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

ALSP Clinical Trial

Official title:

A Phase 2 Safety, Tolerability, and Proof-of-Concept Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP) (The Ignite Study)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

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

Study information

• Verified date: April 2024
• Source: Vigil Neuroscience, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a multicenter, open-label study to assess the safety and tolerability of 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 VGL101 on imaging and biomarkers of disease progression in subjects with ALSP. Participants will receive infusions of VGL101 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.

Related Conditions & MeSH terms

• ALSP
• Leukoencephalopathies

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	Adverse Events	To evaluate the safety and tolerability of VGL101 for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) by adverse events	Week 24
Secondary	To evaluate the effects of VGL101 on volumetric magnetic resonance imaging in participants with ALSP	Change from Baseline to Week 24 in volumetric magnetic resonance imaging (MRI). The unit of this measure is ml.	Week 24
Secondary	To evaluate the effects of VGL101 on biomarkers of disease progression in participants with ALSP	Change from Baseline to Week 24 in neurofilament light chain (NfL) in cerebrospinal fluid (CSF) and blood	Week 24
Secondary	To evaluate the effects of VGL101 on biomarkers of target engagement in participants with ALSP	Change from Baseline to Week 24 in soluble colony-stimulating factor 1 receptor (sCSF1R) in CSF	Week 24
Secondary	To evaluate the effects of VGL101 on the ALSP severity score based on MRI in participants with ALSP	Change from Baseline to Week 24 in the ALSP severity score based on MRI. The scale has a minimum score of 0 and maximum score of 57. Higher scores represent greater radiographical disease burden.	Week 24