Open Label Study Looking at Canakinumab (ACZ885) in TRAPS

• Research type

  Research Study

• Full title

  An open-label, multicenter, efficacy and safety study of 4-month canakinumab treatment with 6-month follow-up in patients with active recurrent or chronic TNF-receptor associated periodic syndrome (TRAPS).

• IRAS ID

  54704

• Contact name

  Helen Lachmann

• Sponsor organisation

  Novartis Pharma Services AG

• Eudract number

  2010-020061-24

• ISRCTN Number

  N/A

• Research summary

  TNF - receptor associated periodic syndrome (TRAPS) is a rare disease from the periodic fever syndromes (PFS) group. Another PFS is cryopyrin associated periodic syndrome (CAPS). People with these diseases have attacks of recurrent high fevers and inflammation in the body, including the skin, gut, muscles and joints causing also skin rashes, fatigue and muscle and joint pains. TRAPS is caused by a genetic mutation which leads to certain immune cells becoming overactive and producing too many chemical messengers, such as Interleukin-1? These cause the disease symptoms, the most serious being amyloidosis. In amyloidosis, a protein called amyloid becomes deposited in body tissues, particularly the kidneys, preventing them from working properly. Approximately 14-25% of TRAPS patients develop amyloidosis, leading to kidney failure. Canakinumab works by binding to Interleukin-1?and preventing it from increasing inflammation.The purpose of this study is to assess if canakinumab is effective in the treatment of TRAPS and induces a response within 15 days of treatment. It will also provide the necessary data to help determine the most effective dosing schedule of canakinumab. The study population will be approximately 20 male and female patients of at least 4 years of age with a confirmed diagnosis of TRAPS. All participants will receive monthly injections (for 4 months) of active canakinumab medication. There will be no placebo/dummy arm in this trial. The dose of canakinumab will be the same as that which is currently approved for use in patients CAPS.Patients will attend 15 study visits (including screening) over a period of approximately 11 months. Patients will undergo the following safety and efficacy tests (every visit): Collection of blood/urine samples, Physical examination, TRAPS assessment. Patients will complete a diary (throughout) and questionnaire (visits 3, 6, 10 & 15). Novartis Pharma AG is sponsoring the study.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  10/H0504/70

• Date of REC Opinion

  15 Nov 2010

• REC opinion

  Further Information Favourable Opinion