Kineret (Anakinra), in Adult Patients With Colchicine-Resistant Familial Mediterranean Fever
Status:
Completed
Trial end date:
2015-06-01
Target enrollment:
Participant gender:
Summary
FMF is the most common periodic fever with a worldwide patient population estimated as
150,000, mainly located in the Eastern Mediterranean basin. colchicine is the established
therapy of choice ,however, around 20.000 patients worldwide fail to respond or cannot
tolerate therapeutic doses, thereby suffering from recurrent debilitating, severe, painful
attacks of peritonitis, pleuritis and synovitis and are at risk to die from reactive
amyloidosis .Mutation-induced reduction in pyrin/ marenostrin activity is thought to underlie
the disease by leading to NALP3 inflammasome activation ,and thereby to IL-1β related burst
of inflammation.
The IL-1 receptor antagonist Kineret (Anakinra), seems to be the most appropriate response to
the uncontrolled IL-1β elevation. Indeed, an increasing number of reports over the last few
years indicate a good response to Kineret (Anakinra), in colchicine-resistant FMF ,also in
children ,however, no controlled study has thoroughly evaluated the efficacy and safety of
this treatment.
Study outline:
The study aims to run at the FMF centre in Sheba Medical Center, covering more than 10,000
patients. The study will evaluate the effect of recombinant IL-1 receptor antagonist, Kineret
(Anakinra), on the frequency of FMF attacks in patients that, despite maximum tolerable dose
of colchicine, present with more than one attack per month.
The study is designed as a randomised, placebo-controlled, double-blind study. 50 patients
will be randomised to treatment with either Kineret (Anakinra), or placebo treatment for 4
months.
Phase:
Phase 3
Details
Lead Sponsor:
Sheba Medical Center
Treatments:
Colchicine Interleukin 1 Receptor Antagonist Protein