infusions administered once weekly over a 48-week period.

    Reporting group values
    		 Viltolarsen Placebo Total
    Number of subjects
    		 38 39 77
    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)
    		 38 39 77
        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
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 38 39 77

    End points

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    End points reporting groups
    Reporting group title
    Viltolarsen
    Reporting group description
    		 All randomized subjects who received 80 mg/kg Viltolarsen injection once weekly over a 48-week period.

    Reporting group title
    Placebo
    Reporting group description
    		 All randomized subjects who received saline solution as intravenous infusions administered once weekly over a 48-week period.

    Subject analysis set title
    Viltolarsen mITT population
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All randomized patients who received at least 1 dose of Viltolarsen and had a baseline assessment and at least 1 post baseline efficacy assessment.

    Subject analysis set title
    Placebo mITT population
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    All randomized patients who received at least 1 dose of placebo and had a baseline assessment and at least 1 post baseline efficacy assessment.

    Primary: Primary: Change from baseline in Time to Stand

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    End point title
    		 Primary: Change from baseline in Time to Stand
    End point description
    End point type
    Primary
    End point timeframe
    49 weeks
    End point values
    		 Viltolarsen mITT population Placebo mITT population
    Number of subjects analysed
    33
    35
    Units: Velocity
        least squares mean (standard error)
    0.009 ( 0.0096 )
    0.013 ( 0.0096 )
    Statistical analysis title
    		 Primary analysis: Time to Stand
    Comparison groups
    Viltolarsen mITT population v Placebo mITT population
    Number of subjects included in analysis
    68
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    		 mixed-effect model for repeated measures
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.004
    Confidence interval
         level
    		 95.1%
         sides
    2-sided
         lower limit
    		 -0.03
         upper limit
    		 0.022
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.013

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All AEs occurring during the course of the study (starting from signing informed consent to study completion) until the follow-up telephone call, 30 days following the last administration of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Viltolarsen
    Reporting group description
    		 All randomized subjects who received 80 mg/kg Viltolarsen injection once weekly over a 48-week period.

    Reporting group title
    Placebo
    Reporting group description
    		 All randomized subjects who received saline solution as intravenous infusions administered once weekly over a 48-week period.

    Serious adverse events
    		 Viltolarsen Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 38 (5.26%)
    3 / 39 (7.69%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Epilepsy
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Adverse drug reaction
    Additional description: Prolonged hospitalization after zoledronate infusion: nausea, dizziness, vomiting, and fever.
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 39 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Influenza
         subjects affected / exposed
    0 / 38 (0.00%)
    1 / 39 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pharyngitis
         subjects affected / exposed
    1 / 38 (2.63%)
    0 / 39 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Viltolarsen Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    37 / 38 (97.37%)
    33 / 39 (84.62%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    10 / 38 (26.32%)
    13 / 39 (33.33%)
         occurrences all number
    12
    19
    Fatigue
         subjects affected / exposed
    3 / 38 (7.89%)
    1 / 39 (2.56%)
         occurrences all number
    3
    1
    Influenza like illness
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 39 (5.13%)
         occurrences all number
    2
    3
    Administration site extravasation
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    12 / 38 (31.58%)
    5 / 39 (12.82%)
         occurrences all number
    15
    7
    Rhinorrhoea
         subjects affected / exposed
    6 / 38 (15.79%)
    2 / 39 (5.13%)
         occurrences all number
    7
    2
    Rhinitis allergic
         subjects affected / exposed
    3 / 38 (7.89%)
    1 / 39 (2.56%)
         occurrences all number
    4
    1
    Investigations
    Blood triglycerides increased
         subjects affected / exposed
    4 / 38 (10.53%)
    2 / 39 (5.13%)
         occurrences all number
    6
    2
    Protein urine present
         subjects affected / exposed
    2 / 38 (5.26%)
    3 / 39 (7.69%)
         occurrences all number
    3
    5
    Blood cholesterol increased
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 39 (5.13%)
         occurrences all number
    4
    2
    Beta 2 microglobulin urine increased
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    3
    Blood fibrinogen increased
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 39 (2.56%)
         occurrences all number
    2
    1
    Crystal urine present
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    3
    Urine protein/creatinine ratio increased
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 39 (2.56%)
         occurrences all number
    3
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    5 / 38 (13.16%)
    4 / 39 (10.26%)
         occurrences all number
    6
    4
    Contusion
         subjects affected / exposed
    3 / 38 (7.89%)
    1 / 39 (2.56%)
         occurrences all number
    4
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    3 / 38 (7.89%)
    2 / 39 (5.13%)
         occurrences all number
    3
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 38 (7.89%)
    5 / 39 (12.82%)
         occurrences all number
    3
    8
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    3 / 38 (7.89%)
    1 / 39 (2.56%)
         occurrences all number
    4
    3
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    9 / 38 (23.68%)
    7 / 39 (17.95%)
         occurrences all number
    10
    13
    Vomiting
         subjects affected / exposed
    11 / 38 (28.95%)
    3 / 39 (7.69%)
         occurrences all number
    12
    3
    Abdominal pain
         subjects affected / exposed
    4 / 38 (10.53%)
    6 / 39 (15.38%)
         occurrences all number
    10
    11
    Nausea
         subjects affected / exposed
    1 / 38 (2.63%)
    3 / 39 (7.69%)
         occurrences all number
    1
    4
    Abdominal pain upper
         subjects affected / exposed
    2 / 38 (5.26%)
    1 / 39 (2.56%)
         occurrences all number
    2
    1
    Constipation
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    2
    Abdominal discomfort
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Faeces soft
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 38 (2.63%)
    3 / 39 (7.69%)
         occurrences all number
    1
    3
    Eczema
         subjects affected / exposed
    3 / 38 (7.89%)
    0 / 39 (0.00%)
         occurrences all number
    3
    0
    Dermatitis allergic
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 39 (0.00%)
         occurrences all number
    2
    0
    Pruritus
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 39 (5.13%)
         occurrences all number
    2
    3
    Urine abnormality
         subjects affected / exposed
    4 / 38 (10.53%)
    0 / 39 (0.00%)
         occurrences all number
    5
    0
    Pyelocaliectasis
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    2
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    3 / 38 (7.89%)
    2 / 39 (5.13%)
         occurrences all number
    4
    2
    Back pain
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    3
    Arthralgia
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    3
    Myalgia
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    4
    Infections and infestations
    COVID-19
         subjects affected / exposed
    12 / 38 (31.58%)
    12 / 39 (30.77%)
         occurrences all number
    13
    13
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 38 (26.32%)
    9 / 39 (23.08%)
         occurrences all number
    18
    13
    Nasopharyngitis
         subjects affected / exposed
    9 / 38 (23.68%)
    7 / 39 (17.95%)
         occurrences all number
    25
    16
    Rhinitis
         subjects affected / exposed
    6 / 38 (15.79%)
    3 / 39 (7.69%)
         occurrences all number
    10
    5
    Gastroenteritis
         subjects affected / exposed
    3 / 38 (7.89%)
    4 / 39 (10.26%)
         occurrences all number
    4
    4
    Influenza
         subjects affected / exposed
    1 / 38 (2.63%)
    3 / 39 (7.69%)
         occurrences all number
    1
    3
    Urinary tract infection
         subjects affected / exposed
    2 / 38 (5.26%)
    2 / 39 (5.13%)
         occurrences all number
    3
    2
    Bronchitis
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    2
    Hordeolum
         subjects affected / exposed
    0 / 38 (0.00%)
    3 / 39 (7.69%)
         occurrences all number
    0
    3
    Gastroenteritis viral
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Pharyngitis
         subjects affected / exposed
    2 / 38 (5.26%)
    0 / 39 (0.00%)
         occurrences all number
    2
    0
    Pneumonia
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    3
    Tonsillitis
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Varicella
         subjects affected / exposed
    0 / 38 (0.00%)
    2 / 39 (5.13%)
         occurrences all number
    0
    2
    Metabolism and nutrition disorders
    Hypercholesterolaemia
         subjects affected / exposed
    1 / 38 (2.63%)
    2 / 39 (5.13%)
         occurrences all number
    1
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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    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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