Ka 2021-12-06, nakong ea US, Acadia Pharmaceuticals (Nasdaq: ACAD) e phatlalalitse liphetho tse ntle tsa maemo a holimo tsa teko ea eona ea bongaka ea Phase III ea mokhethoa oa eona oa lithethefatsi, Trofinetide.Teko ea phase III, e bitsoang Lavender, e sebelisoa haholo ho lekola polokeho le katleho ea Trofinetide kalafong ea Rett syndrome (RS).Kakaretso ea lithuto tse 189 li ile tsa ngolisoa, basali bohle ba lilemo tse 5-20 ba nang le RS.
Lavender e ne e le teko e laoloang ka makhetlo a mabeli, e sa tloaelehang, e laoloang ke placebo ea libeke tsa 12 e nang le lintlha tse ka sehloohong tse kopantsoeng tsa RS Behavioral Questionnaire (RSBQ) le Clinical Outcome Global Rating Scale (CGI-I), joalokaha e hlahlojoa ke basebeletsi ba baoki le lingaka, ka ho latellana;senotlolo Qetellong ea bobeli ke Sekala sa Ntlafatso ea Boitšoaro ba Puisano le Matšoao bakeng sa Masea le Bana (CSBS-DP-IT-Social), e sebelisoang haholo-holo ho lekola tsoelo-pele ea boitšoaro ba sechaba, mantsoe, le tšoantšetso ho masea le bana ba banyenyane 6–24 likhoeli tsa lilemo, 'me e ka sebelisoa bakeng sa autism Ho hlahloba pele ho nako bakeng sa matšoao a ho lieha ho nts'etsopele le ho lieha ho hong ha nts'etsopele, joalo ka ha ho hlahlojoa ke basebetsi ba baoki.
Liphetho li bonts'itse hore Trofinetide e bile le lintlafatso tse kholo ho lipheletsong tsa mantlha ha li bapisoa le placebo.Liphetoho ho tloha motheong oa RSBQ bakeng sa placebo le Trofinetide Bekeng ea 12 e ne e le -1.7 vs -5.1 (p=0.0175);Lintlha tsa CGI-I e ne e le 3.8 vs 3.5 (p=0.0030).Ho sa le joalo, phetoho ho tloha motheong oa CSBS-DP-IT-Social e ne e le -1.1 le -0.1 bakeng sa placebo le Trofinetide, ka ho latellana.
Liphello tsa mantlha le tsa mantlha tsa Lavender li bonts'itse bokhoni ba Trofinetide bakeng sa kalafo ea RS, leha ho le joalo, ho lokela ho hlokomeloa hore sekhahla sa ho khaotsa kalafo ea thuto e amanang le liketsahalo tse mpe tse hlahang (TEAEs) se ne se le holimo ho letsoho la Trofinetide ha se bapisoa le placebo. , Ka bobeli e ne e le 2.1% le 17.2%, ka ho latellana.Har'a tsona, liketsahalo tse mpe tse atileng haholo e ne e le:
① Letšollo - Trofinetide e ne e le 80.6% (97.3% ea eona e neng e le bonolo ho isa boemong bo itekanetseng) le placebo e ne e le 19.1%;
② Ho hlatsa - Trofinetide e ne e le 26.9% (96% ea eona e ne e le bonolo ho isa boemong bo itekanetseng) le placebo e ne e le 9.6%;
③ Liketsahalo tse mpe tse tebileng li etsahetse ho 3.2% ea lithuto lihlopheng ka bobeli.
Barupeluoa tekong ea Lavender ba tla tsoelapele ho fumana Trofinetide ka mor'a ho phethoa ha teko kapa lithutong tse bulehileng tsa Lilac le Lilac-2, 'me >95% ea barupeluoa ba qetileng thuto ea Lavender ba khethile ho fetohela ho Lilac open-label. katoloso Lipatlisiso, liphuputso li tla hlahisoa sebokeng se tlang sa bongaka.
Nako ea poso: Feb-17-2022