Yazan: David, Do\u011frulayan: Ionis<\/p>\n\n\n\n
Ionis\u2019in yakla\u015fan bir klinik deneme hakk\u0131nda yapt\u0131\u011f\u0131 son duyurunun ard\u0131ndan, bu denemeyi daha net bir \u015fekilde anlamay\u0131 hedefliyoruz. Bu durum, \u00f6zellikle \u00e7ocuklar\u0131n\u0131 klinik \u00e7al\u0131\u015fmalara dahil etmeyi d\u00fc\u015f\u00fcnen aileler i\u00e7in, \u00f6zellikle de MECP2 Duplikasyon Sendromu (MDS) gibi nadir hastal\u0131klar s\u00f6z konusu oldu\u011funda, b\u00fcy\u00fck \u00f6nem ta\u015f\u0131maktad\u0131r.<\/p>\n\n\n\n
Klinik deneme, yeni potansiyel tedavileri ara\u015ft\u0131ran, bunlar\u0131n insanlar \u00fczerindeki g\u00fcvenli\u011fini ve etkilerini de\u011ferlendiren bir ara\u015ft\u0131rma \u00e7al\u0131\u015fmas\u0131d\u0131r.\nKlinik denemeler genellikle daha geni\u015f kapsaml\u0131 bir klinik geli\u015ftirme program\u0131n\u0131n par\u00e7as\u0131 olarak y\u00fcr\u00fct\u00fcl\u00fcr.\nKlinik geli\u015ftirme program\u0131, yeni bir ilac\u0131n veya tedavinin yeterince g\u00fcvenli ve ger\u00e7ekten etkili olup olmad\u0131\u011f\u0131n\u0131 belirlemek amac\u0131yla a\u015famalar halinde (\u00f6rn. Faz 1-3) y\u00fcr\u00fct\u00fclen klinik denemeler dizisidir.<\/p>\n\n\n\n
MDS vakas\u0131nda, Ionis yak\u0131nda ION440 adl\u0131 yeni bir potansiyel tedaviyi de\u011ferlendirmek i\u00e7in ilk klinik \u00e7al\u0131\u015fmay\u0131 ba\u015flatacak. Bu \u00e7al\u0131\u015fma, named ATTUNE, yak\u0131n zamanda Ionis taraf\u0131ndan clinicaltrials.gov'da resmi ba\u015fl\u0131\u011f\u0131 \"MECP2 Duplikasyon Sendromlu Hastalarda \u0130ntratekal Olarak Uygulanan ION440'\u0131n G\u00fcvenli\u011fini, Tolere Edilebilirli\u011fini, Farmakokineti\u011fini ve Farmakodinami\u011fini De\u011ferlendirmek i\u00e7in Faz 1-2, \u00c7ift K\u00f6r, Sham Kontroll\u00fc \u00c7oklu Artan Doz \u00c7al\u0131\u015fmas\u0131\" ile duyuruldu.<\/p>\n\n\n\n
Bunun ne anlama geldi\u011fini a\u00e7\u0131klayal\u0131m.<\/p>\n\n\n\n
Faz 1-2 Klinik \u00c7al\u0131\u015fma:<\/strong> Faz 1, bir ilac\u0131n insanlara ilk kez uyguland\u0131\u011f\u0131 a\u015famad\u0131r (genellikle \u201cinsanda ilk uygulama\u201d \u00e7al\u0131\u015fmalar\u0131 olarak adland\u0131r\u0131l\u0131r).\nFaz 1\u2019in temel amac\u0131, ara\u015ft\u0131rma a\u015famas\u0131ndaki ilac\u0131n farkl\u0131 dozlarda ba\u015flang\u0131\u00e7 g\u00fcvenli\u011fini ve tolere edilebilirli\u011fini belirlemektir; bu \u00e7al\u0131\u015fmalar genellikle k\u00fc\u00e7\u00fck \u00f6l\u00e7ekli ve nispeten k\u0131sa s\u00fcrelidir.\nFaz 2 \u00e7al\u0131\u015fmalar\u0131, ara\u015ft\u0131rma ilac\u0131n\u0131n daha fazla ki\u015fi \u00fczerinde daha uzun bir s\u00fcre incelenmesiyle g\u00fcvenlik hakk\u0131nda ek kan\u0131t sa\u011flar.\n\u00c7o\u011fu Faz 2 \u00e7al\u0131\u015fmas\u0131nda, ATTUNE da d\u00e2hil olmak \u00fczere, ara\u015ft\u0131rmac\u0131lar ayr\u0131ca insanlar\u0131n ara\u015ft\u0131rma ilac\u0131n\u0131n farkl\u0131 dozlar\u0131na nas\u0131l yan\u0131t verdi\u011fini anlamaya \u00e7al\u0131\u015f\u0131rlar.\nATTUNE \u00e7al\u0131\u015fmas\u0131, Faz 1 ve Faz 2 \u00e7al\u0131\u015fmalar\u0131n\u0131n bir kombinasyonudur.<\/p>\n\n\n\n Randomize, \u00c7ift-K\u00f6r, Sham-Kontroll\u00fc:<\/strong> Bu, kat\u0131l\u0131mc\u0131lar\u0131n rastgele olarak ION440 tedavisi veya sham (sahte) tedavi (ION440 enjeksiyonu olmadan, ancak tedavi grubundakiyle ayn\u0131 sedasyon prosed\u00fcr\u00fc) almaya atand\u0131\u011f\u0131 anlam\u0131na gelir. Oran 3:1\u2019dir (tedavi almayan her 1 hasta i\u00e7in 3 hasta ION440 alacakt\u0131r).\nNe kat\u0131l\u0131mc\u0131lar ne de ara\u015ft\u0131rmac\u0131lar kimin ger\u00e7ek tedaviyi, kimin sham tedavisini ald\u0131\u011f\u0131n\u0131 bilmez.<\/p>\n\n\n\n Bir kontrol kolu genellikle (1) hastal\u0131\u011f\u0131n belirti ve semptomlar\u0131 ki\u015fiden ki\u015fiye farkl\u0131l\u0131k g\u00f6sterdi\u011finde (2) ara\u015ft\u0131rmada incelenen belirli bir pop\u00fclasyonda s\u0131n\u0131rl\u0131 do\u011fal ge\u00e7mi\u015f oldu\u011funda (3) klinik ara\u015ft\u0131rman\u0131n hedefleri \u00f6znel veya \u00f6nyarg\u0131ya tabi olarak de\u011ferlendirilebilecek bir \u015fekilde ele al\u0131nabildi\u011finde ve (4) ION440'\u0131 uygulamak i\u00e7in gereken sedasyon ve lomber ponksiyon gibi belirli prosed\u00fcr t\u00fcrlerine kaydolan belirli pop\u00fclasyonun nas\u0131l yan\u0131t verece\u011fi konusunda s\u0131n\u0131rl\u0131 bir anlay\u0131\u015f oldu\u011funda kullan\u0131l\u0131r.<\/p>\n\n\n\n \u00c7oklu Artan Doz (MAD):<\/strong> Bu, ara\u015ft\u0131rmac\u0131lar\u0131n ara\u015ft\u0131rma ilac\u0131n\u0131 farkl\u0131 kat\u0131l\u0131mc\u0131 gruplar\u0131na s\u0131rayla artan dozlarda uygulayarak, g\u00fcvenli bir \u015fekilde ne kadar ila\u00e7 verilebilece\u011fini g\u00f6rmek anlam\u0131na gelir.<\/p>\n\n\n\n \u00c7al\u0131\u015fma B\u00f6l\u00fcm 1 ve B\u00f6l\u00fcm 2:<\/strong> ATTUNE iki b\u00f6l\u00fcme ayr\u0131lm\u0131\u015ft\u0131r. \u00c7al\u0131\u015fmaya kabul edilen herkes B\u00f6l\u00fcm 1\u2019den ba\u015flayacakt\u0131r.\nB\u00f6l\u00fcm 1\u2019de, kat\u0131l\u0131mc\u0131lar\u0131n \u2019i yakla\u015f\u0131k 36 hafta boyunca ION440 alacak, \u2019i ise sham (sahte) tedavi alacakt\u0131r.\nIonis, \u00e7al\u0131\u015fman\u0131n ilk b\u00f6l\u00fcm\u00fcnde g\u00fcvenlik incelemesini ba\u015far\u0131yla tamamlad\u0131ktan sonra, B\u00f6l\u00fcm 1\u2019i bitiren kat\u0131l\u0131mc\u0131lar, a\u00e7\u0131k etiketli uzatma (LTE) olarak bilinen B\u00f6l\u00fcm 2\u2019de 156 haftaya kadar ION440 alma f\u0131rsat\u0131na sahip olacaklard\u0131r.<\/p>\n\n\n\n G\u00fcvenlik, Tolere Edilebilirlik, Farmakokinetik, Farmakodinamik: <\/strong>Bunlar, ION440\u2019\u0131n Faz 1-2 \u00e7al\u0131\u015fmas\u0131n\u0131n hedeflerini tan\u0131mlar. Ama\u00e7, ION440 uygulamas\u0131ndan sonra g\u00f6zlemlenebilecek yan etkiler olup olmad\u0131\u011f\u0131n\u0131, v\u00fccudun ION440\u2019a ne yapt\u0131\u011f\u0131n\u0131, ION440\u2019\u0131n insan v\u00fccudu \u00fczerinde ne yapt\u0131\u011f\u0131n\u0131 de\u011ferlendirmektir.<\/p>\n\n\n\n Dahil Etme ve Hari\u00e7 Tutma Kriterleri:<\/strong> Bunlar, klinik \u00e7al\u0131\u015fmaya kimin kat\u0131labilece\u011fini belirleyen kurallard\u0131r.\n\u00d6rne\u011fin, bu \u00e7al\u0131\u015fmada kat\u0131l\u0131mc\u0131lar\u0131n erkek olmas\u0131, 2 ila 65 ya\u015f aras\u0131nda olmas\u0131, do\u011frulanm\u0131\u015f bir MDS tan\u0131s\u0131na sahip olmas\u0131 ve t\u00fcm \u00e7al\u0131\u015fma prosed\u00fcrlerini tamamlayabilmesi gerekmektedir.\nAyr\u0131ca, birinin kat\u0131lmas\u0131n\u0131 engelleyecek baz\u0131 durumlar da vard\u0131r; \u00f6rne\u011fin MDS\u2019nin karma\u015f\u0131k bir formuna sahip olmak, belirli sa\u011fl\u0131k sorunlar\u0131 veya baz\u0131 di\u011fer ila\u00e7larla tedavi ediliyor olmak.<\/p>\n\n\n\n Klinik \u00e7al\u0131\u015fmalara kat\u0131l\u0131m\u0131n g\u00f6n\u00fcll\u00fc oldu\u011funu ve ailelerin, kat\u0131l\u0131m\u0131n \u00e7ocuklar\u0131 i\u00e7in faydal\u0131 olmad\u0131\u011f\u0131n\u0131 d\u00fc\u015f\u00fcnd\u00fckleri anda istedikleri zaman \u00e7al\u0131\u015fmadan \u00e7ekilebileceklerini unutmamak \u00f6nemlidir.<\/p>\n\n\n\n \u00c7al\u0131\u015fman\u0131n Ba\u015flang\u0131c\u0131:<\/strong> ATTUNE i\u00e7in tahmini ba\u015flang\u0131\u00e7 tarihi A\u011fustos 2024't\u00fcr.\nAncak, bu takvim, etik kurul onay\u0131, s\u00f6zle\u015fme imzalanmas\u0131 ve \u00e7al\u0131\u015fmay\u0131 y\u00fcr\u00fctecek her bir hastanede gerekli di\u011fer sahaya \u00f6zg\u00fc onaylar gibi d\u00fczenleyici ad\u0131mlara ba\u011fl\u0131 olarak de\u011fi\u015febilir.\nHasta al\u0131m\u0131, hem Ionis hem de hastane taraf\u0131ndan t\u00fcm onaylar imzaland\u0131ktan sonra ba\u015flayacakt\u0131r.<\/p>\n\n\n\n L\u00fctfen klinik \u00e7al\u0131\u015fmalar\u0131n t\u0131bbi bilgiyi ilerletmek ve yeni tedaviler bulmak i\u00e7in \u00e7ok \u00f6nemli oldu\u011funu ve ayr\u0131ca nadir hastal\u0131klardan etkilenen bireyler ve aileler i\u00e7in umut sa\u011flad\u0131\u011f\u0131n\u0131 unutmay\u0131n.<\/p>\n\n\n\n ION440 ara\u015ft\u0131rma a\u015famas\u0131nda olan bir ila\u00e7t\u0131r ve MDS d\u00e2hil hi\u00e7bir hastal\u0131\u011f\u0131n tedavisi i\u00e7in hi\u00e7bir \u00fclkede sat\u0131\u015f\u0131 onaylanmam\u0131\u015ft\u0131r.\nSa\u011fl\u0131k de\u011fi\u015fiklikleri ve genel sa\u011fl\u0131k hizmetleri ile ilgili sorular\u0131 olan bireyler, kendi sa\u011fl\u0131k hizmeti sa\u011flay\u0131c\u0131lar\u0131 ile g\u00f6r\u00fc\u015fmelidir.<\/p>\n\n\n\n Eyl\u00fcl 2024\u2019\u00fcn sonunda ba\u015flat\u0131lmas\u0131 beklenen klinik \u00e7al\u0131\u015fmalar ve ATTUNE hakk\u0131ndaki web seminerimiz i\u00e7in bizi takip edin.<\/p>\n\n\n\n