An Open-Label Study of Canikinumab in Paediatric Patients with CAPS

• Research type

  Research Study

• Full title

  A one-year open-label, multi-centre trial to assess efficacy, safety and tolerability of canakinumab (ACZ885) and the efficacy and safety of childhood vaccinations in patients aged 4 years or younger with Cryopyrin Associated Periodic Syndromes (CAPS)

• IRAS ID

  57582

• Contact name

  PA Brogan

• Eudract number

  2009-016859-22

• ISRCTN Number

  N/a

• Research summary

  This is a Phase III study to provide efficacy, safety and tolerability data for canakinumab and concomitant inactivated vaccines in infants aged 4 years and younger with CAPS. Two centres in the UK will be involved. Each will be expected to recruit 2 patients over the next three years. Based on the available literature on CAPS, clinical symptoms typically occur from the early neonatal period, especially in the most severe and life-threatening form of CAPS: Neonatal-Onset Multisystem Inflammatory Disorder (NOMID) with the presence of a typical rash present in the neonatal period. Both Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) might be diagnosed in early infancy as well. With the current increasing knowledge, better diagnostic tools and increased awareness among physicians, it is likely that CAPS will be diagnosed earlier and more frequently in children. Canakinumab is indicated for the treatment of CAPS in patients =4 years of age rendering a lack of an approved treatment in younger patients. Treatment with canakinumab (ACZ885) might have a disease modifying effect on related symptoms and severe complications such as amyloidosis and aseptic meningitis, while early treatment with canakinumab in the paediatric CAPS population might be beneficial in reducing acute clinical symptoms and pain. The current study fulfills a commitment towards the EMEA (PDCO and CHMP) to study very young CAPS patients in order to increase the experience in children with CAPS aged = 4 years of age treated with canakinumab as well as to study the efficacy and safety of childhood vaccinations in this population. In order to study this aspect, subjects will be assessed for the development of protective antibody levels following immunization with inactivated (killed) vaccines according to their respective local vaccination guidelines.

• REC name

  London - Central Research Ethics Committee

• REC reference

  10/H0718/64

• Date of REC Opinion

  15 Oct 2010

• REC opinion

  Further Information Favourable Opinion