VGL101 for Leukoencephalopathy

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

20 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What makes the drug VGL101 unique for treating leukoencephalopathy?

There is no specific information available about VGL101 in the provided research articles, so its unique aspects compared to other treatments for leukoencephalopathy are not detailed. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Phenotypic variation in leukoencephalopathy with vanishing white matter. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

Leukoencephalopathy with brainstem and spinal cord involvement and high brain lactate: report of three Brazilian patients. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Haploinsufficiency of CSF-1R and clinicopathologic characterization in patients with HDLS. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

MRI characteristics and scoring in HDLS due to CSF1R gene mutations. [2021]

5. Germany pubmed.ncbi.nlm.nih.gov

Early-onset LBSL: how severe does it get? [2012]