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    Summary
    EudraCT Number:2017-004153-17
    Sponsor's Protocol Code Number:20171012
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-03-26
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2017-004153-17
    A.3Full title of the trial
    Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study.
    Effekten af Triheptanoin på fedtforbrænding og arbejdstolerance i patienter med debrancher deficiency, glycogenin-1 deficiency og phosphofructokinase deficiency i hvile og under arbejde. Et randomiseret, dobbelt-blindet, placebo-kontrolleret, cross-over studie.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses
    Effekten af Triheptanoin på arbejde i voksne og unge med glykogenoser.
    A.3.2Name or abbreviated title of the trial where available
    Triheptanoin in Glycogenoses
    Triheptanoin hos glykogenoser
    A.4.1Sponsor's protocol code number20171012
    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 SponsorCopenhagen Neuromuscular Center
    B.1.3.4CountryDenmark
    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 supportNovo Nordisk Foundation
    B.4.2CountryDenmark
    B.4.1Name of organisation providing supportA.P. Møller Foundation
    B.4.2CountryDenmark
    B.4.1Name of organisation providing supportUltragenyx Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRigshospitalet
    B.5.2Functional name of contact pointCopenhagen Neuromuscular Center
    B.5.3 Address:
    B.5.3.1Street AddressBlegdamsvej 9
    B.5.3.2Town/ cityCopenhagen
    B.5.3.3Post code2100
    B.5.3.4CountryDenmark
    B.5.4Telephone number4535456135
    B.5.5Fax number4535453861
    B.5.6E-maildaniel.emil.tadeusz.raaschou-pedersen.01@region.dk
    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 liquid
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.1CAS number 210-647-2
    D.3.9.2Current sponsor codeUX0007
    D.3.9.3Other descriptive nameTRIHEPTANOIN
    D.3.9.4EV Substance CodeSUB129584
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 liquid
    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
    Cori Forbe's Disease
    Also called: glycogen storage disease Type III or debrancher deficiency.
    Tarui's disease
    Also called: glycogen storage disease Type VII or phosphofructokinase deficiency.
    Glycogenin-1 deficiency or glycogen storage disease Type XV.
    E.1.1.1Medical condition in easily understood language
    GSD III and GSD XV are inborn genetic defects in muscle cells causing deficient glycogen metabolism.
    GSD VII is a genetic defect in muscle cells causing deficient glucose metabolism.
    GSD III, GSD VII og GSD XV er medfødte gendefekter i muskler, hvilket medfører enten nedsat glykogenforbrænding eller nedsat glukoseforbrænding i muskelcellerne.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10053241
    E.1.2Term Glycogen storage disease type VII
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10053250
    E.1.2Term Glycogen storage disease type III
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053255
    E.1.2Term Tarui disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10016983
    E.1.2Term Forbes' 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
    To investigate the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise.

    E.2.2Secondary objectives of the trial
    To investigate the effect of Triheptanoin treatment on:
    * Self-rated daily function scores on a modified SF-36 questionnaire
    * Maximal workload capacity (Wmax).
    * Changes in plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines, malate (a TCA intermediate), C5, and hormones.
    * Rate of Perceived Exertion (Borg score) during constant workload cycling.
    * Maximal oxidative capacity.
    * Bouchards energy expenditure questionnaire.
    * Glucose rate of appearance and disappearance.


    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male/Female Age > 15 years
    - Genetically and/or biochemically verified diagnosis of GSD III, GSD VII or GSD XV
    - Capacity to consent
    - For women in fertile age on contraceptive treatment with: Birth control
    pills, coil, ring, transdermal hormone patch injection of gestagen or
    subdermal implant.
    E.4Principal exclusion criteria
    - Significant cardiac or pulmonary disease
    - Pregnancy (confirmed by urine stix or plasma-HCG) or breastfeeding.
    - Inability to perform cycling exercise
    - Any other significant disorder that may confound the interpretation of
    the findings
    E.5 End points
    E.5.1Primary end point(s)
    - Heart rate during constant load cycling exercise (HR_const)
    - Fatty acid oxidation (FAO)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Before and after 14 days of treatment with Triheptanoin and before and
    after 14 days of placebo treatment.
    E.5.2Secondary end point(s)
    • Self-reported health and well-being addressed in an SF-36 Questionnaire.
    • Maximal workload capacity (Wmax).
    • Plasma concentrations of lactate, ammonia, glucose, FFA, acyl-carnitines and malate (a TCA intermediate), creatine kinase, and hormones.
    • Rate of Perceived Exertion during constant workload cycling (RPEconst).
    • Total fatty acid oxidation and total carbohydrate oxidation measured by indirect calorimetry.
    • Glucose rate of appearance and disappearance.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Before and after 14 days of treatment with Triheptanoin and before and after 14 days of placebo treatment.
    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 Yes
    E.6.4Safety No
    E.6.5Efficacy No
    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 No
    E.8.1.6Cross over Yes
    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 No
    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 Yes
    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
    When all data is collected and the last safety follow-up has been done
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days31
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 4
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 14
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 state20
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 20
    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)
    When subjects are done with the study they will return to their usual treatment planned by their own doctor.
    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 Decision2018-05-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-08-24
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