Phase 12 Study to Evaluate Safety and Effectiveness of MVX-220 Gene Therapy Given by Injection for Children and Adults with Angelman Syndrome

What this Trial Is About

Researchers are evaluating the safety and effectiveness of MVX-220 gene therapy in children and adults with Angelman syndrome who have specific genetic types including UBE3A gene deletion, uniparental disomy, or imprinting center defects. This phase 12 study aims to understand how well this gene replacement therapy works and its safety profile in these participants. The trial is sponsored by MavriX Bio, LLC and involves a single group of participants without a control group. Participants will receive a single dose of MVX-220 administered by injection into the cisterna magna. The study includes two primary groups adults aged 18 to 50 years and children aged 4 to 8 years. There is also an optional third group that may include adults and children aged 4 to 50 years. All participants will take steroids before and briefly during the study to help reduce immune responses related to the gene therapy. Safety data from adults will be reviewed before enrolling children. Participants will be monitored closely for safety and effectiveness for two years after treatment, with assessments including physical exams, laboratory tests, ECGs, and questionnaires measuring communication, development, behavior, sleep, and quality of life. After the initial period, monitoring will continue less frequently for an additional three years, totaling five years of follow-up. Researchers will also measure viral DNA levels and EEG parameters to assess treatment impact and safety throughout the study.

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome