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Clinical trials for Epi-743

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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    3 result(s) found for: Epi-743. Displaying page 1 of 1.
    EudraCT Number: 2012-001294-84 Sponsor Protocol Number: OPBGC&RS2011-004_CA2012 Start Date*: 2012-09-03
    Sponsor Name:EDISON PHARMACEUTICALS INC.
    Full Title: Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease
    Medical condition: Leigh Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2012-005021-76 Sponsor Protocol Number: OPBGC&RS_12_003 Start Date*: 2013-01-31
    Sponsor Name:EDISON PHARMACEUTICALS INC.
    Full Title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome
    Medical condition: Rett Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10021605 Inborn errors of metabolism HLGT
    Population Age: Children, Adolescents, Under 18 Gender: Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-003265-38 Sponsor Protocol Number: PTC743-NEU-005-FA Start Date*: 2022-10-22
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age
    Medical condition: Friedreich Ataxia
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
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    Query did not match any studies.
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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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