{"id":7683,"date":"2026-04-21T17:37:11","date_gmt":"2026-04-21T14:37:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7683"},"modified":"2026-04-21T17:37:12","modified_gmt":"2026-04-21T14:37:12","slug":"elevidys-phase-4-enhance-clinical-trial-nct07542314","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/elevidys-phase-4-enhance-clinical-trial-nct07542314\/","title":{"rendered":"\u00c9tude ELEVIDYS ENHANCE\u00a0: D\u00e9but d\u2019un essai de phase\u00a04 pour \u00e9valuer l\u2019innocuit\u00e9 et l\u2019efficacit\u00e9 du traitement de la dystrophie musculaire de Duchenne."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>L\u2019\u00e9tude ENHANCE est un essai clinique de phase 4 con\u00e7u pour \u00e9valuer la s\u00e9curit\u00e9 et l\u2019efficacit\u00e9 r\u00e9elles d\u2019ELEVIDYS chez les patients atteints de dystrophie musculaire de Duchenne (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Cette \u00e9tude est particuli\u00e8rement importante car elle porte sur des patients trait\u00e9s dans un contexte post-commercialisation, c&#039;est-\u00e0-dire apr\u00e8s que le traitement a d\u00e9j\u00e0 \u00e9t\u00e9 approuv\u00e9 et est utilis\u00e9 en pratique clinique.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;essai est sponsoris\u00e9 par Sarepta Therapeutics et porte l&#039;identifiant d&#039;\u00e9tude SRP-9001-402.<\/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=\"#primary-goal-understanding-liver-safety-risks\">Objectif principal : Comprendre les risques pour la s\u00e9curit\u00e9 du foie<\/a><\/li><li><a href=\"#study-design-and-key-details\">Conception de l&#039;\u00e9tude et d\u00e9tails cl\u00e9s<\/a><\/li><li><a href=\"#who-can-participate\">Qui peut participer ?<\/a><\/li><li><a href=\"#who-cannot-participate\">Qui ne peut pas participer ?<\/a><\/li><li><a href=\"#why-this-study-is-important-for-the-dmd-community\">Pourquoi cette \u00e9tude est importante pour la communaut\u00e9 DMD<\/a><\/li><li><a href=\"#contact-information\">Coordonn\u00e9es<\/a><\/li><li><a href=\"#clinical-trial-locations-not-yet-announced\">Les lieux des essais cliniques n&#039;ont pas encore \u00e9t\u00e9 annonc\u00e9s.<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"primary-goal-understanding-liver-safety-risks\">Objectif principal : Comprendre les risques pour la s\u00e9curit\u00e9 du foie<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-liver-safety-matters\">Pourquoi la protection du foie est importante<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">L\u2019une des principales pr\u00e9occupations concernant les th\u00e9rapies g\u00e9niques comme ELEVIDYS est le risque de l\u00e9sions h\u00e9patiques aigu\u00ebs (LHA).<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"role-of-sirolimus\">R\u00f4le du sirolimus<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">L\u2019\u00e9tude \u00e9value si l\u2019ajout de sirolimus comme traitement immunosuppresseur pr\u00e9ventif (prophylactique) peut contribuer \u00e0 r\u00e9duire les complications h\u00e9patiques.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En termes simples :<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS contribue \u00e0 d\u00e9livrer un g\u00e8ne de dystrophine fonctionnel.<\/li>\n\n\n\n<li>Le sirolimus contribue \u00e0 contr\u00f4ler la r\u00e9action du syst\u00e8me immunitaire au traitement.<\/li>\n\n\n\n<li>L\u2019objectif est de rendre le traitement plus s\u00fbr sans en r\u00e9duire l\u2019efficacit\u00e9.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"study-design-and-key-details\">Conception de l&#039;\u00e9tude et d\u00e9tails cl\u00e9s<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"trial-overview\">Aper\u00e7u de l&#039;essai<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Type d&#039;\u00e9tude :<\/strong> interventionnel<\/li>\n\n\n\n<li><strong>Phase:<\/strong> Phase 4<\/li>\n\n\n\n<li><strong>Effectifs estim\u00e9s\u00a0:<\/strong> 20 participants<\/li>\n\n\n\n<li><strong>Date de d\u00e9but:<\/strong> 30 avril 2026<\/li>\n\n\n\n<li><strong>Date d&#039;ach\u00e8vement :<\/strong> 31 mars 2027<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"study-groups-cohorts\">Groupes d&#039;\u00e9tude (cohortes)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;essai comprend deux groupes de participants\u00a0:<\/p>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"cohort-1\">Cohorte 1 :<\/h4>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Gar\u00e7ons \u00e2g\u00e9s de 4 ans et plus<\/li>\n\n\n\n<li>Doit \u00eatre capable de se d\u00e9placer (pouvoir marcher)<\/li>\n\n\n\n<li>\u00c9ligible au traitement commercial ELEVIDYS<\/li>\n<\/ul>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"cohort-2\">Cohorte 2 :<\/h4>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Traitement ant\u00e9rieur par ELEVIDYS<\/li>\n\n\n\n<li>J&#039;avais d\u00e9j\u00e0 re\u00e7u du sirolimus et des corticost\u00e9ro\u00efdes avant le traitement.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-can-participate\">Qui peut participer ?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"key-inclusion-criteria\">Crit\u00e8res d&#039;inclusion cl\u00e9s<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Les participants doivent :<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00catre de sexe masculin \u00e0 la naissance<\/li>\n\n\n\n<li>R\u00e9pondre aux crit\u00e8res d&#039;\u00e2ge et d&#039;admissibilit\u00e9 au traitement<\/li>\n\n\n\n<li>Avoir un aidant ou \u00eatre capable de suivre les proc\u00e9dures de l&#039;\u00e9tude<\/li>\n\n\n\n<li>Fournir un consentement \u00e9clair\u00e9 (ou faire en sorte qu&#039;un tuteur l\u00e9gal le fasse)<\/li>\n\n\n\n<li>Utilisez une contraception appropri\u00e9e si vous \u00eates sexuellement active.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"who-cannot-participate\">Qui ne peut pas participer ?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"key-exclusion-criteria\">Principaux crit\u00e8res d&#039;exclusion<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Les participants ne peuvent pas adh\u00e9rer s&#039;ils\u00a0:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Impossible de recevoir ELEVIDYS en toute s\u00e9curit\u00e9<\/li>\n\n\n\n<li>Avoir des infections telles que\u00a0:\n<ul class=\"wp-block-list\">\n<li>VIH<\/li>\n\n\n\n<li>H\u00e9patite B ou C<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Avez-vous r\u00e9cemment contract\u00e9 une infection ou re\u00e7u une vaccination\u00a0?<\/li>\n\n\n\n<li>Pr\u00e9senter des conditions susceptibles d&#039;affecter la s\u00e9curit\u00e9 ou les r\u00e9sultats de l&#039;\u00e9tude<\/li>\n\n\n\n<li>Ne tol\u00e8re pas le sirolimus<\/li>\n\n\n\n<li>Pr\u00e9sentent des plaies ou des blessures susceptibles de mal cicatriser sous immunosuppression.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-this-study-is-important-for-the-dmd-community\">Pourquoi cette \u00e9tude est importante pour la communaut\u00e9 DMD<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"real-world-evidence\">Preuves issues du monde r\u00e9el<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Contrairement aux essais pr\u00e9c\u00e9dents, cette \u00e9tude refl\u00e8te les conditions r\u00e9elles de traitement, aidant ainsi les m\u00e9decins \u00e0 comprendre\u00a0:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Dans quelle mesure ELEVIDYS est-il s\u00fbr en dehors des environnements contr\u00f4l\u00e9s ?<\/li>\n\n\n\n<li>Comment les patients r\u00e9agissent au fil du temps<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"improving-treatment-safety\">Am\u00e9liorer la s\u00e9curit\u00e9 des traitements<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Si l&#039;\u00e9tude s&#039;av\u00e8re concluante, elle pourrait permettre de\u00a0:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>R\u00e9duire les risques li\u00e9s au foie<\/li>\n\n\n\n<li>Am\u00e9liorer les protocoles de traitement<\/li>\n\n\n\n<li>Accro\u00eetre la confiance dans l&#039;utilisation de la th\u00e9rapie g\u00e9nique<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"contact-information\">Coordonn\u00e9es<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Pour plus de d\u00e9tails sur l&#039;\u00e9tude\u00a0:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Organisation:<\/strong> Sarepta Th\u00e9rapeutique<\/li>\n\n\n\n<li><strong>T\u00e9l\u00e9phone:<\/strong> 1-888-SAREPTA (1-888-727-3782)<\/li>\n\n\n\n<li><strong>E-mail:<\/strong> <a>SareptAlly@sarepta.com<\/a><\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"clinical-trial-locations-not-yet-announced\">Les lieux des essais cliniques n&#039;ont pas encore \u00e9t\u00e9 annonc\u00e9s.<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Les lieux de l&#039;essai clinique ENHANCE n&#039;ont pas encore \u00e9t\u00e9 rendus publics. Le promoteur de l&#039;\u00e9tude, Sarepta Therapeutics, devrait annoncer les centres participants ult\u00e9rieurement. Les patients, leurs aidants et les professionnels de sant\u00e9 sont invit\u00e9s \u00e0 consulter r\u00e9guli\u00e8rement les mises \u00e0 jour officielles d\u00e8s que les informations relatives aux centres seront disponibles.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Suivre cette page<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Tous les essais cliniques pour la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>The ENHANCE study is a Phase 4 clinical trial designed to evaluate the real-world safety and effectiveness of ELEVIDYS in patients with Duchenne muscular dystrophy (DMD). This study is particularly important because it focuses on patients treated in a post-marketing setting, meaning after the therapy has already been approved and is being used in clinical [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7684,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,33,179,702,61,65,703],"class_list":["post-7683","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-elevidys","tag-elevidys-gene-therapy","tag-enhance","tag-gene-therapy","tag-sarepta","tag-sirolimus"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/7683","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=7683"}],"version-history":[{"count":1,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/7683\/revisions"}],"predecessor-version":[{"id":7685,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/7683\/revisions\/7685"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/7684"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=7683"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=7683"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=7683"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}