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Clinical trials for Aicardi Goutieres 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: Aicardi Goutieres Syndrome. Displaying page 1 of 1.
    EudraCT Number: 2025-000001-16 Sponsor Protocol Number: I4V-JE-JAJE Start Date*: 2025-02-19
    Sponsor Name:Eli Lilly and Company
    Full Title: A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients with NNS/CANDLE, SAVI, and AGS
    Medical condition: Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated Temperature Syndrome, STING-Associated Vasculopathy with Onset in Infancy, Aicardi Goutieres Sy...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10073960 CANDLE syndrome PT
    21.1 10047065 - Vascular disorders 10047115 Vasculitis PT
    27.1 10010331 - Congenital, familial and genetic disorders 10054935 Aicardi's syndrome PT
    Population Age: Children, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2022-000064-21 Sponsor Protocol Number: TPN-101-AGS-201 Start Date*: 2022-09-20
    Sponsor Name:Transposon Therapeutics Inc
    Full Title: A Phase 2a Study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)
    Medical condition: Aicardi-Goutières Syndrome (AGS)
    Disease: Version SOC Term Classification Code Term Level
    22.1 10010331 - Congenital, familial and genetic disorders 10083189 Aicardi-Goutieres syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-003424-31 Sponsor Protocol Number: I4V-MC-JAGA Start Date*: Information not available in EudraCT
    Sponsor Name:Eli Lilly and Company
    Full Title: Treatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis
    Medical condition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis, Aicardi-Goutieres Syndrome
    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: (No results available)
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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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