What side effects are associated with this type of intervention?

Antisense oligonucleotide therapies, such as Zilganersen (ION373) being studied for Alexander Disease, commonly have side effects that may include injection site reactions, flu-like symptoms, and elevated liver enzymes. Other potential side effects can include thrombocytopenia (low platelet count), renal toxicity, and immune reactions. These side effects vary depending on the specific antisense oligonucleotide and the condition being treated.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of Alexander Disease in the provided context. Zilganersen (ION373) is an antisense oligonucleotide being studied for its efficacy in improving or stabilizing gross motor function in patients with Alexander Disease. Antisense oligonucleotides are a class of drugs designed to target and regulate gene expression, and they have been explored in various genetic disorders, but specific FDA-approved treatments for Alexander Disease are not detailed in the provided information.

What other types of research exist?

Currently, Zilganersen (ION373) is a primary investigational treatment for Alexander Disease. For novel alternatives, patients should consult with their healthcare provider about ongoing clinical trials or emerging therapies in the field of neurogenetics and rare neurological disorders. This may include other antisense oligonucleotides, gene therapies, or symptomatic treatments being researched.

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Small Molecule

Zilganersen for Alexander Disease

Phase 3

Waitlist Available

Research Sponsored by Ionis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline and week 61

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new drug called zilganersen to see if it can help people with Alexander Disease improve or keep their ability to move. The study involves giving the drug in different doses to find out its effects on movement abilities.

Who is the study for?

This trial is for people aged 2 to 65 with Alexander Disease (AxD), confirmed by specific brain imaging and a genetic mutation in the GFAP gene. Children under 18 need a caregiver to participate. Participants must be able to travel for study requirements but can't join if they've had recent major surgery, other experimental brain treatments, or are on another clinical trial.

What is being tested?

The study tests zilganersen (ION373) against a placebo to see if it improves or stabilizes gross motor function in AxD patients. It's designed to compare the effects of this potential new treatment with no active treatment over time.

What are the potential side effects?

While specific side effects of ION373 aren't listed here, common ones from similar treatments may include reactions at injection sites, flu-like symptoms, headache, fatigue, and potential liver toxicity. Each person's experience could vary.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline and week 61

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline and week 61

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and week 61 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percent Change from Baseline in the 10-Meter Walk Test (10MWT)

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: zilganersenExperimental Treatment1 Intervention

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 and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229.

Group II: PlaceboPlacebo Group1 Intervention

Matching placebo will be administered by ITB injection once every 12 weeks through Week 49. It will be followed by the open-label and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Antisense oligonucleotides (ASOs) are short, synthetic strands of nucleic acids designed to bind to specific mRNA sequences, thereby modulating gene expression. In the context of Alexander Disease, ASOs like zilganersen (ION373) target the mRNA of genes implicated in the disease, aiming to reduce the production of abnormal proteins that contribute to the pathology. This mechanism is crucial for Alexander Disease patients as it offers a targeted approach to potentially stabilize or improve gross motor function by directly addressing the underlying genetic cause of the disease.

Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study.