{"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\/tr\/elevidys-phase-4-enhance-clinical-trial-nct07542314\/","title":{"rendered":"ELEVIDYS ENHANCE \u00c7al\u0131\u015fmas\u0131: Duchenne Kas Distrofisinde G\u00fcvenlik ve Etkinli\u011fi De\u011ferlendirmek \u0130\u00e7in Faz 4 \u00c7al\u0131\u015fmas\u0131 Ba\u015flad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>ENHANCE \u00e7al\u0131\u015fmas\u0131, Duchenne kas distrofisi (DMD) hastalar\u0131nda ELEVIDYS&#039;in ger\u00e7ek d\u00fcnya ko\u015fullar\u0131ndaki g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f bir Faz 4 klinik \u00e7al\u0131\u015fmas\u0131d\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc pazarlama sonras\u0131 d\u00f6nemde, yani tedavi zaten onayland\u0131ktan ve klinik uygulamada kullan\u0131lmaya ba\u015fland\u0131ktan sonra tedavi edilen hastalara odaklanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma Sarepta Therapeutics taraf\u0131ndan desteklenmektedir ve \u00e7al\u0131\u015fma kimlik numaras\u0131 SRP-9001-402&#039;dir.<\/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=\"#primary-goal-understanding-liver-safety-risks\">Birincil Ama\u00e7: Karaci\u011fer G\u00fcvenli\u011fi Risklerini Anlamak<\/a><\/li><li><a href=\"#study-design-and-key-details\">\u00c7al\u0131\u015fma Tasar\u0131m\u0131 ve Temel Ayr\u0131nt\u0131lar<\/a><\/li><li><a href=\"#who-can-participate\">Kimler Kat\u0131labilir?<\/a><\/li><li><a href=\"#who-cannot-participate\">Kimler kat\u0131lamaz?<\/a><\/li><li><a href=\"#why-this-study-is-important-for-the-dmd-community\">Bu \u00c7al\u0131\u015fma DMD Toplulu\u011fu \u0130\u00e7in Neden \u00d6nemli?<\/a><\/li><li><a href=\"#contact-information\">\u0130leti\u015fim Bilgileri<\/a><\/li><li><a href=\"#clinical-trial-locations-not-yet-announced\">Klinik Ara\u015ft\u0131rma Yerleri Hen\u00fcz A\u00e7\u0131klanmad\u0131<\/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\">Birincil Ama\u00e7: Karaci\u011fer G\u00fcvenli\u011fi Risklerini Anlamak<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-liver-safety-matters\">Karaci\u011fer G\u00fcvenli\u011fi Neden \u00d6nemlidir?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVIDYS gibi gen terapilerinde en b\u00fcy\u00fck endi\u015felerden biri akut karaci\u011fer hasar\u0131 (ALI) riskidir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"role-of-sirolimus\">Sirolimus&#039;un Rol\u00fc<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma, sirolimusun \u00f6nleyici (profilaktik) imm\u00fcnosupresif tedavi olarak eklenmesinin karaci\u011ferle ilgili komplikasyonlar\u0131 azaltmaya yard\u0131mc\u0131 olup olamayaca\u011f\u0131n\u0131 de\u011ferlendirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Basit\u00e7e ifade etmek gerekirse:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS, i\u015flevsel bir distrofin geninin iletilmesine yard\u0131mc\u0131 olur.<\/li>\n\n\n\n<li>Sirolimus, ba\u011f\u0131\u015f\u0131kl\u0131k sisteminin tedaviye verdi\u011fi tepkiyi kontrol etmeye yard\u0131mc\u0131 olur.<\/li>\n\n\n\n<li>Ama\u00e7, etkinli\u011fi azaltmadan tedaviyi daha g\u00fcvenli hale getirmektir.<\/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\">\u00c7al\u0131\u015fma Tasar\u0131m\u0131 ve Temel Ayr\u0131nt\u0131lar<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"trial-overview\">Deneme Genel Bak\u0131\u015f\u0131<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u00c7al\u0131\u015fma T\u00fcr\u00fc:<\/strong> Giri\u015fimsel<\/li>\n\n\n\n<li><strong>Faz:<\/strong> Faz 4<\/li>\n\n\n\n<li><strong>Tahmini Kay\u0131t Say\u0131s\u0131:<\/strong> 20 kat\u0131l\u0131mc\u0131<\/li>\n\n\n\n<li><strong>Ba\u015flang\u0131\u00e7 Tarihi:<\/strong> 30 Nisan 2026<\/li>\n\n\n\n<li><strong>Tamamlanma Tarihi:<\/strong> 31 Mart 2027<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"study-groups-cohorts\">\u00c7al\u0131\u015fma Gruplar\u0131 (Kohortlar)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmaya iki kat\u0131l\u0131mc\u0131 grubu dahil edilmi\u015ftir:<\/p>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"cohort-1\">1. Grup:<\/h4>\n\n\n\n<ul class=\"wp-block-list\">\n<li>4 ya\u015f ve \u00fczeri erkek \u00e7ocuklar<\/li>\n\n\n\n<li>Y\u00fcr\u00fcyebilecek durumda olmal\u0131d\u0131r<\/li>\n\n\n\n<li>Ticari ELEVIDYS tedavisi i\u00e7in uygun olmal\u0131d\u0131r<\/li>\n<\/ul>\n\n\n\n<h4 class=\"wp-block-heading\" id=\"cohort-2\">2. Grup:<\/h4>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Daha \u00f6nce ELEVIDYS ile tedavi edilmi\u015f olmal\u0131d\u0131r<\/li>\n\n\n\n<li>Tedaviden \u00f6nce sirolimus ve kortikosteroid alm\u0131\u015f olmal\u0131d\u0131r<\/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\">Kimler Kat\u0131labilir?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"key-inclusion-criteria\">Temel Dahil Edilme Kriterleri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Kat\u0131l\u0131mc\u0131lar\u0131n \u015funlar\u0131 yapmas\u0131 gerekmektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Do\u011fumda erkek olmak<\/li>\n\n\n\n<li>Ya\u015f ve tedavi uygunluk \u015fartlar\u0131n\u0131 kar\u015f\u0131lamak<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fma prosed\u00fcrlerini takip edebilecek bak\u0131c\u0131 olmal\u0131d\u0131r<\/li>\n\n\n\n<li>Bilgilendirilmi\u015f onam verilecek (veya yasal vasinizin vermesini sa\u011flay\u0131n).<\/li>\n\n\n\n<li>Cinsel olarak aktifseniz uygun do\u011fum kontrol y\u00f6ntemleri kullan\u0131lmal\u0131d\u0131r<\/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\">Kimler kat\u0131lamaz?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"key-exclusion-criteria\">Ba\u015fl\u0131ca Hari\u00e7 Tutma Kriterleri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">A\u015fa\u011f\u0131daki durumlarda kat\u0131l\u0131mc\u0131lar programa kat\u0131lamazlar:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS'i g\u00fcvenli bir \u015fekilde alamayacaklar<\/li>\n\n\n\n<li>A\u015fa\u011f\u0131daki gibi enfeksiyonlara sahipseniz:\n<ul class=\"wp-block-list\">\n<li>HIV<\/li>\n\n\n\n<li>Hepatit B veya C<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Yak\u0131n zamanda enfeksiyon ge\u00e7irdiniz veya a\u015f\u0131 oldunuz<\/li>\n\n\n\n<li>G\u00fcvenli\u011fi veya \u00e7al\u0131\u015fma sonu\u00e7lar\u0131n\u0131 etkileyebilecek ko\u015fullara sahip olmak<\/li>\n\n\n\n<li>Sirolimus'u tolere edemeyecek<\/li>\n\n\n\n<li>Ba\u011f\u0131\u015f\u0131kl\u0131k sistemini bask\u0131layan ila\u00e7lar alt\u0131nda iyile\u015fmesi zor olabilecek yara veya incinmelere sahip olmak<\/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\">Bu \u00c7al\u0131\u015fma DMD Toplulu\u011fu \u0130\u00e7in Neden \u00d6nemli?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"real-world-evidence\">Ger\u00e7ek D\u00fcnya Kan\u0131tlar\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u00d6nceki \u00e7al\u0131\u015fmalardan farkl\u0131 olarak, bu \u00e7al\u0131\u015fma ger\u00e7ek hayattaki tedavi ko\u015fullar\u0131n\u0131 yans\u0131t\u0131yor ve doktorlar\u0131n \u015funlar\u0131 anlamalar\u0131na yard\u0131mc\u0131 oluyor:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS kontroll\u00fc ortamlar d\u0131\u015f\u0131nda ne kadar g\u00fcvenli?<\/li>\n\n\n\n<li>Hastalar\u0131n zaman i\u00e7inde nas\u0131l tepki verdi\u011fi<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"improving-treatment-safety\">Tedavi G\u00fcvenli\u011finin \u0130yile\u015ftirilmesi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u015far\u0131l\u0131 olmas\u0131 durumunda, \u00e7al\u0131\u015fma \u015fu sonu\u00e7lara yol a\u00e7abilir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Karaci\u011ferle ilgili risklerin azalmas\u0131<\/li>\n\n\n\n<li>Tedavi protokollerinin iyile\u015ftirilmesi<\/li>\n\n\n\n<li>Gen terapisi kullan\u0131m\u0131na olan g\u00fcvenin artmas\u0131<\/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\">\u0130leti\u015fim Bilgileri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmayla ilgili daha fazla ayr\u0131nt\u0131 i\u00e7in:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Organizasyon:<\/strong> Sarepta Therapeutics<\/li>\n\n\n\n<li><strong>Telefon:<\/strong> 1-888-SAREPTA (1-888-727-3782)<\/li>\n\n\n\n<li><strong>E-posta:<\/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\">Klinik Ara\u015ft\u0131rma Yerleri Hen\u00fcz A\u00e7\u0131klanmad\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENHANCE klinik \u00e7al\u0131\u015fmas\u0131n\u0131n yap\u0131laca\u011f\u0131 yerler hen\u00fcz kamuoyuna a\u00e7\u0131klanmad\u0131. \u00c7al\u0131\u015fman\u0131n sponsoru olan Sarepta Therapeutics&#039;nin, kat\u0131l\u0131mc\u0131 merkezleri daha sonraki bir a\u015famada duyurmas\u0131 bekleniyor. Hastalar\u0131n, hasta yak\u0131nlar\u0131n\u0131n ve sa\u011fl\u0131k \u00e7al\u0131\u015fanlar\u0131n\u0131n, merkez bilgileri yay\u0131nland\u0131k\u00e7a resmi g\u00fcncellemeleri takip etmeleri \u00f6nerilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/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\/tr\/wp-json\/wp\/v2\/posts\/7683","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=7683"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7683\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7684"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7683"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7683"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7683"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}