Phase 2 Study of GTX-102 Treatment for Children and Adults With Different Types of Angelman Syndrome

What this Trial Is About

Researchers are evaluating the safety and effectiveness of GTX-102 in people with Angelman syndrome, a genetic condition, across different ages and genetic types. This Phase 2 open-label basket study includes subprotocols A, B, C, and D, each following a similar design with Screening, Loading, and Maintenance periods. Some subprotocols involve randomization, and participants can continue treatment in a long-term extension after completing the study. Participants receive GTX-102 via intrathecal injections every three months. Dosing starts at a lower level and increases until a target dose is reached. Different subprotocols include groups based on age and Angelman syndrome genotype, with some receiving no treatment initially before starting GTX-102. The study evaluates treatment effects in groups ranging from children as young as 1 year old to adults up to 65 years old. Throughout the study, participants undergo scheduled visits with assessments including cognitive and motor function tests, behavior rating scales, and safety monitoring for adverse events. Researchers collect data up to about 17 months from baseline, with monitoring of treatment-emergent and serious adverse events. Participants also have imaging and lab tests like lumbar punctures and blood work. After the initial study, participants may opt to continue in a long-term extension to further assess GTX-102s effects and safety.

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)