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Ann Rheum Dis doi:10.1136/ard.2009.108936

The IL-1 inhibitor rilonacept in treatment of chronic gouty arthritis: results of a placebo-controlled, monosequence crossover, nonrandomized, single-blind pilot study

  1. R Terkeltaub (rterkeltaub{at}ucsd.edu)
  1. VA Medical Center/University of California San Diego, United States
    1. J S Sundy (john.sundy{at}duke.edu)
    1. Duke University Medical Center, United States
      1. H R Schumacher
      1. University of Pennsylvania, VA Medical Center, United States
        1. F Murphy (ftmurphy{at}earthlink.net)
        1. Altoona Center for Clinical Research, United States
          1. S Bookbinder (ocalarr{at}aol.com)
          1. Ocala Rheumatology Research Center, United States
            1. S Biedermann (stephanie.biedermann{at}regeneron.com)
            1. Regeneron Pharmaceuticals, Inc., United States
              1. R Wu (richard.wu{at}regeneron.com)
              1. Regeneron Pharmaceuticals, Inc., United States
                1. S Mellis (scott.mellis{at}regeneron.com)
                1. Regeneron Pharmaceuticals, Inc., United States
                  1. A Radin (allen.radin{at}regeneron.com)
                  1. Regeneron Pharmaceuticals, Inc., United States
                    • Published Online First 26 July 2009

                    Abstract

                    Objectives: Recent studies suggest that blockade of the NLRP3 (cryopyrin) inflammasome IL-1β pathway may offer a new treatment strategy for gout. This proof-of-concept study explored the potential utility of rilonacept (IL-1 Trap) in patients with chronic active gouty arthritis.

                    Methods: This 14-week, multicenter, nonrandomized, single-blind, monosequence crossover study of 10 patients with chronic active gouty arthritis included a placebo run-in (2-weeks), active rilonacept treatment (6-weeks), and a 6 week post-treatment follow-up.

                    Results: Rilonacept was generally well tolerated. No deaths and no serious AEs occurred during the study. One patient withdrew due to an injection-site reaction. Patients’ self-reported median pain VAS scores significantly decreased from Week 2 (following placebo run-in) to Week 4 (2 weeks of rilonacept) (5.0 to 2.8; P < 0.049), with sustained improvement at Week 8 (1.3; P < 0.049); 5 of 10 patients reported at least a 75% improvement. Median symptom severity-adjusted joint scores were significantly decreased. hs-CRP levels fell significantly.

                    Conclusions: This proof-of-concept study demonstrated that rilonacept is generally well tolerated and may offer therapeutic benefit in reducing pain in patients with chronic refractory gouty arthritis, supporting the need for larger, randomized, controlled studies of IL-1 antagonism such as with rilonacept for this clinical indication.