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    Clinical Trial Results:
    A Phase Ib, Multicenter, Open-Label, 6-Week Study With a 48-Week Extension to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children Ages 2-4 Years With Autism Spectrum Disorder

    Summary
    EudraCT number
    		 2019-000989-38
    Trial protocol
    		 ES  
    Global end of trial date
    06 May 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Oct 2020
    First version publication date
    07 Oct 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    WP40877
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04049578
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Hoffmann-La Roche
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, CH-4070
    Public contact
    F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com
    Scientific contact
    F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.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
    06 May 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 May 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to evaluate the pharmacokinetics, safety, andtolerability of 4 mg balovaptan once a day administered for 6 weeks to children 2-4 years old with Autism Spectrum Disorder.
    Protection of trial subjects
    All study subjects were required to read and sign an Informed Consent Form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    19 Dec 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 2
    Worldwide total number of subjects
    2
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    2
    Adolescents (12-17 years)
    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
    		 Participants in this study included children 2-4 years old with autism spectrum disorder (ASD).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Balovaptan
    Arm description
    		 Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Balovaptan
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Dispersible tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Balovaptan dispersible tablet was administered once a day (QD) orally.

    Number of subjects in period 1
    		Balovaptan
    Started
    2
    Completed
    1
    Not completed
    1
         Physician decision
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Balovaptan
    Reporting group description
    		 Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.

    Reporting group values
    		 Balovaptan Total
    Number of subjects
    		 2 2
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 2 2
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 3.45 ( 0.78 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 0 0
        Male
    		 2 2
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 2 2
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 1 1
        Not Hispanic or Latino
    		 1 1
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    Balovaptan
    Reporting group description
    		 Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.

    Primary: Area Under the Curve at Steady State (AUCss) of Balovaptan

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    End point title
    		 Area Under the Curve at Steady State (AUCss) of Balovaptan [1]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [2]
    Units: day*ug/mL
        arithmetic mean (standard deviation)
    ( )
    Notes
    [2] - Due to low enrollment number, analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Primary: Plasma Concentration of Balovaptan

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    End point title
    		 Plasma Concentration of Balovaptan [3]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    Notes
    [3] - 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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [4]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    Notes
    [4] - Due to low enrollment, number analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Primary: Plasma Concentration of M2 Metabolite, as Applicable

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    End point title
    		 Plasma Concentration of M2 Metabolite, as Applicable [5]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    Notes
    [5] - 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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [6]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    Notes
    [6] - Due to low enrollment number, analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Primary: Plasma Concentation of M3 Metabolite

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    End point title
    		 Plasma Concentation of M3 Metabolite [7]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    Notes
    [7] - 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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [8]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    Notes
    [8] - Due to low enrollment number, analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Primary: Plasma Concentration Ratio of M2 to Balovaptan, as Applicable

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    End point title
    		 Plasma Concentration Ratio of M2 to Balovaptan, as Applicable [9]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    Notes
    [9] - 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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [10]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    Notes
    [10] - Due to low enrollment number, analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Primary: Plasma Concentration Ratio of M3 to Balovaptan

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    End point title
    		 Plasma Concentration Ratio of M3 to Balovaptan [11]
    End point description
    End point type
    Primary
    End point timeframe
    Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
    Notes
    [11] - 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 analysis for this end point.
    End point values
    		 Balovaptan
    Number of subjects analysed
    0 [12]
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    ( )
    Notes
    [12] - Due to low enrollment number, analysis are not provided to protect participant confidentiality.
    No statistical analyses for this end point

    Secondary: Number of Participants With Adverse Events

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    End point title
    		 Number of Participants With Adverse Events
    End point description
    End point type
    Secondary
    End point timeframe
    Up to approximately week 20
    End point values
    		 Balovaptan
    Number of subjects analysed
    2
    Units: Participants
        number (not applicable)
    2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
    Adverse event reporting additional description
    The safety population is defined as patients who received any amount of study treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Balovaptan
    Reporting group description
    		 Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.

    Serious adverse events
    		 Balovaptan
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Balovaptan
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 2 (100.00%)
    Investigations
    Blood thyroid stimulating hormone increased
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences all number
    1
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences all number
    1
    Nervous system disorders
    Psychomotor hyperactivity
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Abnormal weight gain
         subjects affected / exposed
    1 / 2 (50.00%)
         occurrences all number
    1

    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.
    Study was terminated. Recent analysis of Phase II balovaptan data in paediatric ASD did not support the continuation of this study. No new safety concerns were identified.
    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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