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Clinical trials for Wolfram syndrome

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   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).

    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: Wolfram syndrome. Displaying page 1 of 1.
    EudraCT Number: 2022-003853-70 Sponsor Protocol Number: PNRR-MR1-2022-12375914 Start Date*: 2023-05-02
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose­ dependent insulinotropic polypeptide and glucagon-like peptide-I receptor agonist) monotherapy in patients wi...
    Medical condition: Wolfram syndrome type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-004594-43 Sponsor Protocol Number: ISTEM02 Start Date*: 2021-05-27
    Sponsor Name:CECS/I-Stem
    Full Title: AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation
    Medical condition: Wolfram syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-001215-37 Sponsor Protocol Number: RG_16_211 Start Date*: 2018-03-19
    Sponsor Name:University of Birmingham
    Full Title: A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome
    Medical condition: Wolfram syndrome
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PL (Completed) FR (Completed)
    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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