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Clinical trials for Triheptanoin

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    12 result(s) found for: Triheptanoin. Displaying page 1 of 1.
    EudraCT Number: 2016-001019-19 Sponsor Protocol Number: UX007 Start Date*: 2016-06-27
    Sponsor Name:LIEGE University Hospital
    Full Title: A pilot trial of triheptanoin for the preventive treatment of migraine
    Medical condition: Migraine
    Disease:
    Population Age: Adults Gender: Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2014-005112-42 Sponsor Protocol Number: C14-62 Start Date*: 2015-06-22
    Sponsor Name:INSERM
    Full Title: A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3)
    Medical condition: Huntington's disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2014-003920-32 Sponsor Protocol Number: C13-37 Start Date*: 2013-11-26
    Sponsor Name:INSERM
    Full Title: Study phase II open label study using triheptanoin in patients with a deficiency of glmucose transporter type 1 (GLUT-1)
    Medical condition: N/A
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10039911 Seizures (incl subtypes) HLGT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2022-001539-10 Sponsor Protocol Number: UX007-CL302 Start Date*: 2022-12-27
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared with Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patient...
    Medical condition: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10077951 Fatty acid oxidation disorder PT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Trial now transitioned) PL (Trial now transitioned) CZ (Trial now transitioned) DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-004228-23 Sponsor Protocol Number: C14-53 Start Date*: 2015-01-08
    Sponsor Name:Inserm
    Full Title: N/A
    Medical condition: see below
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-000322-19 Sponsor Protocol Number: UX007-CL202 Start Date*: 2016-06-06
    Sponsor Name:Ultragenyx Pharmaceutial Inc.
    Full Title: An Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
    Medical condition: Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2014-003644-12 Sponsor Protocol Number: 2014-003644-12 Start Date*: 2015-02-20
    Sponsor Name:Neuromuscular Research Unit, Rigshospitalet
    Full Title: The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V)
    Medical condition: McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10026970 McArdles disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10026969 McArdle's disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10018462 Glycogen storage disease type V PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-004153-17 Sponsor Protocol Number: 20171012 Start Date*: 2018-05-03
    Sponsor Name:Copenhagen Neuromuscular Center
    Full Title: 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 an...
    Medical condition: 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. ...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053241 Glycogen storage disease type VII PT
    20.1 10010331 - Congenital, familial and genetic disorders 10053250 Glycogen storage disease type III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10053255 Tarui disease LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10016983 Forbes' disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-004830-14 Sponsor Protocol Number: UX007-CL201 Start Date*: 2014-02-26
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-Label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    Medical condition: Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-003771-35 Sponsor Protocol Number: UX007G-CL201 Start Date*: 2014-02-11
    Sponsor Name:Ultragenyx Pharmaceutical Inc
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Adaptive Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
    Medical condition: Glucose Transporter Type 1 deficiency syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029205 - Nervous system disorders 10039911 Seizures (incl subtypes) HLGT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) GB (Completed) FR (Ongoing) HU (Completed) ES (Completed) DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-000389-69 Sponsor Protocol Number: UX007G-CL202 Start Date*: 2017-01-27
    Sponsor Name:Ultragenyx Pharmaceutical Inc
    Full Title: An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
    Medical condition: Glucose Transporter Type 1 deficiency syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10061032 Carbohydrate transport disorder LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) ES (Prematurely Ended) DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-005536-17 Sponsor Protocol Number: UX007G-CL301 Start Date*: 2017-05-17
    Sponsor Name:Ultragenyx Pharmaceutical Inc
    Full Title: A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1...
    Medical condition: Glucose Transporter Type 1 deficiency syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10061032 Carbohydrate transport disorder LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) ES (Prematurely Ended) FR (Completed) GB (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
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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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