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    Clinical Trial Results:
    A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3)

    Summary
    EudraCT number
    		 2014-005112-42
    Trial protocol
    		 FR   NL  
    Global end of trial date
    02 Jan 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    21 Apr 2022
    First version publication date
    21 Apr 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    C14-62
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    INSERM
    Sponsor organisation address
    8, rue de la croix Jarry, Paris, France, 75013
    Public contact
    Sonia GUEGUEN, INSERM, 33 144236041, rqrc.siege@inserm.fr
    Scientific contact
    Sonia GUEGUEN, INSERM, 33 144236041, rqrc.siege@inserm.fr
    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
    20 Apr 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    02 Dec 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Jan 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    the primary objectice is to evaluate the efficacy of triheptanoin in - increasing the energy response in the metabolic profile of the brain of early affected HD patients , as captured by 31-Phosphorus Magnetic Resonance Spectroscopy - slowing atrophy in the caudate of early affected HD patients as measured with volumetric resonance imaging
    Protection of trial subjects
    Trial was performed as described on the CPP (Committee for people's protection) decision #33-15 .
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 May 2015
    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
    Netherlands: 48
    Country: Number of subjects enrolled
    France: 52
    Worldwide total number of subjects
    100
    EEA total number of subjects
    100
    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)
    100
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 TRIHEP 3 is a multi-centre (Paris and Leiden) randomized, double-blind, controlled study recruiting 100 early HD patients. Patients will receive either triheptanoin or a placebo for 6 months followed by a 6 month open-label phase with triheptanoin. At the end of the open-label phase, an extension period of 1 year may be proposed.

    Pre-assignment
    Screening details
    		 A screening visit will be conducted in which information about the study will be provided and patients will have the opportunity to ask any questions. Inclusion/non-inclusion criteria including the ability to undergo MRI scanning will be verified to confirm the patient’s eligibility for the study.

    Period 1
    Period 1 title
    Full study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator
    Blinding implementation details
    To ensure acceptability for patients, we conducted a 6-month randomized controlled bi-centric trial (Paris and Leiden) called TRIHEP3 (NCT02453061), comparing triheptanoin 1g/kg/day vs placebo in 100 patients (ratio 1/1) at an early stage of HD, followed by a 6-month open label phase. After one year, patients could opt for a one-year extension study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Active
    Arm description
    		 triheptanoin treated arm
    Arm type
    		 Active comparator

    Investigational medicinal product name
    triheptanoin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral liquid, Oral solution, Oral solution in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    triheptanoin 1g/kg/day

    Arm title
    		 Comparator arm
    Arm description
    		 To perform a comparative analysis of triheptanoin versus placebo over one year, we used the placebo arm of a one-year randomized controlled trial (NCT02336633), conducted in parallel with identical methods, in HD patients with similar clinical characteristics (age, disease duration, TMS, CAG repeats).
    Arm type
    		 Placebo

    Investigational medicinal product name
    Safflower oil
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    1g/kg/dqy

    Number of subjects in period 1
    		Active Comparator arm
    Started
    50
    50
    Completed
    50
    50

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Active
    Reporting group description
    		 triheptanoin treated arm

    Reporting group title
    Comparator arm
    Reporting group description
    		 To perform a comparative analysis of triheptanoin versus placebo over one year, we used the placebo arm of a one-year randomized controlled trial (NCT02336633), conducted in parallel with identical methods, in HD patients with similar clinical characteristics (age, disease duration, TMS, CAG repeats).

    Reporting group values
    		 Active Comparator arm Total
    Number of subjects
    		 50 50 100
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 46 47 93
        From 65-84 years
    		 4 3 7
    Gender categorical
    Units: Subjects
        Female
    		 32 30 62
        Male
    		 18 20 38
    Subject analysis sets

    Subject analysis set title
    Comparator arm
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    external placebo control group

    Subject analysis set title
    Active arm
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    To ensure acceptability for patients, we conducted a 6-month randomized controlled bi-centric trial (Paris and Leiden) called TRIHEP3 (NCT02453061), comparing triheptanoin 1g/kg/day vs placebo in 100 patients (ratio 1/1) at an early stage of HD, followed by a 6-month open label phase. After one year, patients could opt for a one-year extension study.

    Subject analysis sets values
    		 Comparator arm Active arm
    Number of subjects
    50
    50
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    46
    47
        From 65-84 years
    4
    3
    Age continuous
    Units:
        
    ±
    ±
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

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    End points reporting groups
    Reporting group title
    Active
    Reporting group description
    		 triheptanoin treated arm

    Reporting group title
    Comparator arm
    Reporting group description
    		 To perform a comparative analysis of triheptanoin versus placebo over one year, we used the placebo arm of a one-year randomized controlled trial (NCT02336633), conducted in parallel with identical methods, in HD patients with similar clinical characteristics (age, disease duration, TMS, CAG repeats).

    Subject analysis set title
    Comparator arm
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    external placebo control group

    Subject analysis set title
    Active arm
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    To ensure acceptability for patients, we conducted a 6-month randomized controlled bi-centric trial (Paris and Leiden) called TRIHEP3 (NCT02453061), comparing triheptanoin 1g/kg/day vs placebo in 100 patients (ratio 1/1) at an early stage of HD, followed by a 6-month open label phase. After one year, patients could opt for a one-year extension study.

    Primary: rate of caudate atrophy at 6 months

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    End point title
    		 rate of caudate atrophy at 6 months
    End point description
    The primary outcome measure was the rate of caudate atrophy at 6 months using cBSI (caudate boundary shift integral).
    End point type
    Primary
    End point timeframe
    6 months
    End point values
    		 Active Comparator arm
    Number of subjects analysed
    50
    50
    Units: Cubic centimetre
        number (not applicable)
    50
    50
    Statistical analysis title
    		 method
    Statistical analysis description
    To ensure acceptability for patients, we conducted a 6-month randomized controlled bi-centric trial followed by a 6-month open label phase. After one year, patients could opt for a one-year extension study. To perform a comparative analysis of triheptanoin versus placebo over one year, we used the placebo arm of a one-year randomized controlled trial (NCT02336633), conducted in parallel with identical methods, in HD patients with similar clinical characteristics (age, disease duration, CAG-rep)
    Comparison groups
    Comparator arm v Active
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 ≤ 0.05
    Method
    		 ANCOVA
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    48 hours
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    1
    Reporting groups
    Reporting group title
    Active
    Reporting group description
    		 triheptanoin treated arm

    Reporting group title
    Comparator arm
    Reporting group description
    		 To perform a comparative analysis of triheptanoin versus placebo over one year, we used the placebo arm of a one-year randomized controlled trial (NCT02336633), conducted in parallel with identical methods, in HD patients with similar clinical characteristics (age, disease duration, TMS, CAG repeats).

    Serious adverse events
    		 Active Comparator arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 50 (0.00%)
    0 / 50 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Active Comparator arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 50 (4.00%)
    2 / 50 (4.00%)
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 50 (4.00%)
    2 / 50 (4.00%)
         occurrences all number
    2
    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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