Acadia Trofinetide Mataki na III na Babban Sakamako na Babban Layi Na Asibiti

A kan 2021-12-06, lokacin Amurka, Acadia Pharmaceuticals (Nasdaq: ACAD) ta ba da sanarwar ingantattun sakamakon layin farko na gwajin asibiti na Mataki na III na ɗan takararta na magani, Trofinetide.Gwajin lokaci na III, wanda ake kira Lavender, ana amfani dashi musamman don kimanta aminci da ingancin Trofinetide a cikin maganin ciwon Rett (RS).An shigar da jimillar batutuwa 189, duk mata masu shekaru 5-20 suna da RS.

Lavender ya kasance makafi sau biyu, bazuwar, gwajin gwajin wuribo na makonni 12 tare da haɗakar mahimman abubuwan ƙarshe na RS Behavioral Questionnaire (RSBQ) da Clinical Outcome Global Rating Scale (CGI-I), kamar yadda ma'aikatan jinya da likitoci suka tantance, bi da bi;maɓalli Ƙarshen ƙarshe na biyu shine Ƙimar Sadarwar Sadarwa da Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararrun Ƙwararru (CSBS-DP-IT-Social), wanda aka fi amfani da shi don tantance ci gaban zamantakewa, magana, da dabi'u na alama a jarirai da ƙananan yara 6-24 watanni masu shekaru, kuma za a iya amfani da shi don gwajin farko na autism don alamun jinkirin ci gaba da sauran jinkirin ci gaba, kamar yadda ma'aikatan jinya suka tantance.

Sakamakon ya nuna cewa Trofinetide ya sami ci gaba mai mahimmanci a cikin duka abubuwan ƙarshe na farko idan aka kwatanta da placebo.Canje-canje daga asali a cikin RSBQ don placebo da Trofinetide a mako na 12 sun kasance -1.7 vs -5.1 (p=0.0175);Makin CGI-I sun kasance 3.8 vs 3.5 (p=0.0030).A halin yanzu, canjin daga asali a cikin CSBS-DP-IT-Social shine -1.1 da -0.1 don placebo da Trofinetide, bi da bi.

Dukansu na farko da na farko na Lavender sun nuna yiwuwar Trofinetide don maganin RS, duk da haka, ya kamata a lura cewa yawan dakatarwar maganin nazarin da ke da alaka da abubuwan da ba su da kyau (TEAEs) ya kasance mafi girma a cikin Trofinetide hannu idan aka kwatanta da placebo. , Biyu sun kasance 2.1% da 17.2%, bi da bi.Daga cikin su, abubuwan da suka fi yawan faruwa sun hada da:

① Diarrhea - Trofinetide ya kasance 80.6% (97.3% daga cikinsu sun kasance masu sauƙi zuwa matsakaici) kuma placebo shine 19.1%;

② Amai - Trofinetide ya kasance 26.9% (96% daga cikinsu sun kasance masu sauƙi zuwa matsakaici) kuma placebo shine 9.6%;

③ Mummunan abubuwan da suka faru a cikin 3.2% na batutuwa a cikin ƙungiyoyin biyu.

Abubuwan da ke cikin gwajin Lavender za su ci gaba da karɓar Trofinetide bayan kammala gwajin ko kuma a cikin binciken fadada Lilac da Lilac-2, kuma> 95% na batutuwan da suka kammala nazarin Lavender sun zaɓi canzawa zuwa alamar bude Lilac. fadada Bincike, za a gabatar da binciken ga taron likita mai zuwa.