{"id":5302,"date":"2025-12-09T17:14:24","date_gmt":"2025-12-09T14:14:24","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5302"},"modified":"2025-12-09T17:25:38","modified_gmt":"2025-12-09T14:25:38","slug":"satellos-receives-fda-and-international-clearance-to-commence-pediatric-phase-2-testing-of-sat-3247-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/satellos-receives-fda-and-international-clearance-to-commence-pediatric-phase-2-testing-of-sat-3247-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Satellos, Duchenne Musk\u00fcler Distrofisi i\u00e7in SAT-3247&#039;nin Pediatrik Faz 2 Testine Ba\u015flamak \u0130\u00e7in FDA ve Uluslararas\u0131 Onay Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Satellos, ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nden (FDA) ve di\u011fer uluslararas\u0131 d\u00fczenleyicilerden, \u00fc\u00e7 ayl\u0131k, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir Faz 2 kavram kan\u0131tlama \u00e7al\u0131\u015fmas\u0131 olan SAT-3247-CL-201&#039;i ba\u015flatmak i\u00e7in Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 (IND) izni ald\u0131\u011f\u0131n\u0131 bildirdi. \u00c7al\u0131\u015fma, Duchenne kas distrofisi (Duchenne veya DMD) te\u015fhisi konmu\u015f 51 ayakta tedavi g\u00f6ren \u00e7ocukta SAT-3247&#039;yi de\u011ferlendirecek. &gt;&gt; <a href=\"https:\/\/dmdwarrior.com\/tr\/potential-of-sat-3247-to-restore-muscle-regeneration-in-duchenne-published-in-nature\/\" target=\"_blank\" rel=\"noreferrer noopener\">SAT-3247&#039;nin Duchenne Hastal\u0131\u011f\u0131nda Kas Yenilenmesini Geri Kazand\u0131rma Potansiyeli Nature Dergisinde Yay\u0131mland\u0131<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SAT-3247&#039;nin Klinik Denemesi<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Plasebo kontroll\u00fc Faz 2 \u00e7al\u0131\u015fmas\u0131, Duchenne hastal\u0131\u011f\u0131 olan ayakta tedavi g\u00f6ren \u00e7ocuklarda \u00fc\u00e7 ay boyunca SAT-3247 tedavisini de\u011ferlendirecektir<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fman\u0131n son noktalar\u0131 aras\u0131nda g\u00fcvenlik ve tolere edilebilirlik, kas kuvveti ve i\u015flevi \u00fczerindeki etki ve kas kalitesi ve rejenerasyonu \u00fczerindeki etki yer almaktad\u0131r<\/li>\n\n\n\n<li>\u0130lk \u00e7al\u0131\u015fma kat\u0131l\u0131mc\u0131s\u0131n\u0131n 2025 y\u0131l\u0131 sonuna kadar kaydedilmesi bekleniyor<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cSatellos&#039;nin kurucu orta\u011f\u0131 ve CEO&#039;su Frank Gleeson, &quot;BASECAMP ad\u0131n\u0131 verdi\u011fimiz SAT-3247&#039;nin 2. Faz pediatrik \u00e7al\u0131\u015fmam\u0131za ba\u015flamak i\u00e7in ABD ve k\u00fcresel onay ald\u0131\u011f\u0131m\u0131z i\u00e7in \u00e7ok mutluyuz. Bu \u00f6nemli d\u00f6n\u00fcm noktas\u0131, Satellos&#039;yi, Duchenne hastal\u0131\u011f\u0131yla ya\u015fayan \u00e7ocuklarda v\u00fccudun kas onar\u0131m ve yenileme yetene\u011fini g\u00fcvenli bir \u015fekilde geri kazand\u0131rma ve hastal\u0131\u011f\u0131n ilerlemesini durdurma potansiyelini g\u00f6sterme konumuna getiriyor,&quot; dedi. <strong>\u201cYeti\u015fkinlerde y\u00fcr\u00fctt\u00fc\u011f\u00fcm\u00fcz Faz 1b \u00e7al\u0131\u015fmam\u0131zdan elde etti\u011fimiz cesaret verici i\u015flevsel verilerle, SAT-3247&#039;nin Duchenne&#039;li \u00e7ocuklarda etki yaratma potansiyeline g\u00fcveniyoruz ve yak\u0131nda \u00e7al\u0131\u015fmaya kay\u0131t yapt\u0131rmaya ba\u015flamaktan heyecan duyuyoruz.\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA iznine ek olarak, Birle\u015fik Krall\u0131k \u0130la\u00e7 ve Sa\u011fl\u0131k \u00dcr\u00fcnleri D\u00fczenleme Ajans\u0131 (MHRA), \u015firketin Klinik Ara\u015ft\u0131rma Ba\u015fvurusu&#039;na (CTA) yetki verdi; Avustralya \u0130nsan Ara\u015ft\u0131rmalar\u0131 Etik Komitesi (HREC), Terap\u00f6tik \u00dcr\u00fcnler \u0130daresi&#039;nin (TGA) Klinik Ara\u015ft\u0131rma Bildirimi (CTN) \u015femas\u0131n\u0131 d\u00fczenleyici yetkilendirme i\u00e7in kabul etti; ve S\u0131rbistan \u0130la\u00e7 ve T\u0131bbi Cihazlar Ajans\u0131 (ALIMS) \u015firketin CTA&#039;s\u0131n\u0131 onaylad\u0131. <strong>Klinik ara\u015ft\u0131rma ba\u015fvurusu, belirlenen zaman \u00e7izelgeleri ve prosed\u00fcrler do\u011frultusunda Avrupa Birli\u011fi ve Kanada&#039;da halen incelenmektedir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SAT-3247, Duchenne&#039;de bozulan bir s\u00fcre\u00e7 olan kaslar\u0131n yenilenme yetene\u011fini v\u00fccudun yeniden canland\u0131rmas\u0131 i\u00e7in tasarlanm\u0131\u015f, oral, k\u00fc\u00e7\u00fck molek\u00fcll\u00fc bir tedavidir.<\/strong> Yeti\u015fkinlerde yap\u0131lan bir Faz 1a\/b \u00e7al\u0131\u015fmas\u0131nda, SAT-3247 g\u00fcvenli ve iyi tolere edilmi\u015f olup, istenen farmakokinetik (FK) profile sahiptir. Duchenne&#039;li yeti\u015fkinlerin 28 g\u00fcnl\u00fck bir s\u00fcre boyunca SAT-3247 ile tedavi edildi\u011fi \u00e7al\u0131\u015fman\u0131n B B\u00f6l\u00fcm\u00fc, bask\u0131n elde maksimum kavrama g\u00fcc\u00fcnde ortalama 118,6% ve bask\u0131n olmayan elde ortalama 97,9% iyile\u015fme g\u00f6stermi\u015ftir; bu da kavrama g\u00fcc\u00fcnde yakla\u015f\u0131k iki kat\u0131na \u00e7\u0131karak yakla\u015f\u0131k 2 kg&#039;dan yakla\u015f\u0131k 4 kg&#039;a ula\u015fm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>BASECAMP, Duchenne hastal\u0131\u011f\u0131 olan 51 ayakta tedavi g\u00f6ren \u00e7ocukta y\u00fcr\u00fct\u00fclen \u00fc\u00e7 ayl\u0131k k\u00fcresel, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, kavram kan\u0131tlama \u00e7al\u0131\u015fmas\u0131d\u0131r. Birincil sonlan\u0131m noktalar\u0131, SAT-3247&#039;nin g\u00fcvenli\u011fini ve tolere edilebilirli\u011fini ve kas kuvveti \u00fczerindeki etkisini de\u011ferlendirecektir.<\/strong> \u0130kincil sonlan\u0131m noktalar\u0131, SAT-3247&#039;nin kas kalitesi, i\u015flevi ve rejenerasyonu \u00fczerindeki etkisini de\u011ferlendirecektir. \u0130lk kat\u0131l\u0131mc\u0131n\u0131n 2025 y\u0131l\u0131 sonuna kadar \u00e7al\u0131\u015fmaya dahil edilmesi beklenmektedir. Satellos, BASECAMP&#039;tan ilk ara verileri 2026&#039;n\u0131n 2. \u00e7eyre\u011finde, DMD&#039;li yeti\u015fkinlerde LT-001 \u00e7al\u0131\u015fmas\u0131ndan ise 2026&#039;n\u0131n 1. \u00e7eyre\u011finde bildirmeyi beklemektedir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SAT-3247 Hakk\u0131nda<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">SAT-3247, Duchenne kas distrofisi ve di\u011fer dejeneratif veya yaralanma durumlar\u0131nda kaybedilen iskelet kas\u0131n\u0131 yenilemek i\u00e7in yeni bir tedavi olarak Satellos taraf\u0131ndan geli\u015ftirilen, patentli, oral, k\u00fc\u00e7\u00fck molek\u00fcll\u00fc bir ila\u00e7t\u0131r. Satellos, distrofin ve ekzon mutasyon durumundan ba\u011f\u0131ms\u0131z olarak, DMD i\u00e7in potansiyel bir tedavi olarak SAT-3247&#039;yi ilerletmektedir.<\/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>Satellos reported that it has obtained Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) and additional international regulators to initiate SAT-3247-CL-201\u2014a three-month, randomized, double-blind, placebo-controlled Phase 2 proof-of-concept trial. The study will evaluate SAT-3247 in 51 ambulatory children diagnosed with Duchenne muscular dystrophy (Duchenne or DMD). >> Potential of SAT-3247 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5304,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,254,251,252,253,259],"class_list":["post-5302","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-frank-gleeson","tag-sat-3247","tag-satellos","tag-satellos-bioscience","tag-stem-cell-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5302","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=5302"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5302\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5304"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5302"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5302"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5302"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}