{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T12:03:06Z","timestamp":1789041786745,"version":"build-2803163510"},"reference-count":28,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2020,1,20]],"date-time":"2020-01-20T00:00:00Z","timestamp":1579478400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009954","name":"Taiho Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009954","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Duchenne muscular dystrophy (DMD) is a progressive muscular disease characterized by chronic cycles of inflammatory and necrotic processes. Prostaglandin D<jats:sub>2<\/jats:sub> (PGD<jats:sub>2<\/jats:sub>) is produced by hematopoietic PGD synthase (HPGDS), which is pathologically implicated in muscle necrosis. This randomized, double\u2010blind, placebo\u2010controlled early phase 2 study (NCT02752048) aimed to assess the efficacy and safety of the novel selective HPGDS inhibitor, TAS\u2010205, with exploratory measures in male DMD patients aged \u22655\u00a0years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients were randomized 1:1:1 to receive low\u2010dose TAS\u2010205 (6.67\u201313.33\u00a0mg\/kg\/dose), high\u2010dose TAS\u2010205 (13.33\u201326.67\u00a0mg\/kg\/dose), or placebo. The primary endpoint was the change from baseline in a 6\u2010minute walk distance (6MWD) at Week 24.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Thirty\u2010six patients were enrolled, of whom 35 patients were analysed for safety. The mean (standard error) changes from baseline to Week 24 in 6MWD were \u221217.0 (17.6) m in the placebo group (<jats:italic>n<\/jats:italic>\u00a0=\u00a010), \u22123.5 (20.3) m in the TAS\u2010205 low\u2010dose group (<jats:italic>n<\/jats:italic>\u00a0=\u00a011), and \u22127.5 (11.2) m in the TAS\u2010205 high\u2010dose group (<jats:italic>n<\/jats:italic>\u00a0=\u00a011). The mean (95% confidence interval) difference from the placebo group was 13.5 (\u221243.3 to 70.2) m in the TAS\u2010205 low\u2010dose group and 9.5 (\u221233.3 to 52.4) m in the TAS\u2010205 high\u2010dose group. No obvious differences were observed in the incidences of adverse events between treatment groups. No adverse drug reactions specific to TAS\u2010205 treatment were observed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>The HPGDS inhibitor TAS\u2010205 showed a favorable safety profile in DMD patients. Further research is required to examine the effectiveness of TAS\u2010205 in a larger trial.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.50978","type":"journal-article","created":{"date-parts":[[2020,1,20]],"date-time":"2020-01-20T10:14:41Z","timestamp":1579515281000},"page":"181-190","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":31,"title":["Early phase 2 trial of TAS\u2010205 in patients with Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0659-1417","authenticated-orcid":false,"given":"Hirofumi","family":"Komaki","sequence":"first","affiliation":[{"name":"Department of Child Neurology National Center Hospital National Center of Neurology and Psychiatry Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshihiro","family":"Maegaki","sequence":"additional","affiliation":[{"name":"Division of Child Neurology Department of Brain and 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