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).

Age range2 Years – 99 Years

SexALL

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

✓. 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.

✕. 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.

NCT IDNCT07487389

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

Ionis Pharmaceuticals, Inc.