{"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\/pt\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/","title":{"rendered":"A empresa chinesa Ractigen Therapeutics est\u00e1 iniciando ensaios cl\u00ednicos para investigar a seguran\u00e7a e a tolerabilidade do RAG-18 em pacientes com DMD (distrofia muscular de Duchenne)."},"content":{"rendered":"<p class=\"wp-block-paragraph\">De acordo com o protocolo Ractigen Therapeutics, este estudo \u00e9 um estudo aberto, de bra\u00e7o \u00fanico e de escalonamento de dose, projetado para avaliar a seguran\u00e7a, a toler\u00e2ncia, a farmacocin\u00e9tica (FC), a farmacodin\u00e2mica (FD) e a efic\u00e1cia preliminar do RAG-18 em pacientes pedi\u00e1tricos com distrofia muscular de Duchenne (DMD).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O estudo recrutar\u00e1 aproximadamente 12 participantes, divididos em quatro grupos, para avaliar a seguran\u00e7a e a tolerabilidade de doses intravenosas crescentes. Os objetivos secund\u00e1rios incluem a caracteriza\u00e7\u00e3o do perfil farmacocin\u00e9tico (FC)\/farmacodin\u00e2mico (FD) e a avalia\u00e7\u00e3o da efic\u00e1cia explorat\u00f3ria por meio de altera\u00e7\u00f5es em biomarcadores musculares, composi\u00e7\u00e3o muscular, fun\u00e7\u00e3o card\u00edaca\/pulmonar e desempenho motor. A decis\u00e3o de aumentar a dose para o pr\u00f3ximo n\u00edvel ser\u00e1 baseada em uma avalia\u00e7\u00e3o abrangente de seguran\u00e7a do grupo anterior. <strong>Ler mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ractigen Therapeutics administra a primeira dose em um paciente no estudo IIT de RAG-18, uma terapia saRNA com potencial para revolucionar o tratamento da distrofia muscular de Duchenne.<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u00cdndice<\/h2><nav><ul><li><a href=\"#what-is-rag-18-sa-rna\">O que \u00e9 RAG-18 saRNA?<\/a><\/li><li><a href=\"#rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Fase Inicial 1 \u2013 NCT07282652<\/a><\/li><li><a href=\"#how-to-participate-in-the-wve-n-531-clinical-trial\">Como participar do ensaio cl\u00ednico de RAG-18 saRNA<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-rag-18-sa-rna\">O que \u00e9 RAG-18 saRNA?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RAG-18 \u00e9 um candidato a saRNA in\u00e9dito, projetado para atingir e ativar especificamente a express\u00e3o do gene UTRN em c\u00e9lulas musculares por meio do mecanismo de RNAa.<\/strong> A prote\u00edna utrofina, codificada pelo gene UTRN, \u00e9 estrutural e funcionalmente semelhante \u00e0 distrofina, e sua regula\u00e7\u00e3o positiva poderia potencialmente servir como um substituto funcional para a distrofina ausente nas c\u00e9lulas musculares da DMD, proporcionando tratamento para todos os pacientes com DMD, independentemente da localiza\u00e7\u00e3o espec\u00edfica da muta\u00e7\u00e3o.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Fase Inicial 1 \u2013 NCT07282652<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Estudo iniciado por investigadores para avaliar a seguran\u00e7a\/tolerabilidade, farmacocin\u00e9tica, farmacodin\u00e2mica e efic\u00e1cia preliminar do RAG-18 em pacientes pedi\u00e1tricos com distrofia muscular de Duchenne.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>In\u00edcio do estudo (estimado)<\/strong>: 2025-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o prim\u00e1ria (estimada)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o do estudo (estimada)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de participantes (estimado)<\/strong>: 12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Tipo de estudo<\/strong>: Intervencionista<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Localiza\u00e7\u00e3o<\/strong>: China<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Idades eleg\u00edveis para estudo<\/strong>: 4 a 15 anos (Crian\u00e7a)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Principais crit\u00e9rios de inclus\u00e3o e exclus\u00e3o. \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\">Como participar do ensaio cl\u00ednico de RAG-18 saRNA<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Para participar do ensaio cl\u00ednico de Fase 1 inicial do RAG-18 saRNA, utilize as informa\u00e7\u00f5es de contato abaixo.\u00a0<strong>Saber mais<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/pt\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Como participar de ensaios cl\u00ednicos para Duchenne (DMD)?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nome<\/strong>: Yi Dai<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de telefone<\/strong>: +86 138 111 77 531<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-mail<\/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>Ler mais<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/pt\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ensaios cl\u00ednicos para distrofia muscular de Duchenne (lista de todas as pesquisas)<\/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\/pt\/wp-json\/wp\/v2\/posts\/5323","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=5323"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/5323\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/5327"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=5323"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=5323"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=5323"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}