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Also known as ION373
zilganersen · 1 trial · 1 indication
10-Meter Walk Test (10MWT) is an assessment of gait speed.
| Arm | Type | Description |
|---|---|---|
| zilganersen | EXPERIMENTAL | Zilganersen will be administered by intrathecal bolus (ITB) injection once every 12 weeks through Week 49. The 60-week double-blind treatment period will be followed by the open-label, long-term and extended long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 469. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo will be administered by ITB injection once every 12 weeks through Week 49. It will be followed by the open-label, long-term and extended long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 469. |
| Name | Type | Description |
|---|---|---|
| zilganersen | DRUG | zilganersen will be administered by ITB injection. |
| Placebo | DRUG | zilganersen-matching placebo will be administered by ITB injection. |
Key Inclusion Criteria: 1. Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease 2. Documented genetic mutation in the GFAP gene 3. Aged ≥ 2 to 65 years old at the time of informed consent 4. Able and willing to meet all study requirements, including travel to Study ...
Zilganersen is an investigational RNA therapy being developed for the treatment of Alexander Disease, a rare neurological disorder. It is not yet approved for any indication and remains in clinical development. The Phase 3 program is evaluating it in patients with Alexander Disease, including pediatric patients as young as 2 years old.
Zilganersen targets GFAP, or glial fibrillary acidic protein. It is an antisense oligonucleotide, part of the -sen chemical class, designed to reduce GFAP expression. GFAP is the protein that accumulates in Alexander Disease, so targeting it addresses the underlying cause of the condition rather than only its symptoms.
Zilganersen is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the clinical program studying the drug in Alexander Disease.
Zilganersen is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The FDA has granted it Priority Review, Fast Track, and Breakthrough Therapy designations for Alexander Disease.
Zilganersen is being studied in one active Phase 3 trial, NCT04849741, titled A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease. The trial is randomized, double-blind, and placebo-controlled, with an enrollment of 54 patients across the United States, Australia, Canada, Israel, Italy, Japan, the Netherlands, and the United Kingdom.
Yes, zilganersen is also known as ION373. Both names refer to the same investigational antisense oligonucleotide developed by Ionis Pharmaceuticals for Alexander Disease. The Phase 3 trial NCT04849741 lists the drug under both names.