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Clinical trials for 2016-000360-42

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
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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   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).

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    1 result(s) found for: 2016-000360-42. Displaying page 1 of 1.
    EudraCT Number: 2016-000360-42 Sponsor Protocol Number: UX001-CL302 Start Date*: 2016-08-02
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myo...
    Medical condition: GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000167584 10075048 Hereditary inclusion body myopathy LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10077945 GNE myopathy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) BG (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    The status and protocol content of GB trials is no longer updated since 1 January 2021.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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