VGL101 for Leukoencephalopathy

No longer recruiting at 10 trial locations

Overseen By Vigil Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vigil Neuroscience, Inc.

Disqualifiers: Alzheimer's, Parkinson's, Stroke, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 39

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VGL101, an experimental drug, for a rare brain condition known as adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). The goal is to determine the safety of VGL101 and its effect on disease progression by examining brain scans and other health markers. Participants will receive an infusion of VGL101 every four weeks for a year, with an option to continue for up to two more years. The trial seeks individuals with a specific gene mutation who exhibit symptoms such as memory problems, movement issues, or seizures, confirmed by specific MRI findings. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that VGL101 is likely to be safe for humans?

Research has shown that VGL101, also known as iluzanebart, has promising safety results from earlier studies. An initial safety review found VGL101 to be generally safe and well-tolerated by participants, with no major safety concerns reported. The treatment uses an antibody to enhance certain cell functions, which might aid in addressing the condition under study. Although still in the testing phase, early findings suggest it could be safe for humans. Always consult a healthcare provider for information specific to your health needs. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for leukoencephalopathy, which typically focus on managing symptoms, VGL101 offers a unique approach by being administered through intravenous infusion, potentially allowing for more direct and efficient delivery to the brain. Researchers are excited about VGL101 because it may target the underlying causes of the disease, rather than just alleviating symptoms. This novel mechanism of action could lead to more effective treatment outcomes and improved quality of life for patients.

What evidence suggests that VGL101 might be an effective treatment for leukoencephalopathy?

Research shows that VGL101 aims to improve the function of important brain cells called microglia by activating a protein known as TREM2. This may help address issues caused by changes in the CSF1R gene, linked to a rare brain condition called adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). However, early results from the IGNITE study indicated that VGL101 did not clearly work for this condition. Although it hasn't shown clear benefits yet, the mechanism of VGL101 still provides a basis for further research to explore its potential. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have a documented CSF1R gene mutation.

Participants must have a study partner willing to provide accurate information about the participant's health and cognitive abilities

I have symptoms and MRI results that match ALSP.

Exclusion Criteria

I have a neurological condition that might be similar to or increase the risk of ALSP.

Participants with conditions that may place them at significant risk or interfere with study participation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Core Study

Participants receive infusions of iluzanebart approximately every 4 weeks for 1 year to assess safety, tolerability, and effects on imaging and biomarkers

52 weeks

13 visits (in-person)

Long-Term Extension

Participants who complete the Core Study may opt to continue treatment for up to an additional 2 years

Up to 104 weeks

26 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VGL101

Trial Overview 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.

How Is the Trial Designed?

Group I: VGL101 Experimental Treatment 1 Intervention

Solution administered via Intravenous Infusion (IV)

Who Is Running the Clinical Trial?

Vigil Neuroscience, Inc.

Lead Sponsor

Trials

3

Recruited

200+

Citations

1. ctv.veeva.com ctv.veeva.com/study/a-study-of-vgl101-in-patients-with-adult-onset-leukoencephalopathy-with-axonal-spheroids-and-pigment

A Study of VGL101 in Patients With Adult-Onset ... This is a multicenter, open-label study to assess the safety and tolerability of iluzanebart (also referred to as VGL101) in subjects with ...

2. firstwordpharma.com firstwordpharma.com/story/5968650

Sanofi-bound Vigil says iluzanebart flops in rare neuro ... Vigil Neuroscience said Wednesday that its lead drug candidate iluzanebart (VGL101) failed to show efficacy in the Phase II IGNITE trial for a rare ...

3. neurology.org neurology.org/doi/10.1212/WNL.0000000000205296

Interim Results on VGL101 from IGNITE IGNITE is a phase 2, proof-of-concept, multicenter, open-label study evaluating safety, tolerability, and clinical effects of VGL101 on MRI and disease ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05677659

NCT05677659 | A Study of VGL101 in Patients With Adult- ... This is a multicenter, open-label study to assess the safety and tolerability of iluzanebart (also referred to as VGL101) in subjects with documentation of ...

5. neurology.org neurology.org/doi/10.1212/WNL.0000000000204565

VGL101: An Immunotherapy that Enhances Microglial ... VGL101 is a potent and selective TREM2 agonistic antibody, align with the hypothesis that TREM2 activation can compensate for CSF1R dysfunction.

6. vigilneuro.com vigilneuro.com/s/AAN-2023-Andreas-Presentation.pdf

blind, Placebo-controlled, Single and Multiple Ascending ... VGL101 Phase 1 Preliminary Safety Data. ▫ In blinded preliminary safety review of completed cohorts, VGL101 was generally safe and well ...

7. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-vgl101-for-adults-with-adult-onset-leukoencephalopathy/

Study on the Safety and Tolerability of VGL101 for Adults ... This study investigates the safety and tolerability of VGL101, administered via intravenous infusion, for adults diagnosed with the rare brain disease, ...