{"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\/fr\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/","title":{"rendered":"La soci\u00e9t\u00e9 chinoise Ractigen Therapeutics lance des essais cliniques pour \u00e9tudier l&#039;innocuit\u00e9 et la tol\u00e9rance du RAG-18 chez les patients atteints de DMD."},"content":{"rendered":"<p class=\"wp-block-paragraph\">Selon Ractigen Therapeutics, cette \u00e9tude est une \u00e9tude ouverte, \u00e0 un seul bras, \u00e0 augmentation de dose con\u00e7ue pour \u00e9valuer la s\u00e9curit\u00e9, la tol\u00e9rance, la pharmacocin\u00e9tique (PK), la pharmacodynamique (PD) et l&#039;efficacit\u00e9 pr\u00e9liminaire du RAG-18 chez les patients p\u00e9diatriques atteints de dystrophie musculaire de Duchenne (DMD).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;\u00e9tude inclura environ 12 sujets r\u00e9partis en quatre cohortes afin d&#039;\u00e9valuer la s\u00e9curit\u00e9 et la tol\u00e9rance de doses intraveineuses croissantes. Les objectifs secondaires comprennent la caract\u00e9risation du profil pharmacocin\u00e9tique (PK)\/pharmacodynamique (PD) et l&#039;\u00e9valuation exploratoire de l&#039;efficacit\u00e9 par l&#039;analyse des variations des biomarqueurs musculaires, de la composition musculaire, des fonctions cardiopulmonaire et motrice. La d\u00e9cision de passer \u00e0 la dose sup\u00e9rieure sera fond\u00e9e sur une \u00e9valuation compl\u00e8te de la s\u00e9curit\u00e9 r\u00e9alis\u00e9e aupr\u00e8s de la cohorte pr\u00e9c\u00e9dente. <strong>En savoir plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ractigen Therapeutics administre la premi\u00e8re dose \u00e0 un patient dans le cadre d&#039;une \u00e9tude de l&#039;IIT sur RAG-18, une th\u00e9rapie saRNA potentiellement r\u00e9volutionnaire pour la dystrophie musculaire de Duchenne.<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Table des mati\u00e8res<\/h2><nav><ul><li><a href=\"#what-is-rag-18-sa-rna\">Qu&#039;est-ce que le saRNA RAG-18\u00a0?<\/a><\/li><li><a href=\"#rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Phase pr\u00e9coce 1 \u2013 NCT07282652<\/a><\/li><li><a href=\"#how-to-participate-in-the-wve-n-531-clinical-trial\">Comment participer \u00e0 l&#039;essai clinique RAG-18 saRNA<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-rag-18-sa-rna\">Qu&#039;est-ce que le saRNA RAG-18\u00a0?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RAG-18 est un candidat saRNA unique en son genre, con\u00e7u pour cibler et activer sp\u00e9cifiquement l&#039;expression du g\u00e8ne UTRN dans les cellules musculaires via le m\u00e9canisme RNAa.<\/strong> La prot\u00e9ine utrophine cod\u00e9e par le g\u00e8ne UTRN est structurellement et fonctionnellement similaire \u00e0 la dystrophine, et sa r\u00e9gulation positive pourrait potentiellement servir de remplacement fonctionnel \u00e0 la dystrophine manquante dans les cellules musculaires DMD, offrant un traitement \u00e0 tous les patients atteints de DMD, quel que soit l&#039;emplacement sp\u00e9cifique de la mutation.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA Phase pr\u00e9coce 1 \u2013 NCT07282652<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c9tude initi\u00e9e par un investigateur pour \u00e9valuer la s\u00e9curit\u00e9\/tol\u00e9rance, la pharmacocin\u00e9tique, la pharmacodynamique et l&#039;efficacit\u00e9 pr\u00e9liminaire du RAG-18 chez les patients p\u00e9diatriques atteints de dystrophie musculaire de Duchenne.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>D\u00e9but de l&#039;\u00e9tude (estim\u00e9)<\/strong>: 2025-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement du primaire (estim\u00e9)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement de l&#039;\u00e9tude (estim\u00e9)<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nombre de participants (estim\u00e9)<\/strong>: 12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Type d&#039;\u00e9tude<\/strong>: Interventionnel<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Emplacement<\/strong>: Chine<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c2ges admissibles aux \u00e9tudes<\/strong>: 4 ans \u00e0 15 ans (enfant)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Principaux crit\u00e8res d\u2019inclusion et d\u2019exclusion. \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\">Comment participer \u00e0 l&#039;essai clinique RAG-18 saRNA<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Pour participer \u00e0 l&#039;essai clinique de phase 1 pr\u00e9coce du saRNA RAG-18, vous pouvez utiliser les informations de contact ci-dessous.\u00a0<strong>Apprendre encore plus<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/fr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Comment participer aux essais cliniques pour la maladie de Duchenne (DMD) ?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nom<\/strong>: Yi Da\u00ef<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Num\u00e9ro de t\u00e9l\u00e9phone<\/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>En savoir plus<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Essais cliniques sur la dystrophie musculaire de Duchenne (Liste de toutes les recherches)<\/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"],"subtitle":"Ractigen Therapeutics, un d\u00e9veloppeur de premier plan de th\u00e9rapies \u00e0 base de petits ARN activateurs (saRNA), a annonc\u00e9 aujourd'hui qu'il allait entamer un essai clinique de phase 1 pr\u00e9coce de son produit \u00e0 base de saRNA RAG-18 pour la dystrophie musculaire de Duchenne.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5323","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=5323"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5323\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/5327"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=5323"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=5323"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=5323"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}