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Clinical trials for Carnitine deficiency

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   44364   clinical trials with a EudraCT protocol, of which   7388   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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    5 result(s) found for: Carnitine deficiency. Displaying page 1 of 1.
    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: 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: 2014-001637-88 Sponsor Protocol Number: 2014-650 Start Date*: 2014-08-12
    Sponsor Name:John Vissing
    Full Title: A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disorder type V)
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2012-002933-12 Sponsor Protocol Number: 11/0090 Start Date*: 2014-04-07
    Sponsor Name:University College London,
    Full Title: A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdom...
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    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
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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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