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    Clinical Trial Results:
    Pharmacodynamic Studies of a Histone Deacetylase Inhibitor in FRDA

    Summary
    EudraCT number
    		 2011-002744-27
    Trial protocol
    		 GB  
    Global end of trial date
    30 Jun 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Oct 2022
    First version publication date
    15 Oct 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CR01849
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Imperial College London
    Sponsor organisation address
    Hammersmith Campus, Hammersmith Hospital, Du Dane Road, , London, United Kingdom, W12 0NN
    Public contact
    Festenstein, Imperial College London, r.festenstein@imperial.ac.uk
    Scientific contact
    Festenstein, Imperial College London, r.festenstein@imperial.ac.uk
    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
    30 Jun 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Jun 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Jun 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary aim is to determine whether Nicotinamide is effective at upregulating the Frataxin (FXN) gene in patients with Friedreich’s ataxia (FRDA) where this gene is abnormally 'switched off'.
    Protection of trial subjects
    N/A
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Aug 2011
    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 Kingdom: 10
    Worldwide total number of subjects
    10
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    10
    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 must have a molecular genetic diagnosis of FRDA, consisting of a GAA-repeat expansion on both alleles of the FXN gene. Exclusion Criteria for the interventional study: Participants with significant clinical dysphagia. Participants taking Sodium Valproate or any other known histone deacetylase inhibitor.

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

    Arms
    Arm title
    		 Nicotinamide
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Nicotinamide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Dose escalation study up to 0.5g to 8g

    Number of subjects in period 1
    		Nicotinamide
    Started
    10
    Completed
    10

    Baseline characteristics

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

    Reporting group values
    		 Overall study Total
    Number of subjects
    		 10 10
    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)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 10 10
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Gender categorical
    Units: Subjects
        Female
    		 6 6
        Male
    		 4 4

    End points

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

    Primary: Significant upregulation of Frataxin in patients

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    End point title
    		 Significant upregulation of Frataxin in patients [1]
    End point description
    End point type
    Primary
    End point timeframe
    Daily administration up to 9 weeks
    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: Bayesian statistical analysis see Libri et al 2014 Lancet.
    End point values
    		 Nicotinamide
    Number of subjects analysed
    10 [2]
    Units: 1.8
        arithmetic mean (confidence interval 95%)
    1.8 (1.5 to 2.2)
    Notes
    [2] - The mean is a fold-increase
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    1 year
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    1.0
    Reporting groups
    Reporting group title
    Nicotinamide
    Reporting group description
    		 -

    Serious adverse events
    		 Nicotinamide
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 10 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Nicotinamide
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 10 (100.00%)
    Hepatobiliary disorders
    Nausea
         subjects affected / exposed
    10 / 10 (100.00%)
         occurrences all number
    10

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Apr 2014
    Essentially the trial was an adapative design and each step was implemented as a substantial ammendment.

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/24794816
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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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