Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Summary

The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).

Timeline

Start

2024-12-03

Primary completion

2026-07

Completion

2027-11

Outcome

Missed primary endpoint

release “results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint” ultragenyx.com ↗

Drugs

Indications