{"id":6211,"date":"2026-02-17T15:37:49","date_gmt":"2026-02-17T12:37:49","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6211"},"modified":"2026-02-17T15:39:39","modified_gmt":"2026-02-17T12:39:39","slug":"data-monitoring-committee-recommended-initiating-an-increased-dose-for-group-2-in-entrada-therapeutics-exon-44-skipping-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/data-monitoring-committee-recommended-initiating-an-increased-dose-for-group-2-in-entrada-therapeutics-exon-44-skipping-study\/","title":{"rendered":"Veri \u0130zleme Komitesi, Entrada'n\u0131n Ekzon 44 Atlama \u00c7al\u0131\u015fmas\u0131nda Grup 2 \u0130\u00e7in Art\u0131r\u0131lm\u0131\u015f Doz Ba\u015flat\u0131lmas\u0131n\u0131 Tavsiye Etti"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Entrada Therapeutics, \u00e7al\u0131\u015fma protokol\u00fcne g\u00f6re, ba\u011f\u0131ms\u0131z bir Veri \u0130zleme Komitesi&#039;nin (DMC), ELEVATE-44-201&#039;nin \u00e7ift k\u00f6r, plasebo kontroll\u00fc, \u00e7oklu artan doz (MAD) b\u00f6l\u00fcm\u00fcn\u00fcn 1. Kohortunda dozlamay\u0131 tamamlayan sekiz hastadan elde edilen t\u00fcm mevcut g\u00fcvenlik ve PK verilerini inceledi\u011fini duyurdu.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Doz art\u0131r\u0131m\u0131 \u00f6nerisi olumlu sonu\u00e7lar\u0131n bir g\u00f6stergesidir.<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cEntrada Therapeutics Ara\u015ft\u0131rma ve Geli\u015ftirme Ba\u015fkan\u0131 Dr. Natarajan Sethuraman, \u201dELEVATE-44-201 \u00e7al\u0131\u015fmam\u0131z\u0131n 1. Kohort verilerini inceledikten sonra, ba\u011f\u0131ms\u0131z Veri \u0130zleme Komitesinin 2. Kohort dozlamas\u0131na 12 mg\/kg&#039;l\u0131k art\u0131r\u0131lm\u0131\u015f dozda ba\u015flanmas\u0131n\u0131 desteklemesinden memnuniyet duyuyoruz\u201d dedi. <strong>\u201cEntrada\u2019n\u0131n n\u00f6rom\u00fcsk\u00fcler programlar\u0131n\u0131n tamam\u0131 ayn\u0131 Endozomal Ka\u00e7\u0131\u015f Arac\u0131n\u0131 kulland\u0131\u011f\u0131 i\u00e7in, bu \u00e7al\u0131\u015fma ilerlemesinin klinik programlar\u0131m\u0131zdaki riskleri \u00f6nemli \u00f6l\u00e7\u00fcde azaltt\u0131\u011f\u0131na ve \u015firketimiz i\u00e7in \u00f6nemli bir klinik d\u00f6n\u00fcm noktas\u0131 olu\u015fturdu\u011funa inan\u0131yoruz.<\/strong> ENTR-601-44'\u00fcn 6 mg\/kg dozunda g\u00fcvenli oldu\u011funun kan\u0131tlanmas\u0131, program i\u00e7in a\u00e7\u0131k bir d\u00f6n\u00fcm noktas\u0131d\u0131r. \u0130kinci \u00e7eyrekte verileri a\u00e7\u0131klad\u0131\u011f\u0131m\u0131zda, 6 mg\/kg dozundaki 1. Kohort verilerimizin \u00e7ift haneli distrofin \u00fcretimi g\u00f6sterece\u011fini bekliyoruz. Dozu 12 mg\/kg'a y\u00fckseltti\u011fimizde, bu y\u0131l\u0131n sonlar\u0131nda a\u00e7\u0131klayaca\u011f\u0131m\u0131z 2. Kohort verileriyle s\u0131n\u0131f\u0131n\u0131n en iyisi distrofin restorasyonuna ula\u015faca\u011f\u0131m\u0131za inan\u0131yoruz.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVATE-44-201, ekzon 44 atlamas\u0131na uygun olan ayakta tedavi g\u00f6ren DMD hastalar\u0131nda ENTR-601-44'\u00fcn g\u00fcvenli\u011fini, tolerans\u0131n\u0131 ve etkinli\u011fini de\u011ferlendiren, k\u00fcresel, iki b\u00f6l\u00fcml\u00fc, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir Faz 1\/2 \u00e7al\u0131\u015fmas\u0131d\u0131r. <strong>\u015eirket, 1. Kohort i\u00e7in \u00e7al\u0131\u015fman\u0131n MAD b\u00f6l\u00fcm\u00fcn\u00fcn dozlamas\u0131n\u0131 tamamlad\u0131 ve kat\u0131l\u0131mc\u0131lar, \u00e7al\u0131\u015fman\u0131n 6 dozluk a\u00e7\u0131k etiketli, Faz 2 b\u00f6l\u00fcm\u00fcne ge\u00e7i\u015f yapt\u0131. \u015eirket, 1. Kohorttan (6 mg\/kg) elde edilen verileri 2026 y\u0131l\u0131n\u0131n ikinci \u00e7eyre\u011finde, 2. Kohorttan (12 mg\/kg) elde edilen verileri y\u0131l sonuna kadar ve 3. Kohorttan (18 mg\/kg&#039;a kadar) elde edilen verileri ise daha sonra raporlamay\u0131 planl\u0131yor. <\/strong>\u015eirket ayr\u0131ca, ELEVATE-44-201 \u00e7al\u0131\u015fmas\u0131nda tedavi edilen kat\u0131l\u0131mc\u0131 say\u0131s\u0131n\u0131 art\u0131rmak amac\u0131yla y\u0131l\u0131n ilerleyen d\u00f6nemlerinde bir geni\u015fletme kohortu a\u00e7may\u0131 planlamaktad\u0131r; zira bu \u00e7al\u0131\u015fma, ABD&#039;de h\u0131zland\u0131r\u0131lm\u0131\u015f bir onay s\u00fcrecini desteklemek \u00fczere tasarlanm\u0131\u015ft\u0131r. Aral\u0131k 2025&#039;te ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA), ENTR-601-44&#039;ye Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 vermi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/upcoming-exon-44-skipping-therapies-for-dmd\/\">Duchenne Tedavisinde Yakla\u015fan Ekzon 44 Atlama Terapileri<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/h2>\n\n\n\n<figure class=\"wp-block-image size-large\"><img 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=\"(max-width: 968px) 100vw, 968px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>\u00a0>>>\u00a0<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>Entrada Therapeutics announced that an independent Data Monitoring Committee (DMC), per study protocol, has reviewed all available safety and PK data from the eight patients who completed dosing in Cohort 1 of the double-blinded, placebo-controlled, multiple ascending dose (MAD) portion of ELEVATE-44-201. Dose increase recommendation is an indicator of positive results \u201cWe are pleased that [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6213,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[233,234,141,127,246],"class_list":["post-6211","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-entrada","tag-entrada-therapeutics","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies"],"subtitle":"Ba\u011f\u0131ms\u0131z Veri \u0130zleme Komitesi, 1. Kohorttaki 6 mg\/kg dozundan 12 mg\/kg dozuna y\u00fckseltilerek 2. Kohortun ba\u015flat\u0131lmas\u0131n\u0131 \u00f6nermi\u015ftir. ELEVATE-44-201, Duchenne kas distrofisi (DMD) geninde ekzon 44 atlamas\u0131na uygun do\u011frulanm\u0131\u015f bir mutasyona sahip hastalarda DMD'nin potansiyel tedavisi i\u00e7in ENTR-601-44'nin Faz 1\/2 MAD klinik \u00e7al\u0131\u015fmas\u0131d\u0131r.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6211","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=6211"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6211\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6213"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6211"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6211"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6211"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}