Protocadherin-19 (PCDH19)-clustering epilepsy is a distinct developmental and epileptic encephalopathy characterized by early-onset seizures that are often treatment refractory. Caused by a mutation of the PCDH19 gene on the X chromosome, this rare epilepsy syndrome primarily affects females with seizure onset commonly in the first year of life. A global, randomized, double-blind, placebo-controlled, phase 2 trial was conducted to evaluate the efficacy, safety, and tolerability of ganaxolone compared with placebo as adjunctive therapy to a standard antiseizure medication regimen in patients with PCDH19-clustering epilepsy
Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy / Joseph Sullivan , Boudewijn Gunning , Muhammad Zafar , Renzo Guerrini , Jozef Gecz , Kristy L Kolc , Yufan Zhao , Maciej Gasior 7 Alex A Aimetti , Debopam Samanta. - In: EPILEPSY RESEARCH. - ISSN 0920-1211. - ELETTRONICO. - 191:(2023), pp. 0-0. [10.1016/j.eplepsyres.2023.107112]
Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy
Renzo Guerrini;
2023
Abstract
Protocadherin-19 (PCDH19)-clustering epilepsy is a distinct developmental and epileptic encephalopathy characterized by early-onset seizures that are often treatment refractory. Caused by a mutation of the PCDH19 gene on the X chromosome, this rare epilepsy syndrome primarily affects females with seizure onset commonly in the first year of life. A global, randomized, double-blind, placebo-controlled, phase 2 trial was conducted to evaluate the efficacy, safety, and tolerability of ganaxolone compared with placebo as adjunctive therapy to a standard antiseizure medication regimen in patients with PCDH19-clustering epilepsy
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