Multiple dose study for a new medication to potentially treat liver diseases

  • Research type

    Research Study

  • Full title

    Investigation of safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of NNC0581-0001 in participants with hepatic steatosis and suspected steatohepatitis

  • IRAS ID

    1010323

  • Contact name

    Jake Bomford

  • Contact email

    Jake.Bomford@parexel.com

  • Sponsor organisation

    Novo Nordisk A/S

  • Research summary

    The trial is a single-centre, randomized or open label trial dependent on cohort. A total of 48 men and women (of non childbearing potential) with increased liver fat and suspected inflammation will take part in this study in the UK. A total of 38 will be randomized into the double blind placebo controlled cohort.
    A total of 10 will take part in the open-label part of the study.

    This study is being performed to determine the safety, tolerability and how the study medicine is absorbed and eliminated from the human body. The study will also explore how the human body is affected by the study medicine.

    NNC0581-0001 is an siRNA (a complex organic substance present in living cells) which is being developed as a treatment for Metabolic dysfunction-associated steatohepatitis (MASH). At the time of starting this study, single doses of up to 1000 mg have previously been tested in 24 healthy volunteers and in 12 patients with increased liver fat. No serious or severe side effects related to the study medicine have been reported.
    The randomized cohort will comprise of:
    - Three treatment periods where participants are hospitalised for three days for baseline assessments, administration of study medicine/placebo and a close safety monitoring.
    - There will be 11 visits at the clinical unit in total
    - 3 visits to NHS hospitals for MRI scans in total
    - 11 Follow up phone calls

    The Open label cohort will comprise of the same as above except for:
    - 2 additional visits to NHS hospitals for liver biopsy (once at during screening period, once 16 weeks after first treatment)

  • REC name

    London - Surrey Borders Research Ethics Committee

  • REC reference

    24/LO/0561

  • Date of REC Opinion

    24 Sep 2024

  • REC opinion

    Further Information Favourable Opinion