{"id":7976,"date":"2026-05-14T20:35:20","date_gmt":"2026-05-14T17:35:20","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7976"},"modified":"2026-06-24T22:01:14","modified_gmt":"2026-06-24T19:01:14","slug":"avidity-biosciences-aoc-1044-safari44-phase-3-dmd-trial-nct07587242","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/avidity-biosciences-aoc-1044-safari44-phase-3-dmd-trial-nct07587242\/","title":{"rendered":"Del-Zota SAFARI44 Faz 3 \u00c7al\u0131\u015fmas\u0131: Duchenne Kas Distrofisi \u0130\u00e7in Yeni Bir Ekzon 44 Atlama \u00c7al\u0131\u015fmas\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne kas distrofisi (DMD) ara\u015ft\u0131rmalar\u0131, delpacibart zotadirsen olarak da bilinen AOC 1044'\u00fc de\u011ferlendiren Del-Zota SAFARI44 Faz 3 klinik \u00e7al\u0131\u015fmas\u0131n\u0131n ba\u015flat\u0131lmas\u0131yla ilerlemeye devam ediyor. Bu deneysel tedavi, ekzon 44 atlamas\u0131na uygun DMD mutasyonlar\u0131na sahip bireyler i\u00e7in geli\u015ftirilmektedir.<\/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-the-safari-44-trial\">SAFARI44 Klinik Denemesi Nedir?<\/a><\/li><li><a href=\"#what-is-aoc-1044-delpacibart-zotadirsen\">Del-Zota (Delpacibart Zotadirsen) nedir?<\/a><\/li><li><a href=\"#how-the-study-will-work\">\u00c7al\u0131\u015fma Nas\u0131l \u0130\u015fleyecek?<\/a><ul><li><a href=\"#randomized-treatment-period\">Rastgele Se\u00e7ilmi\u015f Tedavi D\u00f6nemi<\/a><\/li><li><a href=\"#open-label-extension-ole\">A\u00e7\u0131k Etiket Uzant\u0131s\u0131 (OLE)<\/a><\/li><li><a href=\"#independent-safety-monitoring\">Ba\u011f\u0131ms\u0131z G\u00fcvenlik \u0130zleme<\/a><\/li><\/ul><\/li><li><a href=\"#safari-44-timeline\">SAFARI44 Zaman \u00c7izelgesi<\/a><ul><li><a href=\"#estimated-study-dates\">Tahmini \u00c7al\u0131\u015fma Tarihleri<\/a><\/li><li><a href=\"#planned-enrollment\">Planl\u0131 Kay\u0131t<\/a><\/li><\/ul><\/li><li><a href=\"#who-can-participate\">Kimler Kat\u0131labilir?<\/a><ul><li><a href=\"#key-inclusion-criteria\">Temel Dahil Edilme Kriterleri<\/a><\/li><li><a href=\"#key-exclusion-criteria\">Ba\u015fl\u0131ca Hari\u00e7 Tutma Kriterleri<\/a><\/li><\/ul><\/li><li><a href=\"#why-this-trial-matters-for-the-dmd-community\">Bu Klinik Deneme DMD Toplulu\u011fu \u0130\u00e7in Neden \u00d6nemli?<\/a><\/li><li><a href=\"#contact-information\">\u0130leti\u015fim Bilgileri<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-is-the-safari-44-trial\" class=\"wp-block-heading\">SAFARI44 Klinik Denemesi Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">SAFARI44 \u00e7al\u0131\u015fmas\u0131, Duchenne kas distrofisi olan ve ekzon 44 atlama tedavisine uygun erkek \u00e7ocuklarda AOC 1044'\u00fcn (Del-Zota) g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir Faz 3 klinik \u00e7al\u0131\u015fmas\u0131d\u0131r. Daha fazla bilgi i\u00e7in:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">Del-zota (EXPLORE44), Normal Distrofin \u00dcretiminin Yakla\u015f\u0131k 30%&#039;sini Sa\u011flar<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma, intraven\u00f6z AOC 1044&#039;\u00fcn, belirli distrofin geni mutasyonlar\u0131n\u0131 ta\u015f\u0131yan DMD hastalar\u0131nda hastal\u0131k sonu\u00e7lar\u0131n\u0131 iyile\u015ftirmeye yard\u0131mc\u0131 olup olamayaca\u011f\u0131n\u0131 ara\u015ft\u0131racakt\u0131r. Tedavi, alt\u0131 haftada bir intraven\u00f6z inf\u00fczyon yoluyla uygulan\u0131r.<\/p>\n\n\n\n<h2 id=\"what-is-aoc-1044-delpacibart-zotadirsen\" class=\"wp-block-heading\">Del-Zota (Delpacibart Zotadirsen) nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Del-Zota, ekzon 44 atlamas\u0131ndan fayda sa\u011flayabilecek distrofin genindeki mutasyonlar\u0131 hedeflemek i\u00e7in geli\u015ftirilen, deneysel bir ekzon atlama terapisidir. Ekzon atlama terapilerinin amac\u0131, v\u00fccudun daha k\u0131sa ancak i\u015flevsel bir distrofin proteini \u00fcretmesine yard\u0131mc\u0131 olmakt\u0131r; bu da Duchenne kas distrofisinde kas dejenerasyonunu yava\u015flatabilir. Daha Fazla Bilgi Edinin:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"968\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR.jpg\" alt=\"Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar\" class=\"wp-image-5275\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR.jpg 1890w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-284x300.jpg 284w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-968x1024.jpg 968w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-768x813.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-1452x1536.jpg 1452w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-11x12.jpg 11w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-397x420.jpg 397w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-150x159.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-300x317.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-696x737.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-1068x1130.jpg 1068w\" sizes=\"auto, (max-width: 968px) 100vw, 968px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Geleneksel ekzon atlama ila\u00e7lar\u0131ndan farkl\u0131 olarak, Del-Zota, tedavinin kas dokusuna daha iyi iletilmesini sa\u011flamak \u00fczere tasarlanm\u0131\u015f antikor oligon\u00fckleotid konjugat teknolojisini kullan\u0131r.<\/p>\n\n\n\n<h2 id=\"how-the-study-will-work\" class=\"wp-block-heading\">\u00c7al\u0131\u015fma Nas\u0131l \u0130\u015fleyecek?<\/h2>\n\n\n\n<h3 id=\"randomized-treatment-period\" class=\"wp-block-heading\">Rastgele Se\u00e7ilmi\u015f Tedavi D\u00f6nemi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">SAFARI44 \u00e7al\u0131\u015fmas\u0131na kat\u0131lan kat\u0131l\u0131mc\u0131lar a\u015fa\u011f\u0131dakilerden birini alacaklard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Del-Zota (delpacibart zotadirsen)<\/li>\n\n\n\n<li>Plasebo<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n ilk 54 haftas\u0131 boyunca toplam dokuz doz olmak \u00fczere, tedaviler alt\u0131 haftada bir uygulanacakt\u0131r.<\/p>\n\n\n\n<h3 id=\"open-label-extension-ole\" class=\"wp-block-heading\">A\u00e7\u0131k Etiket Uzant\u0131s\u0131 (OLE)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Rastgele atama a\u015famas\u0131 tamamland\u0131ktan sonra, t\u00fcm kat\u0131l\u0131mc\u0131lar 54 haftal\u0131k A\u00e7\u0131k Etiketli Uzatma a\u015famas\u0131na girebilirler. Bu d\u00f6nemde, her kat\u0131l\u0131mc\u0131 orijinal tedavi atamas\u0131na bak\u0131lmaks\u0131z\u0131n Del-Zota alacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Son tedavi dozunun 102. haftada uygulanmas\u0131 bekleniyor, ard\u0131ndan 114. haftaya kadar ek de\u011ferlendirmeler ve g\u00fcvenlik takibi yap\u0131lacak.<\/p>\n\n\n\n<h3 id=\"independent-safety-monitoring\" class=\"wp-block-heading\">Ba\u011f\u0131ms\u0131z G\u00fcvenlik \u0130zleme<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u011f\u0131ms\u0131z bir Veri \u0130zleme Komitesi (IDMC), \u00e7al\u0131\u015fma boyunca g\u00fcvenlik, tolere edilebilirlik ve etkinlik verilerini d\u00fczenli olarak inceleyecektir.<\/p>\n\n\n\n<h2 id=\"safari-44-timeline\" class=\"wp-block-heading\">SAFARI44 Zaman \u00c7izelgesi<\/h2>\n\n\n\n<h3 id=\"estimated-study-dates\" class=\"wp-block-heading\">Tahmini \u00c7al\u0131\u015fma Tarihleri<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c7al\u0131\u015fman\u0131n Ba\u015flang\u0131\u00e7 Tarihi: Haziran 2026<\/li>\n\n\n\n<li>Birincil Tamamlama: May\u0131s 2029<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fman\u0131n Tamamlanma Tarihi: Temmuz 2030<\/li>\n<\/ul>\n\n\n\n<h3 id=\"planned-enrollment\" class=\"wp-block-heading\">Planl\u0131 Kay\u0131t<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar bu 3. a\u015fama m\u00fcdahale \u00e7al\u0131\u015fmas\u0131na yakla\u015f\u0131k 70 kat\u0131l\u0131mc\u0131 kaydetmeyi planl\u0131yor.<\/p>\n\n\n\n<h2 id=\"who-can-participate\" class=\"wp-block-heading\">Kimler Kat\u0131labilir?<\/h2>\n\n\n\n<h3 id=\"key-inclusion-criteria\" class=\"wp-block-heading\">Temel Dahil Edilme Kriterleri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">SAFARI44 \u00e7al\u0131\u015fmas\u0131na kat\u0131lmaya hak kazanmak i\u00e7in kat\u0131l\u0131mc\u0131lar\u0131n a\u015fa\u011f\u0131daki \u015fartlar\u0131 sa\u011flamas\u0131 gerekmektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Duchenne kas distrofisi tan\u0131s\u0131 konmu\u015f, y\u00fcr\u00fcyebilen erkekler<\/li>\n\n\n\n<li>Ekzon 44 atlamas\u0131na uygun, do\u011frulanm\u0131\u015f bir distrofin geni mutasyonuna sahip olmak<\/li>\n\n\n\n<li>2 ya\u015f ve \u00fczeri olmal\u0131d\u0131r (\u00c7ocuk, Yeti\u015fkin, Ya\u015fl\u0131 Yeti\u015fkin)<\/li>\n\n\n\n<li>Tarama s\u0131ras\u0131nda gerekli fonksiyonel de\u011ferlendirmeleri tamamlamak<\/li>\n\n\n\n<li>Tedaviye ba\u015flamadan \u00f6nce en az alt\u0131 ay boyunca istikrarl\u0131 bir kortikosteroid rejimine devam etmek<\/li>\n<\/ul>\n\n\n\n<h3 id=\"key-exclusion-criteria\" class=\"wp-block-heading\">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 yar\u0131\u015fmaya kat\u0131lmaya hak kazanamayabilir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Daha \u00f6nce gen terapisi veya h\u00fccre terapisi alm\u0131\u015f olmak<\/li>\n\n\n\n<li>Kay\u0131ttan \u00f6nceki alt\u0131 ay i\u00e7inde ba\u015fka bir oligon\u00fckleotid tedavisi kullanmak<\/li>\n\n\n\n<li>Laboratuvar de\u011ferleri protokol s\u0131n\u0131rlar\u0131n\u0131n d\u0131\u015f\u0131nda ise<\/li>\n\n\n\n<li>Son zamanlarda b\u00fcy\u00fcme hormonu, testosteron veya givinostat gibi tedavilerde de\u011fi\u015fiklikler ya\u015fanmas\u0131<\/li>\n<\/ul>\n\n\n\n<h2 id=\"why-this-trial-matters-for-the-dmd-community\" class=\"wp-block-heading\">Bu Klinik Deneme DMD Toplulu\u011fu \u0130\u00e7in Neden \u00d6nemli?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ekzon atlama terapileri, Duchenne kas distrofisi ara\u015ft\u0131rmalar\u0131nda en \u00f6nemli tedavi yakla\u015f\u0131mlar\u0131ndan biri olmaya devam etmektedir. Di\u011fer ekzon hedefleri i\u00e7in halihaz\u0131rda bir\u00e7ok ekzon atlama ilac\u0131 mevcut olsa da, ekzon 44 atlamas\u0131na uygun mutasyonlar\u0131 olan hastalar i\u00e7in se\u00e7enekler hala s\u0131n\u0131rl\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SAFARI44 deneyi, AOC 1044&#039;\u00fcn bu hasta pop\u00fclasyonunda distrofin restorasyonunu ve klinik sonu\u00e7lar\u0131 g\u00fcvenli bir \u015fekilde iyile\u015ftirip iyile\u015ftiremeyece\u011fi konusunda \u00f6nemli bilgiler sa\u011flayabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/upcoming-exon-44-skipping-therapies-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Tedavisinde Yakla\u015fan Ekzon 44 Atlama Terapileri<\/a><\/p>\n\n\n\n<h2 id=\"contact-information\" class=\"wp-block-heading\">\u0130leti\u015fim Bilgileri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma Avidity Biosciences taraf\u0131ndan desteklenmektedir.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Telefon: 858-771-7038<\/li>\n\n\n\n<li>E-posta: <a>medinfo@aviditybio.com<\/a><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eu anda herhangi bir \u00e7al\u0131\u015fma yeri kamuoyuna a\u00e7\u0131klanmam\u0131\u015ft\u0131r.<\/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>Duchenne muscular dystrophy (DMD) research continues to move forward with the launch of the Del-Zota SAFARI44 Phase 3 clinical trial evaluating AOC 1044, also known as delpacibart zotadirsen. This investigational therapy is being developed for individuals with DMD mutations amenable to exon 44 skipping. What Is the SAFARI44 Trial? The SAFARI44 study is a randomized, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7979,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[459,126,137,298,397,141,127,246,77,178,779],"class_list":["post-7976","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-aoc-1044","tag-avidity-biosciences","tag-clinical-trial","tag-del-zota","tag-delpacibart-zotadirsen","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-exon-skipping","tag-exon-skipping-therapies","tag-safari44"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7976","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=7976"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7976\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7979"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7976"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7976"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7976"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}