AVAILABLENot Applicable

Zilganersen Expanded Access Program for Individuals With Alexander Disease

Plain-language summary

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

Who can participate

Age range

2 Years – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Exclusion criteria

. 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 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.).
. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

Trial details

NCT IDNCT07487389

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

Contact for this trial

Ionis Pharmaceuticals, Inc.

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

View on ClinicalTrials.gov More Alexander Disease trials