Скачать с ютуб Initiation of the Astroscape Clinical Trial for TSC and FCD в хорошем качестве

Initiation of the Astroscape Clinical Trial for TSC and FCD

Bruce Leuchter, MD, CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the initiation of the Astroscape clinical trial for tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II. TSC is characterized by the growth of benign tumors throughout the body, including in the heart, brain, and kidneys. Certain symptoms develop before birth, such as heart tumors, while others become more obvious in childhood, such as developmental delay, seizures, and skin changes. Lung and kidney tumors are more likely to develop in adulthood. TSC is caused by dysfunctioning TSC1 or TSC2 genes. FCD type II is a cerebral developmental malformation that results in a clinical phenotype of intractable epilepsy, usually requiring surgery. FCD has been classified histologically into 2 subtypes: a type without balloon cells, known as type IIA, and a type with balloon cells, known as type IIB. Affected individuals have refractory seizures, usually with onset in early childhood, and may have persistent intellectual disability. Most patients require neurosurgical resection of affected brain tissue to ameliorate seizure frequency and severity. The Astroscape clinical trial is an open-label study evaluating the dosing, safety, tolerability, and pharmacokinetics of radiprodil in children and adolescents with TSC or FCD type II who experience treatment-resistant seizures. The medication’s effect on the frequency and severity of epileptic seizures and other symptoms related to behavior, sleep, and quality of life will also be analyzed. Radiprodil is an investigational, selective and potent negative allosteric modulator of the NR2B. The therapy has previously been investigated in a phase 1b open-label trial for GRIN-related neurodevelopmental disorder where it was well tolerated. Additionally, a median reduction of 86% in seizure frequency versus baseline was observed. These results are a driving force behind the Astroscape clinical trial for TSC and FCD type II.