4345: N8 Given as ITI Treatment

  • Research type

    Research Study

  • Full title

    Evaluation of safety following Immune Tolerance Induction treatment with turoctocog alfa in patients with haemophilia A following inhibitor development in NN7170-4213 trial.

  • IRAS ID

    220052

  • Contact name

    Pratima Chowdary

  • Contact email

    p.chowdary@nhs.net

  • Sponsor organisation

    Novo Nordisk A/S

  • Eudract number

    2016-003821-40

  • Clinicaltrials.gov Identifier

    U1111-1187-7323, UTN (Universal Trial Number)

  • Duration of Study in the UK

    4 years, 5 months, 0 days

  • Research summary

    Patients with severe haemophilia A have a deficiency in a blood clotting factor called factor VIII (FVIII) which means they are at risk of suffering bleeds. They are usually treated with FVIII by intravenous injection (injection into a vein) to prevent &/or treat bleeds.
    Treatment with FVIII can sometimes result in development of antibodies which neutralise (block) the activity of the FVIII. These antibodies are called inhibitors. When this happens it is common to try immune tolerance induction (ITI). ITI is an established treatment for getting rid of inhibitors. It involves giving regular high doses of factor FVIII. Repeated exposure to FVIII eventually causes the body to stop making the antibody (inhibitor) which disappears and FVIII is once again effective in the treatment and prevention of bleeds.
    Patients included in this trial will all have participated in a previous clinical trial NN7170-4213 which investigates the possibility of giving a FVIII medication (called N8-GP) by subcutaneous injection (injection under the skin). There is a risk that giving N8-GP by subcutaneous injection could result in the development of inhibitors. The development of inhibitors is not expected to happen in the NN7170-4213 trial but it is a possibility.
    The current trial is necessary to ensure that any patient who develops an inhibitor whilst participating in NN7170-4213 will have access to ITI and this will be at no cost to themselves or to the NHS.
    Patients will be in the current trial for a maximum of 31 months, with a maximum of 24 months on treatment. After 12 months a stop/go visit will take place to assess the ITI treatment response.

  • REC name

    London - Hampstead Research Ethics Committee

  • REC reference

    17/LO/0046

  • Date of REC Opinion

    15 Feb 2017

  • REC opinion

    Further Information Favourable Opinion