Rilonacept for Treatment of Familial Mediterranean Fever (FMF)
Status:
Completed
Trial end date:
2011-09-01
Target enrollment:
Participant gender:
Summary
Familial Mediterranean fever (FMF) is a genetic disease resulting in recurrent attacks of
fever, abdominal pain, chest pain, arthritis and rash. There are 5-15% of patients who
continue to have FMF attacks despite treatment with colchicine or who cannot tolerate
colchicine. Currently there are no alternatives to colchicine. Pyrin, the protein that has a
defect in FMF has an important role in the regulation of a molecule called interleukin (IL)-1
beta production and activity. This molecule is very important in the process of inflammation
in FMF.
Therefore we propose to use IL-1 Trap (Rilonacept), a medication that binds and neutralizes
IL-1.
We will enroll in this study 17 subjects from the age of 4 years, including adults with
active FMF despite colchicine therapy. Subjects will receive in random order two 3-month
courses of Rilonacept at 2.2 mg/kg (maximum 160 mg) by weekly subcutaneous injection and two
3-month courses of placebo injection. If patients have at least two FMF attacks during a
treatment course they will be able to get if they choose the other treatment until the end of
that treatment course. Our hypothesis is that Rilonacept will decrease the number of acute
FMF attacks and will be safe to use. This study may confirm the importance of IL-1 in the
cause of FMF.
Funding source - FDA Office of Orphan Products Development