Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF)

Summary

A study designed to evaluate the role of treatment with a biological agent - Canakinumab in pediatric (age 4-20) Familial Mediterranean Fever (FMF) patients that are intolerant or resistant for colchicine treatment. The study hypothesis is that Canakinumab will reduce attack frequency and severity.

Timeline

Start

2010-12

Primary completion

2012-02

Completion

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Publications

• Background Lidar M, Livneh A. Familial Mediterranean fever: clinical, molecular and management advancements. Neth J Med. 2007 Oct;65(9):318-24.
• Background Bhat A, Naguwa SM, Gershwin ME. Genetics and new treatment modalities for familial Mediterranean fever. Ann N Y Acad Sci. 2007 Sep;1110:201-8. doi: 10.1196/annals.1423.022.
• Background Mor A, Shinar Y, Zaks N, Langevitz P, Chetrit A, Shtrasburg S, Rabinovitz E, Livneh A. Evaluation of disease severity in familial Mediterranean fever. Semin Arthritis Rheum. 2005 Aug;35(1):57-64. doi: 10.1016/j.semarthrit.2005.02.002.
• Background Masters SL, Simon A, Aksentijevich I, Kastner DL. Horror autoinflammaticus: the molecular pathophysiology of autoinflammatory disease (*). Annu Rev Immunol. 2009;27:621-68. doi: 10.1146/annurev.immunol.25.022106.141627.
• Background Soylemezoglu O, Arga M, Fidan K, Gonen S, Emeksiz HC, Hasanoglu E, Buyan N. Unresponsiveness to colchicine therapy in patients with familial Mediterranean fever homozygous for the M694V mutation. J Rheumatol. 2010 Jan;37(1):182-9. doi: 10.3899/jrheum.090273. Epub 2009 Dec 15.
• Background Roldan R, Ruiz AM, Miranda MD, Collantes E. Anakinra: new therapeutic approach in children with Familial Mediterranean Fever resistant to colchicine. Joint Bone Spine. 2008 Jul;75(4):504-5. doi: 10.1016/j.jbspin.2008.04.001. Epub 2008 Jun 9.
• Background Kuijk LM, Govers AM, Frenkel J, Hofhuis WJ. Effective treatment of a colchicine-resistant familial Mediterranean fever patient with anakinra. Ann Rheum Dis. 2007 Nov;66(11):1545-6. doi: 10.1136/ard.2007.071498. No abstract available.
• Background Moser C, Pohl G, Haslinger I, Knapp S, Rowczenio D, Russel T, Lachmann HJ, Lang U, Kovarik J. Successful treatment of familial Mediterranean fever with Anakinra and outcome after renal transplantation. Nephrol Dial Transplant. 2009 Feb;24(2):676-8. doi: 10.1093/ndt/gfn646. Epub 2008 Nov 25.
• Background Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, Gitton X, Widmer A, Patel N, Hawkins PN; Canakinumab in CAPS Study Group. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med. 2009 Jun 4;360(23):2416-25. doi: 10.1056/NEJMoa0810787.
• Background Manuel O, Kumar D. QuantiFERON-TB Gold assay for the diagnosis of latent tuberculosis infection. Expert Rev Mol Diagn. 2008 May;8(3):247-56. doi: 10.1586/14737159.8.3.247.
• Results Brik R, Butbul-Aviel Y, Lubin S, Ben Dayan E, Rachmilewitz-Minei T, Tseng L, Hashkes PJ. Canakinumab for the treatment of children with colchicine-resistant familial Mediterranean fever: a 6-month open-label, single-arm pilot study. Arthritis Rheumatol. 2014 Nov;66(11):3241-3. doi: 10.1002/art.38777. No abstract available.

Drugs

Indications