Open Label Treprostinil Raynaud's Study

Summary

Raynaud's phenomenon is a condition where the blood vessels in participants fingers and toes get too narrow when cold or stressed. This makes participants fingers and toes change colors - they might turn white, then blue, and finally red as blood flow returns. It can be painful and cause numbness or tingling. When participants have Raynaud's, blood vessels react too strongly to cold or stress. Fingers and toes may turn white (blood moves away from the area), blue (lack of oxygen), or red and feel painful or tingly when warming up. These episodes usually last from a few minutes to several hours. There are two types of Raynaud's. Primary Raynaud's (also called Raynaud's disease) itself and isn't connected to other health problems. It's the most common type and affects mostly women under 30. Secondary Raynaud's (also called Raynaud's phenomenon) is caused by other diseases like lupus, scleroderma, or rheumatoid arthritis. This type tends to be more serious and may cause painful sores on fingertips called digital ulcers. For mild cases, staying warm might be enough. But if symptoms are severe, participants doctor might prescribe various medications including calcium channel blockers - blood pressure medicines that help open blood vessels, or other vasodilators - medicines that widen blood vessels. About 40% of people with scleroderma develop painful sores on their fingertips called digital ulcers. These happen when there isn't enough blood flow to heal small injuries. For severe cases with digital ulcers, doctors might use prostacyclin therapy - medicines that mimic a natural substance that opens blood vessels. Oral treprostinil is a newer pill form of prostacyclin therapy that helps improve blood flow. The investigators are conducting a research study testing whether oral treprostinil - a pill that mimics prostacyclin (a natural blood vessel opener) - can help people with severe Raynaud's that doesn't respond to usual treatments. This represents hope for better treatment options for people with the most challenging cases of this condition.

Timeline

Start

2025-10-15

Primary completion

2028-09-30

Completion

2029-03-01

Publications

• Background Maverakis E, Patel F, Kronenberg DG, Chung L, Fiorentino D, Allanore Y, Guiducci S, Hesselstrand R, Hummers LK, Duong C, Kahaleh B, Macgregor A, Matucci-Cerinic M, Wollheim FA, Mayes MD, Gershwin ME. International consensus criteria for the diagnosis of Raynaud's phenomenon. J Autoimmun. 2014 Feb-Mar;48-49:60-5. doi: 10.1016/j.jaut.2014.01.020. Epub 2014 Feb 1.
• Background Shah AA, Schiopu E, Hummers LK, Wade M, Phillips K, Anderson C, Wise R, Boin F, Seibold JR, Wigley F, Rollins KD. Open label study of escalating doses of oral treprostinil diethanolamine in patients with systemic sclerosis and digital ischemia: pharmacokinetics and correlation with digital perfusion. Arthritis Res Ther. 2013 Apr 18;15(2):R54. doi: 10.1186/ar4216.
• Background Milio G, Corrado E, Genova C, Amato C, Raimondi F, Almasio PL, Novo S. Iloprost treatment in patients with Raynaud's phenomenon secondary to systemic sclerosis and the quality of life: a new therapeutic protocol. Rheumatology (Oxford). 2006 Aug;45(8):999-1004. doi: 10.1093/rheumatology/kel038. Epub 2006 Feb 16.
• Background Roustit M, Blaise S, Allanore Y, Carpentier PH, Caglayan E, Cracowski JL. Phosphodiesterase-5 inhibitors for the treatment of secondary Raynaud's phenomenon: systematic review and meta-analysis of randomised trials. Ann Rheum Dis. 2013 Oct;72(10):1696-9. doi: 10.1136/annrheumdis-2012-202836. Epub 2013 Feb 20.
• Background Denton CP, Hachulla E, Riemekasten G, Schwarting A, Frenoux JM, Frey A, Le Brun FO, Herrick AL; Raynaud Study Investigators. Efficacy and Safety of Selexipag in Adults With Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Randomized, Placebo-Controlled, Phase II Study. Arthritis Rheumatol. 2017 Dec;69(12):2370-2379. doi: 10.1002/art.40242.
• Background Garcia de la Pena Lefebvre P, Nishishinya MB, Pereda CA, Loza E, Sifuentes Giraldo WA, Roman Ivorra JA, Carreira P, Rua-Figueroa I, Pego-Reigosa JM, Munoz-Fernandez S. Efficacy of Raynaud's phenomenon and digital ulcer pharmacological treatment in systemic sclerosis patients: a systematic literature review. Rheumatol Int. 2015 Sep;35(9):1447-59. doi: 10.1007/s00296-015-3241-1. Epub 2015 Apr 1.
• Background Costa E, Cunha-Santos F, Dourado E, Oliveira D, Falzon L, Romao V, Duarte AC, Cordeiro A, Santiago T, Sepriano A. Systematic literature review to inform the Portuguese recommendations for the management of Raynaud's phenomenon and digital ulcers in systemic sclerosis and other connective tissue diseases. ARP Rheumatol. 2024 Jul 1;3(Apr-Jun):128-144. doi: 10.63032/YHBL8967.
• Background Khouri C, Lepelley M, Bailly S, Blaise S, Herrick AL, Matucci-Cerinic M, Allanore Y, Trinquart L, Cracowski JL, Roustit M. Comparative efficacy and safety of treatments for secondary Raynaud's phenomenon: a systematic review and network meta-analysis of randomised trials. Lancet Rheumatol. 2019 Dec;1(4):e237-e246. doi: 10.1016/S2665-9913(19)30079-7.
• Background Flavahan NA, Flavahan S, Mitra S, Chotani MA. The vasculopathy of Raynaud's phenomenon and scleroderma. Rheum Dis Clin North Am. 2003 May;29(2):275-91, vi. doi: 10.1016/s0889-857x(03)00021-8.
• Background Stjernbrandt A, Pettersson H, Lundstrom R, Liljelind I, Nilsson T, Wahlstrom J. Incidence, remission, and persistence of Raynaud's phenomenon in the general population of northern Sweden: a prospective study. BMC Rheumatol. 2022 Jul 21;6(1):41. doi: 10.1186/s41927-022-00272-0.
• Background Maciejewska M, Sikora M, Maciejewski C, Alda-Malicka R, Czuwara J, Rudnicka L. Raynaud's Phenomenon with Focus on Systemic Sclerosis. J Clin Med. 2022 Apr 28;11(9):2490. doi: 10.3390/jcm11092490.
• Background Curtiss P, Svigos K, Schwager Z, Lo Sicco K, Franks AG Jr. Part I: Epidemiology, pathophysiology, and clinical considerations of primary and secondary Raynaud's phenomenon. J Am Acad Dermatol. 2024 Feb;90(2):223-234. doi: 10.1016/j.jaad.2022.06.1199. Epub 2022 Jul 7.

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Indications