Topical Ruxolitinib in the Treatment of Adults With Pyoderma Gangrenosum

Summary

This is a single-center, open-label, single-arm pilot study evaluating topical ruxolitinib (OPZELURA) as a treatment for adults with Pyoderma Gangrenosum (PG), a chronic inflammatory skin condition that causes painful ulcers. Approximately 12 participants will apply ruxolitinib cream twice daily for 24 weeks while continuing standard wound care, with the goal of determining whether the drug helps target ulcers heal completely without the need for systemic immunosuppressive therapy. Participants will be followed for a total of 28 weeks, with the option to continue treatment in a 48-week open-label extension if they respond well and remain safe on the drug. The study is sponsored by Oregon Health \& Science University and funded by Incyte Corporation.

Timeline

Start

2026-09-01

Primary completion

2029-09

Completion

2029-12

Publications

• Background Spitzer RL, Kroenke K, Williams JB, Lowe B. A brief measure for assessing generalized anxiety disorder: the GAD-7. Arch Intern Med. 2006 May 22;166(10):1092-7. doi: 10.1001/archinte.166.10.1092.
• Background Ortega-Loayza AG, Friedman MA, Reese AM, Liu Y, Greiling TM, Cassidy PB, Marzano AV, Gao L, Fei SS, Rosenbaum JT. Molecular and Cellular Characterization of Pyoderma Gangrenosum: Implications for the Use of Gene Expression. J Invest Dermatol. 2022 Apr;142(4):1217-1220.e14. doi: 10.1016/j.jid.2021.08.431. Epub 2021 Sep 15. No abstract available.
• Background Orfaly VE, Kovalenko I, Tolkachjov SN, Ortega-Loayza AG, Nunley JR. Tofacitinib for the treatment of refractory pyoderma gangrenosum. Clin Exp Dermatol. 2021 Aug;46(6):1082-1085. doi: 10.1111/ced.14683. Epub 2021 May 29.
• Background Rosmarin D, Pandya AG, Lebwohl M, Grimes P, Hamzavi I, Gottlieb AB, Butler K, Kuo F, Sun K, Ji T, Howell MD, Harris JE. Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. Lancet. 2020 Jul 11;396(10244):110-120. doi: 10.1016/S0140-6736(20)30609-7.
• Background Kim BS, Howell MD, Sun K, Papp K, Nasir A, Kuligowski ME; INCB 18424-206 Study Investigators. Treatment of atopic dermatitis with ruxolitinib cream (JAK1/JAK2 inhibitor) or triamcinolone cream. J Allergy Clin Immunol. 2020 Feb;145(2):572-582. doi: 10.1016/j.jaci.2019.08.042. Epub 2019 Oct 17.
• Background Sedano R, Jairath V. Tofacitinib for the Treatment of Three Immune-mediated Conditions in One Patient: Ulcerative Colitis, Pyoderma Gangrenosum, and Alopecia Areata. Inflamm Bowel Dis. 2021 Apr 15;27(5):e65. doi: 10.1093/ibd/izab005. No abstract available.
• Background Ortega-Loayza AG, Nugent WH, Lucero OM, Washington SL, Nunley JR, Walsh SW. Dysregulation of inflammatory gene expression in lesional and nonlesional skin of patients with pyoderma gangrenosum. Br J Dermatol. 2018 Jan;178(1):e35-e36. doi: 10.1111/bjd.15837. Epub 2017 Dec 5. No abstract available.
• Background Palanivel JA, Macbeth AE, Levell NJ. Pyoderma gangrenosum in association with Janus kinase 2 (JAK2V617F) mutation. Clin Exp Dermatol. 2013 Jan;38(1):44-6. doi: 10.1111/j.1365-2230.2012.04375.x. Epub 2012 May 21.
• Background Kochar B, Herfarth N, Mamie C, Navarini AA, Scharl M, Herfarth HH. Tofacitinib for the Treatment of Pyoderma Gangrenosum. Clin Gastroenterol Hepatol. 2019 Apr;17(5):991-993. doi: 10.1016/j.cgh.2018.10.047. Epub 2018 Nov 4.
• Background Nasifoglu S, Heinrich B, Welzel J. Successful therapy for pyoderma gangrenosum with a Janus kinase 2 inhibitor. Br J Dermatol. 2018 Aug;179(2):504-505. doi: 10.1111/bjd.16468. Epub 2018 May 21. No abstract available.
• Background Shanmugam VK, McNish S, Shara N, Hubley KJ, Kallakury B, Dunning DM, Attinger CE, Steinberg JS. Chronic leg ulceration associated with polycythemia vera responding to ruxolitinib (Jakafi((R))). J Foot Ankle Surg. 2013 Nov-Dec;52(6):781-5. doi: 10.1053/j.jfas.2013.07.003. Epub 2013 Aug 14.
• Background Alves de Medeiros AK, Speeckaert R, Desmet E, Van Gele M, De Schepper S, Lambert J. JAK3 as an Emerging Target for Topical Treatment of Inflammatory Skin Diseases. PLoS One. 2016 Oct 6;11(10):e0164080. doi: 10.1371/journal.pone.0164080. eCollection 2016.
• Background Siegfried EC, Jaworski JC, Kaiser JD, Hebert AA. Systematic review of published trials: long-term safety of topical corticosteroids and topical calcineurin inhibitors in pediatric patients with atopic dermatitis. BMC Pediatr. 2016 Jun 7;16:75. doi: 10.1186/s12887-016-0607-9.
• Background Del Rosso J, Friedlander SF. Corticosteroids: options in the era of steroid-sparing therapy. J Am Acad Dermatol. 2005 Jul;53(1 Suppl 1):S50-8. doi: 10.1016/j.jaad.2005.04.030.
• Background Thomas KS, Ormerod AD, Craig FE, Greenlaw N, Norrie J, Mitchell E, Mason JM, Johnston GA, Wahie S, Williams HC; UK Dermatology Clinical Trials Network's STOP GAP Team. Clinical outcomes and response of patients applying topical therapy for pyoderma gangrenosum: A prospective cohort study. J Am Acad Dermatol. 2016 Nov;75(5):940-949. doi: 10.1016/j.jaad.2016.06.016. Epub 2016 Aug 5.
• Background Braswell SF, Kostopoulos TC, Ortega-Loayza AG. Pathophysiology of pyoderma gangrenosum (PG): an updated review. J Am Acad Dermatol. 2015 Oct;73(4):691-8. doi: 10.1016/j.jaad.2015.06.021. Epub 2015 Aug 5.

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Indications