{"id":5323,"date":"2025-12-15T21:53:45","date_gmt":"2025-12-15T18:53:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5323"},"modified":"2025-12-15T21:57:24","modified_gmt":"2025-12-15T18:57:24","slug":"ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/","title":{"rendered":"\u00c7in Merkezli Ractigen Therapeutics, DMD Hastalar\u0131nda RAG-18'in G\u00fcvenli\u011fini ve Tolerans\u0131n\u0131 Ara\u015ft\u0131rmak \u00dczere Klinik Denemelere Ba\u015fl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Ractigen Therapeutics&#039;ye g\u00f6re, bu \u00e7al\u0131\u015fma, Duchenne Musk\u00fcler Distrofisi (DMD) olan pediatrik hastalarda RAG-18&#039;in g\u00fcvenli\u011fini, tolerans\u0131n\u0131, farmakokineti\u011fini (PK), farmakodinami\u011fini (PD) ve \u00f6n etkinli\u011fini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f, a\u00e7\u0131k etiketli, tek kollu, doz art\u0131r\u0131ml\u0131 bir \u00e7al\u0131\u015fmad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmada, artan intraven\u00f6z dozlar\u0131n g\u00fcvenli\u011fini ve tolerans\u0131n\u0131 de\u011ferlendirmek amac\u0131yla d\u00f6rt kohorta yakla\u015f\u0131k 12 denek dahil edilecektir. \u0130kincil ama\u00e7lar aras\u0131nda farmakokinetik (PK)\/farmakodinamik (PD) profilin karakterize edilmesi ve kas biyobelirte\u00e7lerinde, kas bile\u015fiminde, kalp\/akci\u011fer fonksiyonunda ve motor performansta meydana gelen de\u011fi\u015fiklikler yoluyla deneysel etkinli\u011fin de\u011ferlendirilmesi yer almaktad\u0131r. Bir sonraki doz seviyesine ge\u00e7me karar\u0131, \u00f6nceki kohortun kapsaml\u0131 bir g\u00fcvenlik de\u011ferlendirmesine dayanacakt\u0131r. <strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ractigen Therapeutics, Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in \u00c7\u0131\u011f\u0131r A\u00e7\u0131c\u0131 Bir SaRNA Terapisi Olan RAG-18&#039;in IIT \u00c7al\u0131\u015fmas\u0131nda \u0130lk Hastaya Doz Verdi<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#what-is-rag-18-sa-rna\">RAG-18 saRNA nedir?<\/a><\/li><li><a href=\"#rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Erken Faz 1 \u2013 NCT07282652<\/a><\/li><li><a href=\"#how-to-participate-in-the-wve-n-531-clinical-trial\">RAG-18 saRNA Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131labilirsiniz?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-rag-18-sa-rna\">RAG-18 saRNA nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RAG-18, RNAa mekanizmas\u0131 yoluyla kas h\u00fccrelerinde UTRN gen ekspresyonunu spesifik olarak hedeflemek ve aktive etmek \u00fczere tasarlanm\u0131\u015f, t\u00fcr\u00fcn\u00fcn ilk \u00f6rne\u011fi bir saRNA aday\u0131d\u0131r.<\/strong> UTRN geni taraf\u0131ndan kodlanan utrofin proteini, yap\u0131sal ve i\u015flevsel olarak distrofine benzer ve yukar\u0131 do\u011fru d\u00fczenlenmesi, DMD kas h\u00fccrelerinde eksik olan distrofinin i\u015flevsel bir ikamesi olarak potansiyel olarak hizmet edebilir ve spesifik mutasyon konumundan ba\u011f\u0131ms\u0131z olarak t\u00fcm DMD hastalar\u0131 i\u00e7in tedavi sa\u011flayabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Erken Faz 1 \u2013 NCT07282652<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne Musk\u00fcler Distrofisi Olan Pediatrik Hastalarda RAG-18&#039;in G\u00fcvenli\u011fi\/Tolerans\u0131, Farmakokineti\u011fi, Farmakodinami\u011fi ve \u00d6n Etkinli\u011finin De\u011ferlendirilmesi Amac\u0131yla Ara\u015ft\u0131rmac\u0131 Taraf\u0131ndan Ba\u015flat\u0131lan Bir \u00c7al\u0131\u015fma.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma Ba\u015flang\u0131c\u0131 (Tahmini)<\/strong>: 2025-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birincil Tamamlanma (Tahmini)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Tamamlanmas\u0131 (Tahmini)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kat\u0131l\u0131mc\u0131 Say\u0131s\u0131 (Tahmini)<\/strong>: 12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma T\u00fcr\u00fc<\/strong>: M\u00fcdahaleci<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Konum<\/strong>: \u00c7in<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fmaya Uygun Ya\u015flar<\/strong>: 4 Ya\u015f - 15 Ya\u015f (\u00c7ocuk)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Temel dahil etme ve hari\u00e7 tutma kriterleri. \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07282652\" target=\"_blank\" data-type=\"link\" data-id=\"https:\/\/clinicaltrials.gov\/study\/NCT07282652\" rel=\"noreferrer noopener\">NCT07282652<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-wve-n-531-clinical-trial\">RAG-18 saRNA Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131labilirsiniz?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">RAG-18 saRNA Erken Faz 1 klinik \u00e7al\u0131\u015fmas\u0131na kat\u0131lmak i\u00e7in a\u015fa\u011f\u0131daki ileti\u015fim bilgilerini kullanabilirsiniz.\u00a0<strong>Daha fazla bilgi edin<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne (DMD) \u0130\u00e7in Klinik Ara\u015ft\u0131rmalara Nas\u0131l Kat\u0131labilirsiniz?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130sim<\/strong>: Yi Dai<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Telefon Numaras\u0131<\/strong>: +86 138 111 77 531<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-posta<\/strong>:\u00a0pumchdy@pumch.cn<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>According to Ractigen Therapeutics, this study is an open-label, single-arm, dose-escalation study designed to evaluate the safety, tolerance, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular Dystrophy (DMD). The study will enroll approximately 12 subjects into four cohorts to assess the safety and tolerability of ascending intravenous doses. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5327,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,137,242,466,467,465,468],"class_list":["post-5323","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-clinical-trial","tag-clinical-trials","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrn-expression"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5323","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5323"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5323\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5327"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5323"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5323"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5323"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}