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    Summary
    EudraCT Number:2014-005112-42
    Sponsor's Protocol Code Number:C14-62
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-22
    Trial results View 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 ConcernedFrance - ANSM
    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
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    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 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
    E.2.2Secondary objectives of the trial
    - to assess the clinical benefit of triheptanoin on motor function in HD patients using scores on the United Huntington's Disease Rating Scale
    - to assess the clinical benefit of triheptanoin on cognitive function ansd psychiatric symptoms in HD patients using scores on the symbol digit test, PBA-S and the HVLT-R
    - to assess the effect on quality of life (SF-36) of HD patients of therapeutic use of triheptanoin
    -to confirm long term clinical and biological tolerance of triheptanoin in HD patients
    - to evaluate long-term compliance with dietary modifications in HD patients
    - o look for correlations between neuroimaging volumetric parameters; brain energy profiles and clinical scores, beofre avec after treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - positive genetic test with CAG repeat lenght ≥ 39 in HTT gene
    - at lmeast 18 years of age
    - signature of informed consent
    - covered by social security
    - UHDRS score between 5 and 40
    - Ability to undergo MRI scanning
    E.4Principal exclusion criteria
    - Hypersensitivity to triheptanoin or to one of its excipients
    - additional psychiatric or neurological conditions
    - severe head injury
    - Participation in another therapeutical trial ( 3 months exclusion period)
    - Pregnancy or breastfeeding
    - Inability to understand information about the protocol
    - persons deprived of their liberty by judicial or unable to consent
    - adult subject under legal protection or unable to consent
    - Patients under tetrabenazine and neuroleptics other than atypical neuroleptics at a small dose
    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.5.2Secondary end point(s)
    a) increase in the index of brain energy restoration using 31P-MRS
    b) decrease in the rate of caudate atrophy after 1 year of treatment with triheptanoin in early affected HD patient.
    c) the clinical benefit of triheptanoin will be evaluated by a decrease in the progression of the UHDRS over 6 months ans 1 year of treatment.
    d) it will be also evaluated using SMDT
    e) The benefit of triheptnaoin use on patient quality of life will be evaluated by using the SFR-36
    f) long term compliance will be performed by regular bioclinical testing from blood abd urine samples
    g) Long term tolerance will be confirmed by clinical exam and by patient phone call
    h) changes in brain energy will be correlated with volumetric measures ans clinical rating scale scores.
    E.5.2.1Timepoint(s) of evaluation of this end point
    a) 6 months and 1 year of treatment
    b) 1 year
    c) 6 months and 1 year
    d) 1 year
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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 Decision2015-06-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-12-03
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