This site uses cookies. By continuing to browse the site you are agreeing to our use of cookies.

Inclisiran in adolescents with heterozygous familial hypercholesterolemia

  • Research type

    Research Study

  • Full title

    Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-16)

  • IRAS ID

    1003480

  • Contact name

    Ian McDowell

  • Contact email

    Mcdowell@cardiff.ac.uk

  • Sponsor organisation

    Novartis Pharma AG

  • Eudract number

    2020-002757-18

  • Research summary

    CKJX839C12301 (ORION-16) is a pivotal phase III study designed to evaluate safety,
    tolerability, and efficacy of inclisiran in 6 adolescents (aged 12 to <18 years) with
    heterozygous familial hypercholesterolemia (HeFH) and elevated LDL-cholesterol (LDLC)
    across 4 research sites. The use of inclisiran (as an addition to stable, optimal
    background lipid-lowering therapy) for the treatment of HeFH in adolescent patients
    who require additional lipid-lowering will be investigated in order to obtain needed
    pediatric information on inclisiran. The follow-up period (Part 2/Year 2) serves to collect
    longer-term data on inclisiran and also allows access of study participants to a
    potentially effective treatment.

  • REC name

    North West - Haydock Research Ethics Committee

  • REC reference

    20/NW/0459

  • Date of REC Opinion

    22 Jan 2021

  • REC opinion

    Further Information Favourable Opinion