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A Study of VGL101 in Patients with Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia

Phase 2
Active, not recruiting
Conditions
ALSP
Interventions
Drug: VGL101
Registration Number
NCT05677659
Lead Sponsor
Vigil Neuroscience, Inc.
Brief Summary

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 a gene mutation in the CSF1R gene for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) and to evaluate the effects of iluzanebart on imaging and biomarkers of disease progression in subjects with ALSP. Participants will receive infusions of iluzanebart approximately every 4 weeks for 1 year. The study includes a 52-week, open-label Core Study, followed by a Long-Term Extension (LTE), which provides subjects who complete the original 52-week study (Core Study) with the option to continue treatment for up to an additional 2 years.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
20
Inclusion Criteria
  • Participants who have documentation of a gene mutation in the CSF1R gene

  • Participants fulfill both (Parts A and B) of the following criteria:

    1. The participant has more than 2 findings of clinical signs or symptoms in the following categories:

      1. Cognitive impairment or psychiatric problem
      2. Pyramidal signs on neurological examination
      3. Extrapyramidal signs, such as rigidity.
      4. Epilepsy
    2. MRI findings consistent with ALSP, specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum, on the Screening MRI.

  • The participant must have a study partner (i.e., caregiver, family member, friend, etc.) who, in the investigator's judgment, has frequent and sufficient contact with the subject so as to be able to provide accurate information about the participant's health and cognitive and functional abilities. The study partner must be willing to sign a study partner ICF.

Key

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Exclusion Criteria
  • The participant has any neurological disease that poses a risk to the participant or can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but not limited to, brain tumor, hydrocephalus, Alzheimer's disease, frontotemporal dementia (FTD), ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's disease, and Down syndrome.
  • Participant with any condition or situation that, in the opinion of the investigator or sponsor medical personnel, may place the subject at significant risk, confound the study results, or interfere significantly with the participant's participation in the study.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
VGL101VGL101Solution administered via Intravenous Infusion (IV)
Primary Outcome Measures
NameTimeMethod
Core Study Adverse EventsWeek 24 and Week 52

To evaluate the safety and tolerability of iluzanebart for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) by adverse events in the Core Study

Long-Term Extension Adverse EventsWeeks 52, 76, 100, 124, and 148

To evaluate the safety and tolerability of iluzanebart for the treatment of ALSP by adverse events in the Long-Term Extension

Secondary Outcome Measures
NameTimeMethod
To evaluate the effects of iluzanebart on biomarkers of disease progression in participants with ALSP in the Core StudyWeek 24 and Week 52

Change from Baseline to Week 24 and Week 52 in neurofilament light chain (NfL) in cerebrospinal fluid (CSF) and blood

To evaluate the effects of iluzanebart on the ALSP on biomarkers of disease progression and clinical outcomes in patients with ALSP in the Core StudyWeek 24 and Week 52

Correlations of Week 24 and Week 52 biomarker changes and clinical outcomes

To evaluate the effects of iluzanebart on biomarkers of target engagement in participants with ALSP in the Core StudyWeek 24 and Week 52

Change from Baseline to Week 24 and Week 52 in soluble colony-stimulating factor 1 receptor (sCSF1R) in CSF

To evaluate the effects of iluzanebart on volumetric magnetic resonance imaging in participants with ALSP in the Core StudyWeek 24 and Week 52

Change from Baseline to Week 24 and Week 52 in volumetric magnetic resonance imaging (MRI). The unit of this measure is ml.

Trial Locations

Locations (10)

Investigative Site 8

🇳🇱

Amsterdam, Netherlands

Investigative Site 7

🇩🇪

Leipzig, Germany

Investigative Site 3

🇺🇸

San Francisco, California, United States

Investigative Site 9

🇩🇪

Tübingen, Germany

Investigative Site 1

🇺🇸

Jacksonville, Florida, United States

Investigative Site 2

🇺🇸

Englewood, Colorado, United States

Investigative Site 5

🇺🇸

Boston, Massachusetts, United States

Investigative Site 6

🇺🇸

Philadelphia, Pennsylvania, United States

Investigative Site 10

🇫🇷

Paris, France

Investigative Site 4

🇬🇧

London, United Kingdom

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