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    Clinical Trial Results:
    A Phase 2a, multi-center, single-blind, within-subject, placebo-controlled study to assess the pharmacodynamics of ACT-709478 in subjects with photosensitive epilepsy

    Summary
    EudraCT number
    2017-000494-36
    Trial protocol
    DE   FR  
    Global end of trial date
    25 Apr 2018

    Results information
    Results version number
    v2(current)
    This version publication date
    07 Nov 2019
    First version publication date
    25 May 2019
    Other versions
    v1
    Version creation reason
    • Correction of full data set
    Change of Sponsor.

    Trial information

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    Trial identification
    Sponsor protocol code
    AC-083-103
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03239691
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    ​Idorsia Pharmaceuticals Ltd
    Sponsor organisation address
    ​Hegenheimermattweg 91, Allschwil, Switzerland, 4123
    Public contact
    Clinical Trial Disclosure Desk, Idorsia Pharmaceuticals Ltd, clinical-trials-disclosure@idorsia.com
    Scientific contact
    Clinical Trial Disclosure Desk, Idorsia Pharmaceuticals Ltd, clinical-trials-disclosure@idorsia.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
    22 Nov 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Apr 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the pharmacodynamics by means of the change in intermittent photic stimulation induced photoparoxysmal response in male and female subjects with photosensitive epilepsy following single dose administration of ACT-709478.
    Protection of trial subjects
    Prior to the start of the study and implementation of the amendments, Independent Ethics Committees were consulted, i.e., review panels that were responsible for ensuring the protection of the rights, safety, and well being of human subjects involved in a clinical investigation.
    Background therapy
    Subjects were allowed to be on stable background treatment (i.e., no dose changes within 4 weeks prior to screening and no changes foreseen during the study) with a maximum of 2 concomitant antiepileptic drugs. During the whole study duration, they received the antiepileptic drugs according to their regular administration schedule.
    Evidence for comparator
    -
    Actual start date of recruitment
    06 Oct 2017
    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
    Germany: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    5
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    5
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study was conducted in 2 countries and 5 sites

    Pre-assignment
    Screening details
    A screening evaluation was performed within 3–28 days before first study treatment administration for male subjects and female subjects of non-childbearing potential and within 10–28 days before first study treatment administration for female subjects of childbearing potential.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Single blind
    Roles blinded
    Subject
    Blinding implementation details
    For safety reasons, this study was conducted in a single-blind fashion. The investigator and study site personnel, the monitors, and the sponsor knew on which study days placebo or ACT-709478 was administered. In contrast, the subjects remained blinded to the study treatment until study closure. The investigational treatment and its matching placebo were indistinguishable.

    Arms
    Arm title
    ACT-709478 single-dose / placebo administration
    Arm description
    Each subject was to receive both placebo and a single dose of active treatment on consecutive days. Placebo was to be administered in the morning of Day 1 and Day 3, and ACT-709478 in the morning of Day 2.
    Arm type
    Experimental

    Investigational medicinal product name
    ACT-709478
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Hard gelatin capsules for oral administration formulated at a strength of 100 mg.

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    Matching placebo available as matching capsules for oral administration.

    Number of subjects in period 1
    ACT-709478 single-dose / placebo administration
    Started
    5
    Completed
    4
    Not completed
    1
         Treatment discontinuation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    -

    Reporting group values
    Overall trial Total
    Number of subjects
    5 5
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    35.6 (19 to 57) -
    Gender categorical
    Units: Subjects
        Female
    5 5
    BMI
    Units: kg/m2
        arithmetic mean (full range (min-max))
    25.8 (23.3 to 29.2) -

    End points

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    End points reporting groups
    Reporting group title
    ACT-709478 single-dose / placebo administration
    Reporting group description
    Each subject was to receive both placebo and a single dose of active treatment on consecutive days. Placebo was to be administered in the morning of Day 1 and Day 3, and ACT-709478 in the morning of Day 2.

    Primary: Positive response described as complete suppression of photoparoxysmal response or a clinically relevant reduction in the standardized photosensitive range

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    End point title
    Positive response described as complete suppression of photoparoxysmal response or a clinically relevant reduction in the standardized photosensitive range [1]
    End point description
    End point type
    Primary
    End point timeframe
    From Day 2 to Day 10
    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: See version 1.
    End point values
    ACT-709478 single-dose / placebo administration
    Number of subjects analysed
    3
    Units: Positive response
        Subject 1
    0
        Subject 2
    1
        Subject 3
    0
    No statistical analyses for this end point

    Secondary: Time (hours) to onset of positive response

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    End point title
    Time (hours) to onset of positive response
    End point description
    Defined by the first time point after ACT-709478 administration at which complete suppression of PPR or reduction in SPR ≥ 3 units compared to baseline is achieved at least at 2 consecutive time points.
    End point type
    Secondary
    End point timeframe
    From Day 2 to Day 10
    End point values
    ACT-709478 single-dose / placebo administration
    Number of subjects analysed
    3
    Units: Hours
        Subject 2 - Eye closure
    55
    No statistical analyses for this end point

    Secondary: Duration (hours) of positive response

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    End point title
    Duration (hours) of positive response
    End point description
    Defined as the time elapsed between the time point of onset of the positive response and the last time point of the positive response after ACT-709478 administration
    End point type
    Secondary
    End point timeframe
    From Day 2 to Day 10
    End point values
    ACT-709478 single-dose / placebo administration
    Number of subjects analysed
    3
    Units: Hours
        Subject 2 - Eye closure
    143
    No statistical analyses for this end point

    Secondary: Maximum SPR reduction

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    End point title
    Maximum SPR reduction
    End point description
    Defined as the largest reduction in SPR achieved at any time point compared to baseline during the positive response after ACT-709478 administration
    End point type
    Secondary
    End point timeframe
    From Day 2 to Day 10
    End point values
    ACT-709478 single-dose / placebo administration
    Number of subjects analysed
    3
    Units: SPR reduction
        Subject 2 - Eye closure
    5
    No statistical analyses for this end point

    Secondary: Time (hours) to maximum SPR reduction

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    End point title
    Time (hours) to maximum SPR reduction
    End point description
    End point type
    Secondary
    End point timeframe
    From Day 2 to Day 10
    End point values
    ACT-709478 single-dose / placebo administration
    Number of subjects analysed
    3
    Units: Hours
        Subject 2 - Eye closure
    127
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events treatment-emergent for placebo (from placebo administration on Day 1 up to ACT-709478 administration on Day 2) and ACT-709478 (from ACT-709478 administration on Day 2 up to EOS).
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    ACT-709478
    Reporting group description
    -

    Reporting group title
    Placebo
    Reporting group description
    -

    Serious adverse events
    ACT-709478 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Nervous system disorders
    generalized tonic-clonic seizure
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (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
    ACT-709478 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    3 / 5 (60.00%)
    Injury, poisoning and procedural complications
    Post procedural discomfort
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    4
    0
    Disturbance in attention
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Headache
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Hyperaesthesia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Myoclonus
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Ear and labyrinth disorders
    Hyperacusis
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Application site hypersensitivity
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Chills
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Feeling of body temperature change
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Catheter site haematoma
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Pre-existing condition improved
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Tension
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    2
    Euphoric mood
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Initial insomnia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Nightmare
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Jul 2017
    Amendment 1 was a substantial amendment finalized before the start of the clinical conduct of the study. This amendment was issued in response to two requests expressed by BfArM: 1) The inclusion of the submission of a protocol amendment in the procedure to continue the study, e.g., with an intermediate dose, if a stopping criterion is met for dose escalation. 2) The C-SSRS was added in order to exclude subjects with a history of suicidal thoughts or attempted suicide and to monitor the subjects for signs of suicidal thoughts or suicidal behavior during the course of the study.
    28 Nov 2017
    Amendment 2 specified the change of sponsorship of the study from Actelion Pharmaceuticals Ltd to Idorsia Pharmaceuticals Ltd, effective from 1 March 2018

    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.
    Due to the late occurrence, the positive response in one subject was not considered relevant.
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