Trial Profile

A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients with Angelman Syndrome (AS)

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 04 Feb 2025

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At a glance

31 Jan 2025 Status changed from active, no longer recruiting to completed.
19 Dec 2024 According to an Ultragenyx Pharmaceutical media release, company presented data from this study the 2024 Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in November.
09 Nov 2024 According to an Ultragenyx Pharmaceutical media release, company to present Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102 at the 2024 Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in Orlando, Florida.

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