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: Recruiting

Phase of Trial: Phase III

Latest Information Update: 24 Jun 2025

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At a glance

Drugs GTX 102-GeneTx Biotherapeutics (Primary)
Indications Angelman syndrome
Focus Registrational; Therapeutic Use
Acronyms Aspire
Sponsors Ultragenyx Pharmaceutical

Most Recent Events

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.
12 Jan 2025 According to an Ultragenyx media release, enrollment in the study is expected to complete in the second half of 2025.
19 Dec 2024 Status changed from not yet recruiting to recruiting, According to an Ultragenyx media release,

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