Phase III study of inclisiran sodium in subjects with HeFH

  • Research type

    Research Study

  • Full title

    A placebo-controlled, double-blind, randomized trial to evaluate the effect of 300mg of inclisiran sodium given as subcutaneous injections in subjects with heterozygous familial hypercholesterolemia (HeFH) and elevated low- density lipoprotein cholesterol (LDL-C)

  • IRAS ID

    233068

  • Contact name

    Devaki Nair

  • Contact email

    devaki.nair@nhs.net

  • Sponsor organisation

    The Medicines Company

  • Eudract number

    2017-002472-30

  • Duration of Study in the UK

    2 years, 2 months, 1 days

  • Research summary

    This is a multicentre study of 300mg inclisiran sodium given as subcutaneous injections in participants with Heterozygous Familial Hypercholesterolemia (HeFH) and elevated low-density lipoprotein cholesterol (LDL-C) also called ‘bad’ cholesterol. The study is funded by The Medicines Company.

    The standard current treatment for lowering ‘bad’ cholesterol is taking one of the statin drugs. However, this treatment alone is sometimes not enough in some patients and the ‘bad’ cholesterol remains too high, also some patients are unable to tolerate statins in standard doses. For this reason there is a need to develop a new drug with the potential to help manage this ‘bad’ cholesterol more effectively. The Medicines Company are developing an investigational drug called Inclisiran which has been tested previously in approximately 500 people and the results showed that inclisiran has the potential to reduce the ‘bad’ cholesterol levels in the blood.

    Approximately 400 participants will be recruited globally with around 60 participants recruited in the UK. Each individual participant’s treatment will last for up to 18 months and total duration for each individual participant is expected to be 554 days.

    Eligible participants will be randomly assigned to receive either inclisiran or matching placebo (pill with no medicine). The chances of receiving inclisiran are in a 1:1 ratio.

    Participants will make a total of 10 visits and receive an injection of inclisiran or matching placebo at 4 of the 10 visits.

  • REC name

    London - Harrow Research Ethics Committee

  • REC reference

    17/LO/1943

  • Date of REC Opinion

    4 Jan 2018

  • REC opinion

    Further Information Favourable Opinion