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Clinical trials for Monogenic disorder

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   44336   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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    4 result(s) found for: Monogenic disorder. Displaying page 1 of 1.
    EudraCT Number: 2017-004420-30 Sponsor Protocol Number: CL3-95008-002 Start Date*: 2018-10-04
    Sponsor Name:Institut de Recherches Internationales Servier
    Full Title: Efficacy and safety of bumetanide oral liquid formulation in children aged from 2 to less than 7 years old with Autism Spectrum Disorder. A 6-month randomised, double-blind, placebo controlled mult...
    Medical condition: Autism Spectrum Disorder (ASD)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10037175 - Psychiatric disorders 10063844 Autism spectrum disorder PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) ES (Prematurely Ended) NL (Not Authorised) HU (Completed) PT (Completed) PL (Prematurely Ended) IE (Prematurely Ended) CZ (Completed) SK (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-004419-38 Sponsor Protocol Number: CL3-95008-001 Start Date*: 2018-10-04
    Sponsor Name:Institut de Recherches Internationales Servier
    Full Title: Efficacy and safety of bumetanide oral liquid formulation in children and adolescents aged from 7 to less than 18 years old with Autism Spectrum Disorder. A 6-month randomised, double-blind, place...
    Medical condition: Autism Spectrum Disorder (ASD)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10037175 - Psychiatric disorders 10063844 Autism spectrum disorder PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Prematurely Ended) DE (Completed) ES (Prematurely Ended) NL (Completed) HU (Completed) PT (Completed) PL (Prematurely Ended) IT (Completed) IE (Prematurely Ended) CZ (Ongoing) SK (Completed)
    Trial results: View results
    EudraCT Number: 2018-002947-27 Sponsor Protocol Number: SIMCODE Start Date*: 2020-01-22
    Sponsor Name:Charité – Universitätsmedizin Berlin
    Full Title: Simvastatin add-on to Escitalopram in patients with comorbid obesity and major depression: A multicenter, randomized, double-blind, placebo-controlled trial
    Medical condition: Patients with comorbid obesity (body mass index ≥ 30) and major depression
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-003596-17 Sponsor Protocol Number: CMAS825D12201 Start Date*: 2021-03-09
    Sponsor Name:Novartis Pharma AG
    Full Title: A three-period multicenter study, with a randomized withdrawal, double-blinded, placebo-controlled design in Period 2 to evaluate the clinical efficacy, safety and tolerability of MAS825 in NLRC4-G...
    Medical condition: NLRC4-GOF, AIFEC (autoinflammation with infantile enterocolitis)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10084306 Autoinflammation with infantile enterocolitis PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    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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