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New hope for rare brain disease: expanded access to experimental drug zilganersen

NCT ID NCT07487389

Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program offers early access to zilganersen for people with Alexander disease, a rare genetic brain disorder. It is for US residents aged 2 and older who have not responded to or cannot tolerate approved treatments. The goal is to provide the drug while it is still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 99 years

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Alexander disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alexander disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

More trials for these conditions

Other studies related to the condition(s) this trial covers.