What side effects are associated with this type of intervention?

The most common treatments for ALS include riluzole, edaravone, and investigational agents like masitinib and reldesemtiv. Riluzole is generally well-tolerated but can cause nausea, weakness, and liver function abnormalities. Edaravone may lead to bruising, gait disturbances, and allergic reactions. Masitinib, a tyrosine kinase inhibitor, has been associated with gastrointestinal issues, rash, and edema. Reldesemtiv, a skeletal muscle activator, can cause fatigue, nausea, and headache. These treatments aim to slow disease progression but come with varying side effect profiles that should be discussed with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Amyotrophic Lateral Sclerosis (ALS), including riluzole and edaravone. Riluzole is believed to reduce damage to motor neurons by decreasing the release of glutamate, while edaravone is thought to act as a free radical scavenger, reducing oxidative stress. These treatments do not specifically target gene mutations like VGL101, which is being studied for its effects on the CSF1R gene mutation in ALSP. Currently, there are no FDA-approved ALS treatments that specifically target gene mutations or the CSF1R pathway.

What other types of research exist?

Promising novel alternatives to VGL101 for ALS patients include edaravone, which has shown efficacy in slowing functional decline in early-stage ALS, and PRE-084, a Sigma-1 receptor agonist with neuroprotective effects demonstrated in mouse models of motor neuron disease.

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Virus Therapy

VGL101 for Leukoencephalopathy

Phase 2

Waitlist Available

Research Sponsored by Vigil Neuroscience, Inc.

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up weeks 52, 76, 100, 124, and 148

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing iluzanebart, a medication given through infusions, in adults with a specific genetic mutation causing ALSP, a rare brain disease. The goal is to see if the medication is safe and can slow down the disease by looking at brain scans and other health markers. Participants will receive the treatment regularly for a year, with an option to continue for additional years.

Who is the study for?

This trial is for adults with a specific brain condition called ALSP, confirmed by MRI and genetic testing showing a CSF1R gene mutation. Participants must have someone to report on their health status. Those with other neurological conditions that could confuse study results or pose significant risks are excluded.

What is being tested?

The study tests VGL101's safety and its effect on disease progression in ALSP patients. It involves regular infusions of the drug every four weeks over one year, monitoring through imaging and biomarkers.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at the infusion site, changes in blood tests or vital signs, and any organ-specific issues due to the new medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 52, 76, 100, 124, and 148

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~weeks 52, 76, 100, 124, and 148

This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 52, 76, 100, 124, and 148 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Core Study Adverse Events

Long-Term Extension Adverse Events

Secondary study objectives

To evaluate the effects of iluzanebart on biomarkers of disease progression in participants with ALSP in the Core Study

To evaluate the effects of iluzanebart on biomarkers of target engagement in participants with ALSP in the Core Study

To evaluate the effects of iluzanebart on the ALSP on biomarkers of disease progression and clinical outcomes in patients with ALSP in the Core Study

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VGL101Experimental Treatment1 Intervention

Solution administered via Intravenous Infusion (IV)

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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

The most common treatments for ALS include riluzole, edaravone, and sodium phenylbutyrate-taurursodiol (PB-TURSO). Riluzole works by inhibiting glutamate release, thereby reducing excitotoxicity, which is believed to contribute to motor neuron death. Edaravone acts as an antioxidant, reducing oxidative stress and slowing functional decline. PB-TURSO combines two drugs that reduce neuronal cell death through different mechanisms, including mitochondrial and endoplasmic reticulum stress reduction. These treatments are crucial for ALS patients as they target various pathways involved in disease progression, offering a multifaceted approach to slowing the decline in motor function. Similar to VGL101, which targets the CSF1R gene mutation pathology in ALSP, these treatments aim to modify disease progression rather than just alleviating symptoms.