The planned safety and efficacy trial will evaluate, in an initial phase, the safety and preliminary efficacy of LYS-GM101, delivered via a one-time injection directly into the CNS (intra-cisternal administration into the cerebrospinal fluid). The second stage of this adaptive study will evaluate the efficacy of LYS-GM101 in a larger group of patients with GM1 gangliosidosis. Patients will be treated at sites in the US and Europe.
Primary outcomes will be measured via physical and neurological examination; quality of life will also be tracked as secondary outcome. A video sub-study will collect video outcomes of patients in their home environment. This innovative, app-based approach is designed to provide further supportive evidence of the treatment effects on children’s development in a convenient and minimally invasive fashion. Video outcomes for this study will be compared to those of a Natural History Video Study initiated in Q1 2020.