Clinical Trial

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

Active, Not Recruiting Phase 3
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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.
Protocol Amendment History 34 amendments
This ClinicalTrials.gov record has been amended 34 times since 2021-04-16; most recent amendment 2026-03-30.
Status change: Enrolling by Invitation → Active, Not Recruiting 2025-07-11
Status change: Recruiting → Enrolling by Invitation 2024-08-01
Status change: Not Yet Recruiting → Recruiting 2021-04-20
Trial Details
NCT Number NCT04849741
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Alexander Disease
Enrollment 54 participants
Start Date 2021-06-01
Primary Completion 2025-08-22 (estimated)
Study Completion 2029-09 (estimated)
Updated on ClinicalTrials.gov 2026-04-03