Zilganersen Expanded Access Program for Individuals With Alexander Disease

March 17, 2026 updated by: Ionis Pharmaceuticals, Inc.
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

This is an expanded access program (EAP) for eligible individuals diagnosed with Alexander disease residing in the United States, prior to approval of zilganersen by the local regulatory agency (FDA).

A medical doctor must decide whether the potential benefits of zilganersen outweigh the potential risks based on the individual patient's medical history and program eligibility criteria.

Access can be requested via the contact information below.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Key Inclusion Criteria:

  1. Approved drug or drugs available for treatment did not work for the patient, or the patient cannot tolerate the side effects of the FDA-approved drug or drugs for treatment of AxD.
  2. Patients who are ≥ 2 years old.
  3. Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD.
  4. Patients who have a documented variant in the GFAP gene.
  5. Patient resides in and is a resident of the US.

Key Exclusion Criteria:

  1. Patients who have any medical history, physical exam findings, or clinically significant laboratory abnormalities that contraindicate performing an LP for ITB administration of zilganersen.
  2. Patients who have current obstructive hydrocephalus.
  3. Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of CSF.
  4. Patients who have any other medical history or current conditions, which, in their Treating Physician's opinion, would make the patient unsuitable for inclusion (e.g., active Hepatitis B virus or hepatitis C virus infection, severe hepatic or renal disease, uncontrolled acute or chronic condition, etc.).
  5. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding.

Other inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ionis Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 17, 2026

First Submitted That Met QC Criteria

March 17, 2026

First Posted (Actual)

March 23, 2026

Study Record Updates

Last Update Posted (Actual)

March 23, 2026

Last Update Submitted That Met QC Criteria

March 17, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ION373-E01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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