Zilganersen Expanded Access Program for Individuals With Alexander Disease

NCT07487389 | Unknown

• 1 other identifier: ION373-E01
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).

Conditions

Alexander Disease

Keywords

Alexander disease; leukodystrophy; expanded access

Interventions

zilganersen DRUG

zilganersen

Eligibility Criteria

• Age: 2 Years - 99 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Patients who are ≥ 2 years old.
• Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD.
• Patients who have a documented variant in the GFAP gene.
• Patient resides in and is a resident of the US.

You may not qualify if:

• Patients who have any medical history, physical exam findings, or clinically significant laboratory abnormalities that contraindicate performing an LP for ITB administration of zilganersen.
• Patients who have current obstructive hydrocephalus.
• Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of CSF.
• Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding.

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Related Links

• Related Info

MeSH Terms

Conditions

Alexander Disease

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Leukoencephalopathies; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Ionis Pharmaceuticals, Inc.

CONTACT

1-833-644-6647 (833-MI-IONIS); MedInfo@ionisph.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 17, 2026
• First Posted: March 23, 2026
• Last Updated: March 23, 2026

Record last verified: 2026-03