Acadia Trofinetide Wajiga III Natiijooyinka Khadka Sare ee Kiliinikada ee Wanaagsan

2021-12-06, waqtiga Mareykanka, Acadia Pharmaceuticals (Nasdaq: ACAD) waxay ku dhawaaqday natiijooyinka safka sare ee togan ee tijaabada caafimaad ee Wajiga III ee musharaxeeda daroogada, Trofinetide.Tijaabada wajiga III, ee loo yaqaan Lavender, ayaa inta badan loo isticmaalaa si loo qiimeeyo badbaadada iyo waxtarka Trofinetide ee daaweynta Rett syndrome (RS).Wadarta 189 maado ayaa la diiwaan galiyay, dhammaan dumarka da'doodu u dhaxayso 5-20 sano waxay qabaan RS.

Lavender wuxuu ahaa laba-indho-indho la'aan, randomized, tijaabada placebo-koontarool ee toddobaadyada 12 oo leh dhammaadka asaasiga ah ee RS Habdhaqanka Su'aalaha (RSBQ) iyo Natiijooyinka Qiimaynta Caalamiga ah ee Qiimaynta (CGI-I), sida ay qiimeeyeen shaqaalaha kalkaalinta iyo dhakhaatiirta, siday u kala horreeyaan;furaha dhamaadka labaad waa Isgaadhsiinta iyo Heerka Horumarinta Habdhaqanka Astaanta ee Dhallaanka iyo Saanqaadayaasha (CSBS-DP-IT-Social), kaas oo inta badan loo isticmaalo in lagu qiimeeyo horumarinta dhaqanka bulsheed, hadal, iyo calaamad muujinaysa dhallaanka iyo carruurta yaryar 6-24 bilaha da'da, waxaana loo isticmaali karaa cudurka autism Baaritaanka hore ee calaamadaha dib u dhaca korriinka iyo dib u dhaca kale ee korriinka, sida ay qiimeeyeen shaqaalaha kalkaalinta.

Natiijooyinku waxay muujiyeen in Trofinetide ay horumar weyn ka samaysay labada dhibcood ee aasaasiga ah marka loo eego placebo.Isbeddellada salka RSBQ ee placebo iyo Trofinetide ee usbuuca 12 waxay ahaayeen -1.7 vs -5.1 (p=0.0175);Dhibcaha CGI-I waxay ahaayeen 3.8 vs 3.5 (p=0.0030).Dhanka kale, isbeddelka ka yimid saldhigga CSBS-DP-IT-Social wuxuu ahaa -1.1 iyo -0.1 ee placebo iyo Trofinetide, siday u kala horreeyaan.

Labada qaybood ee aasaasiga ah iyo kuwa hoose ee Lavender labaduba waxay muujiyeen suurtogalnimada Trofinetide ee daaweynta RS, si kastaba ha ahaatee, waa in la ogaadaa in heerka joojinta daaweynta daraasadda ee la xidhiidha dhacdooyinka xun ee degdega ah (TEAEs) uu ka sarreeyay cududda Trofinetide marka la barbardhigo placebo. , Labadu waxay ahaayeen 2.1% iyo 17.2%, siday u kala horreeyaan.Waxaa ka mid ahaa, dhacdooyinka xun xun ee ugu badan:

Shuban - Trofinetide wuxuu ahaa 80.6% (97.3% kuwaas oo ahaa mid fudud ilaa dhexdhexaad) iyo placebo waxay ahayd 19.1%;

② Matagga - Trofinetide wuxuu ahaa 26.9% (96% kuwaas oo ahaa mid fudud ilaa dhexdhexaad) iyo placebo wuxuu ahaa 9.6%;

③ Dhacdooyin halis ah ayaa ka dhacay 3.2% maadooyinka labada kooxood.

Mawduucyada tijaabada Lavender waxay sii wadi doonaan helitaanka Trofinetide ka dib marka la dhammeeyo tijaabada ama calaamadda furan ee Lilac iyo Lilac-2 daraasaadka ballaarinta, iyo> 95% maadooyinka dhameeyay daraasadda Lavender waxay doorteen inay u beddelaan calaamadda furan ee Lilac Baadhitaanka balaadhinta, natiijooyinka waxaa loo soo bandhigi doonaa kulan caafimaad oo soo socda.