A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

Summary

The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.

Timeline

Start

2021-06-01

Primary completion

2025-08-22

Completion

2029-09

Outcome

Met primary endpoint

release “The study met its primary endpoint in individuals ≥5 years of age, with zilganersen 50 mg demonstrating statistically significant and clinically meaningful stabilization” ionis.com ↗

release “The study met its primary endpoint in individuals ≥ 5 years of age, with zilganersen 50 mg demonstrating statistically significant and clinically meaningful stabilization” ionis.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	zilganersen	Unknown	—	Intrathecal

Indications