Trial Profile

A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 09 Sep 2026

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At a glance

03 Sep 2026 According to an Ultragenyx media release, Primary endpoint (Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338) has not been met.
03 Sep 2026 Results presented in the Ultragenyx Pharmaceutical Media Release
04 Aug 2026 According to an Ultragenyx media release, key updates for the second half of 2026; PDUFA dates and a pivotal data readout from the Phase 3 Aspire study of GTX-102 in Angelman syndrome in September or October timeframe.

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