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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05677659
Other study ID # VGL101-01.201
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date December 14, 2022
Est. completion date March 31, 2027

Study information

Verified date May 2024
Source Vigil Neuroscience, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 20
Est. completion date March 31, 2027
Est. primary completion date July 31, 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Key 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 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
VGL101
Solution administered via Intravenous Infusion (IV)

Locations

Country Name City State
France Investigative Site 10 Paris
Germany Investigative Site 7 Leipzig
Germany Investigative Site 9 Tübingen
Netherlands Investigative Site 8 Amsterdam
United Kingdom Investigative Site 4 London
United States Investigative Site 5 Boston Massachusetts
United States Investigative Site 2 Englewood Colorado
United States Investigative Site 1 Jacksonville Florida
United States Investigative Site 6 Philadelphia Pennsylvania
United States Investigative Site 3 San Francisco California

Sponsors (1)

Lead Sponsor Collaborator
Vigil Neuroscience, Inc.

Countries where clinical trial is conducted

United States,  France,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Core Study Adverse Events 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 Week 24 and Week 52
Primary Long-Term Extension Adverse Events To evaluate the safety and tolerability of iluzanebart for the treatment of ALSP by adverse events in the Long-Term Extension Weeks 52, 76, 100, 124, and 148
Secondary To evaluate the effects of iluzanebart on volumetric magnetic resonance imaging in participants with ALSP in the Core Study Change from Baseline to Week 24 and Week 52 in volumetric magnetic resonance imaging (MRI). The unit of this measure is ml. Week 24 and Week 52
Secondary To evaluate the effects of iluzanebart on biomarkers of disease progression in participants with ALSP in the Core Study Change from Baseline to Week 24 and Week 52 in neurofilament light chain (NfL) in cerebrospinal fluid (CSF) and blood Week 24 and Week 52
Secondary To evaluate the effects of iluzanebart on biomarkers of target engagement in participants with ALSP in the Core Study Change from Baseline to Week 24 and Week 52 in soluble colony-stimulating factor 1 receptor (sCSF1R) in CSF Week 24 and Week 52
Secondary 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 Correlations of Week 24 and Week 52 biomarker changes and clinical outcomes Week 24 and Week 52
See also
  Status Clinical Trial Phase
Recruiting NCT05020743 - Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Enrolling by invitation NCT04503213 - A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project