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

NCT04849741 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2026-04-03

No results posted yet for this study

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.

Conditions

  • Alexander Disease

Interventions

DRUG

zilganersen

zilganersen will be administered by ITB injection.

DRUG

Placebo

zilganersen-matching placebo will be administered by ITB injection.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-01
Primary Completion
2025-08-22
Completion
2029-09-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Israel
  • Italy
  • Japan
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04849741 on ClinicalTrials.gov