A4250 in paediatric cholestasis

  • Research type

    Research Study

  • Full title

    An Exploratory Phase II Study to demonstrate the Safety and Efficacy of A4250 in Children with Cholestatic Pruritus

  • IRAS ID

    212132

  • Contact name

    Richard Thompson

  • Contact email

    richardthompson@nhs.net

  • Sponsor organisation

    Albireo AB

  • Eudract number

    2015-001157-32

  • Clinicaltrials.gov Identifier

    NCT02630875

  • Duration of Study in the UK

    0 years, 5 months, 31 days

  • Research summary

    Chronic liver disease with impaired bile flow (cholestasis) makes bile acids increase in the blood. Assuming those endogenous bile acids and their transformation products from the intestine triggers the itch the study aims to investigate whether A4250 is effective against this itching and against the liver disease as such. There are currently no approved drugs available for treatment of the liver disease or itch.
    A4250 is a drug that is being developed to improve the itching in people with chronic liver disease and to improve the liver function. A4250 reduces the absorption of bile acids from the intestine and thus lower their blood levels. The drug is only absorbed in minimal amounts from the gut and has so far been tested on healthy volunteers and in an ongoing study in adult patients.
    This is a Phase II single and multiple dosing open-label study of orally administered A4250 to evaluate the safety and efficacy of A4250 when given for four weeks in patients diagnosed with cholestatic pruritus. This study is planned to include 24 patients in the age between 12 months and 18 years and will be performed at clinics located in Europe.
    In UK 4 patients are planned to be included. No randomization or stratification is planned.

  • REC name

    London - Dulwich Research Ethics Committee

  • REC reference

    16/LO/1585

  • Date of REC Opinion

    25 Oct 2016

  • REC opinion

    Further Information Favourable Opinion