Alexander DiseaseNCT07487389
Zilganersen Expanded Access Program for Individuals With Alexander Disease
A study testing zilganersen in people with Alexander Disease, run by Ionis Pharmaceuticals, Inc..
Trial summary
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD). Read more about this study
- Who is it for?
- Ages 2 to 99 years
- Conditions the study is for
- Alexander Disease
- What is being tested?
- zilganersen
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 99 years
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
- Other inclusion/exclusion criteria may apply.
About this study
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
Read the 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.
- Conditions
- Alexander Disease
- Being tested
- zilganersen
- Keywords
- Alexander disease, leukodystrophy, expanded access
- Medical subject headings
- Alexander Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zilganersenDrug
zilganersen
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alexander DiseaseStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07487389
- Sponsor's protocol ID
- ION373-E01
- Status last verified
- Sep 2026
- First submitted
- 17 Mar 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Zilganersen Expanded Access Program for Individuals With Alexander Disease
- ClinicalTrials.gov ID
- NCT07487389
- Acronym / sponsor ID
- ION373-E01
- Conditions
- Alexander Disease
- Keywords
- Alexander disease, leukodystrophy, expanded access
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 23 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
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 design
Who may qualify
- Ages
- 2 to 99 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| zilganersenzilganersen | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing zilganersen in people with Alexander Disease, run by Ionis Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.