MYPAN

• Research type

  Research Study

• Full title

  An Open Label Randomised Controlled Trial of Mycophenolate Mofetil Versus Cyclophosphamide for the Induction of Remission of Childhood Polyarteritis Nodosa

• IRAS ID

  143363

• Contact name

  Paul Brogan

• Contact email

  p.brogan@ucl.ac.uk

• Sponsor organisation

  University College London

• Eudract number

  2013-004668-71

• Research summary

  Polyarteritis Nodosa (PAN) is a type of vasculitis that can be life threatening if left untreated. Currently, the standard treatment for PAN is oral steroids, taken in combination with a drug called cyclophosphamide. These drugs are taken until the disease goes into remission, and then another drug called azathioprine is prescribed, along with the steroids to replace the cyclophosphamide and in order to sustain the remission.
  
  Although this treatment with cyclophosphamide (CYC) is effective, there are a number of undesirable side effects, both in the short and the long term. These include nausea, vomiting, sepsis and alopecia. Longer term side effects include infertility and a possible increased risk of developing some cancers.

  A drug called mycophenolate mofetil (MMF) is potentially a safer alternative for treatment of PAN, and has fewer side effects. This study will assess the effectiveness and safety of MMF in childhood PAN compared to CYC.
  The study aims to recruit 40 participants between the ages of 4 and 18 years from hospitals in the UK, Europe and Turkey, who have been newly diagnosed with PAN. Each participant will be in the study for 18 months.

  Participants will be randomly split into two groups;
  1. CYC and steroids
  2. MMF and steroids.

  When remission has been achieved (after 3-6 months treatment) both groups will receive azathioprine and steroids.

  Information about the study participants will be collected at scheduled hospital visits. Participants will not be required to come to hospital any more frequently than they usually would for PAN treatment.

  Participants will be asked to complete medication diaries at home, and at selected hospital visits they (or their parents or carers) will be asked to fill in questionnaires designed to assess their quality of life.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  14/LO/1565

• Date of REC Opinion

  25 Sep 2014

• REC opinion

  Favourable Opinion