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: 21 Aug 2025

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At a glance

06 Aug 2025 Status changed from recruiting to active, no longer recruiting.
31 Jul 2025 According to an Ultragenyx media release, company expects to complete Phase 3 Aspire study in the second half of 2026 Ultragenyx plans to move with urgency to provide topline data and progress to regulatory submission.
13 Feb 2025 According to an Ultragenyx media release, enrollment in the Phase 3 Aspire study is expected to complete in the second half of 2025.

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