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    Summary
    EudraCT Number:2014-005112-42
    Sponsor's Protocol Code Number:C14-62
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-08-07
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2014-005112-42
    A.3Full title of the trial
    A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3)
    A.4.1Sponsor's protocol code numberC14-62
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorINSERM
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportUltragenyx Pharmaceutical
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationINSERM
    B.5.2Functional name of contact pointSonia GUEGUEN
    B.5.3 Address:
    B.5.3.1Street Address8 rue de la Croix Jarry
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75013
    B.5.3.4CountryFrance
    B.5.4Telephone number33144236041
    B.5.5Fax number33144236710
    B.5.6E-mailrqrc.siege@inserm.fr
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTriheptanoin
    D.3.2Product code UX007
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Huntington's disease
    E.1.1.1Medical condition in easily understood language
    huntington's disease
    E.1.1.2Therapeutic area Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of TRIHEP 3 is to evaluate the efficacy of triheptanoin in
    (i) increasing the short term energy response in the metabolic profile of the brain of early affected HD patients, as captured by 31-Phosphorus Magnetic Resonance Spectroscopy, and
    (ii) slowing atrophy in the caudate of early affected HD patients as measured with volumetric magnetic resonance imaging
    E.2.2Secondary objectives of the trial
    To assess the clinical benefit of triheptanoin on motor function in HD patients using scores on the UHDRS.
    To assess the clinical benefit of triheptanoin on cognitive function and psychiatric symptoms in HD patients using scores on the neuropsychological battery and the PBA-S.
    To investigate complex phenomena that are difficult to measure quantitatively, especially components of patients’ daily life that may have been affected by treatment, short videos of patients (3 min) will be recorded at the end of V3; a team of experts blind to treatment group will then be asked to classify each video according to whether he/she thinks that the patient had received the study drug or not, using a 4-point Likert scale,SF-36 will also be used.
    To confirm long-term clinical and biological tolerance of triheptanoin in HD patients.
    To look for correlations between neuroimaging volumetric parameters, brain energy profiles and clinical scores, before and after treatment.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Positive genetic test with CAG repeat length ≥39 in HTT gene
    • At least 18 years of age
    • Signature of informed consent
    • Covered by social security
    • UHDRS score between 5 and 40
    • Ability to undergo MRI scanning
    • BMI between 18 and 30
    E.4Principal exclusion criteria
    • Treatment with sodium valproate
    • Treatment with tetrabenazine
    • Treatment with inhibitors of pancreatic lipases (e.g. orlistat)
    • Hypersensitivity to triheptanoin or to one of its excipients
    • Additional major comorbidities
    • History of severe head injury
    • Participation in another therapeutic trial (3 month exclusion period)
    • For women of childbearing age, the absence of two forms of effective contraception (with the exception of those who are abstinent)
    • For men, the absence of an effective form of contraception (e.g. a condom) throughout the study period
    • Pregnancy or breastfeeding
    • Inability to understand information about the protocol
    • Persons deprived of their liberty by judicial or administrative decision
    • Adult subject under legal protection or unable to consent
    E.5 End points
    E.5.1Primary end point(s)
    - an increase in the index of brain energy restoration as defined by the difference between Pi/PCr ration during visual stimulation and the mean of Pi/PCr ratio during rest and recovery using 31P-MRS after 3 months
    - a decrease in the rate of caudate atrophy, using volumetric MRI, after six months of treatment with triheptanoin in early HD patients
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months
    6 months
    E.5.2Secondary end point(s)
    a.Sustained restoration of brain energy metabolism using 31P-MRS
    b.Decrease in the rate of caudate atrophy, using volumetric MRI
    c.Improved diffusivity and/or fiber integrity, using DWI
    d.The benefit of triheptanoin on motor function will be evaluated by a decrease in the progression of the UHDRS, the HD clinical reference scale with a motor score of up to 124 and of the TFC
    e.The benefit of triheptanoin on cognitive function will be evaluated using a neuropsychological battery including the SDMT , a test of visuomotor coordination, the Stroop test, a test evaluating concentration and capacity for inhibition, the Digit-Span, a test evaluating attention and working memory, and the Trail Making Test to evaluate mental flexibility
    f.The effect of triheptanoin on psychiatric symptoms will be evaluated every with the PBA-S, an evaluation of problem behaviors associated with HD
    g. The global impact of triheptanoin on patients' daily life will be evaluated at the end of the blinded treatment period using qualitative research methods. To statistically test whether experts classify treated and not treated patients better than could be expected by chance, a permutation test based on a modified version of Fisher’s Lady tasting tea procedure will be used (Fischer 1935). A standardized quality of life questionnaire, the SF-36, will also be used
    h. For patients who wish to participate to the extension phase of the study, brain energy profile (31P-MRS), caudate atrophy (volumetry) and diffusivity/fiber integrity (DWI) will be evaluated, as well as motor (UHDRS, TFC), cognitive (SMDT, Stroop test, Digit-Span, Trail Making Test) psychiatric (PBA-S) and quality of life (SF-36) parameters.
    i. Safety of triheptanoin will be evaluated based on review of adverse events and changes in clinical labs and physical examination/vital signs.
    j. Long-term tolerance will be confirmed by clinical exam at study visits and by patient report during phone calls and home visits.
    k. Changes in brain energy profiles will be correlated with volumetric measures and clinical rating scale scores.

    E.5.2.1Timepoint(s) of evaluation of this end point
    a) 6 months and 1 year 2 years of treatment
    b) 1 year and 2 years of treatment
    c) 6 months and 1 year and 2 years of treatment
    d) over 6 months, 1 year and 2 years of treatment
    e) over 2 years
    f) 0- 3- 6-9-12-24 months
    g) SF36: 0-6-12-24 months
    h) over 2 years
    i) over 2 years
    j) over 2 years
    k) over 2 years


    i)
    j)
    k)

    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA1
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Netherlands
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    n/a
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-08-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-12-03
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