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    Clinical Trial Results:
    Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation

    Summary
    EudraCT number
    		 2019-001493-28
    Trial protocol
    		 AT  
    Global end of trial date
    25 Apr 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Apr 2024
    First version publication date
    27 Apr 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    OPA1
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Medical University of Graz
    Sponsor organisation address
    Neue Stiftingtalstraße 6, Graz, Austria, 8010
    Public contact
    Clinical trials information, Medizinische Universität Graz, Univ.-Augenklinik, 0043 0316385 82899, katharina.valentin@medunigraz.at
    Scientific contact
    Clinical trials information, Medizinische Universität Graz, Univ.-Augenklinik, 0043 0316385 82899, katharina.valentin@medunigraz.at
    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
    26 Apr 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Apr 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Apr 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluation of the therapeutic effect of 900mg Raxone® per day regarding visual acuity in ADOA patients with OPA1 mutation within a 12 month period.
    Protection of trial subjects
    The study was conducted according to GCP and local regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Oct 2020
    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
    Austria: 16
    Worldwide total number of subjects
    16
    EEA total number of subjects
    16
    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)
    2
    Adults (18-64 years)
    14
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Recruitment period was between 10/2020 and 05/2021

    Pre-assignment
    Screening details
    		 16 patients have signed the informed consent, 1 patient was lost to follow-up. There were no screening failures.

    Period 1
    Period 1 title
    overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Raxone
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Raxone 150 mg film-coated tablets
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    150 mg 3x2 daily for 12 months

    Number of subjects in period 1
    		Raxone
    Started
    16
    Completed
    15
    Not completed
    1
         Lost to follow-up
    1

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Raxone
    Reporting group description
    		 -

    Primary: Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the right eye

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    End point title
    		 Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the right eye [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and follow-up
    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: single arm study - was not possible to enter data in the systeme
    End point values
    		 Raxone
    Number of subjects analysed
    16
    Units: logMAR
    arithmetic mean (standard deviation)
        Baseline
    0.52 ( 0.32 )
        Follow-up
    0.44 ( 0.32 )
    No statistical analyses for this end point

    Primary: Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the left eye

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    End point title
    		 Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the left eye [2]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and follow-up
    Notes
    [2] - 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: single arm study - was not possible to enter data in the systeme
    End point values
    		 Raxone
    Number of subjects analysed
    16
    Units: logMAR
    arithmetic mean (standard deviation)
        Baseline
    0.54 ( 0.36 )
        Follow-up
    0.48 ( 0.38 )
    No statistical analyses for this end point

    Primary: Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the better-seeing eye

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    End point title
    		 Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts on the better-seeing eye [3]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline Follow-up
    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: single arm study - was not possible to enter data in the systeme
    End point values
    		 Raxone
    Number of subjects analysed
    16
    Units: logMAR
    arithmetic mean (standard deviation)
        Baseline
    0.46 ( 0.32 )
        Follow-up
    0.41 ( 0.35 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    12 months
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    Raxone
    Reporting group description
    		 -

    Serious adverse events
    		 Raxone
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Raxone
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 16 (56.25%)
    General disorders and administration site conditions
    Headache
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Anorexia
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Sore throat
         subjects affected / exposed
    1 / 16 (6.25%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    anemia
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Elevation of liver parameters
         subjects affected / exposed
    2 / 16 (12.50%)
         occurrences all number
    2
    Gastrointestinal disorders
    Pyrosis
         subjects affected / exposed
    1 / 16 (6.25%)
         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.
    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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