Graves-PCD

  • Research type

    Research Study

  • Full title

    Randomised controlled trial of plasma cell depletion for severe Graves’ disease

  • IRAS ID

    1003652

  • Contact name

    Simon Pearce

  • Contact email

    simon.pearce@ncl.ac.uk

  • Sponsor organisation

    The Newcastle upon Tyne Hospitals NHS Foundation Trust

  • Eudract number

    2020-005635-78

  • Research summary

    Graves' disease gives symptoms such as heart palpitations, heat intolerance, unintended weight loss, enlarged thyroid, red and swollen eyelids, protuberant eyeballs and double vision. Patients with severe Graves' disease frequently have disabling eye disease and occasionally loss of vision. Treatment with antithyroid drugs lead to remission in only around 50% of people, which falls to around 20% for people with severe Graves' disease, and does not improve the eye problems. Patients with severe Graves' disease often have their thyroid gland removed surgically, followed by several eye operations to correct the visual function and appearance of the eyes. These are expensive operations with low overall patient satisfaction. Better treatments are needed.

    The antibodies (known as TRAb) that drive the thyroid and eye symptoms are produced by a type of white blood cell called a plasma cell, whose sole purpose is to make large quantities of antibodies. This Medical Research Council funded trial will find out whether a new treatment called daratumumab that has been developed to treat plasma cell cancer, could also be used to target the benign plasma cells in patients with severe Graves' disease. By removing plasma cells in Graves' disease, we would expect a reduction in the disease-causing TRAb antibody concentration and a rapid improvement in the thyroid overactivity and eye symptoms.

    This study will perform a 2-stage randomised trial of daratumumab in 30 patients with severe Graves' disease. Because daratumumab has not been used in Graves' disease before, the first part of our study will administer 4 different doses or a placebo to small groups of participants to see which of the doses works best. Then we will use one or 2 of the best daratumumab doses or a placebo to treat a larger groups of patients. The daratumumab is given twice by intravenous infusion to each participant, and participants will be followed up in a further 4 clinic appointments across 6 months.

  • REC name

    London - Hampstead Research Ethics Committee

  • REC reference

    21/LO/0449

  • Date of REC Opinion

    21 Jul 2021

  • REC opinion

    Further Information Favourable Opinion