ALSPPhase 2NCT05677659
A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
A Phase 2 study testing VGL101 in people with ALSP, run by Vigil Neuroscience, Inc. at 10 sites in 5 countries.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ALSP
- What is being tested?
- VGL101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 10 sitesin 5 countries
- Study dates
- 14 Dec 2022to 4 Jun 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants who have documentation of a gene mutation in the CSF1R gene
- Participants fulfill both (Parts A and B) of the following criteria:
- The participant has more than 2 findings of clinical signs or symptoms in the following categories:
- Cognitive impairment or psychiatric problem
- Pyramidal signs on neurological examination
- Extrapyramidal signs, such as rigidity.
Show all 9Show fewer
- Epilepsy
- 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.
Who cannot join
- 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.
Trial locations and contacts
10 sites in 5 countries.
10 sites in 5 countries
United States5 sites
Investigative Site 5
Boston, Massachusetts, 02114
Investigative Site 2
Englewood, Colorado, 80113
Investigative Site 1
Jacksonville, Florida, 32224
Investigative Site 6
Philadelphia, Pennsylvania, 19104
Investigative Site 3
San Francisco, California, 94158
About this study
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)
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.
- Conditions
- ALSP
- Being tested
- VGL101
- Keywords
- ALSP, CSF1R, leukoencephalopathy, Hereditary Diffuse Leukoencephalopathy with Spheroids, HDLS, CSF1R-related Leukoencephalopathy, Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia, CSF1R gene mutation, IGNITE
- Medical subject headings
- Leukoencephalopathies, Hereditary Diffuse Leukoencephalopathy with Spheroids
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VGL101Drug
Solution administered via Intravenous Infusion (IV)
| Study group | Role | What they receive |
|---|---|---|
| VGL101 | Experimental | Solution administered via Intravenous Infusion (IV) Receives: Drug: VGL101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Through Week 52
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
Measured: Week 52 through Week 148
Secondary outcomes (10)
To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Core Study
Percentage of subjects with minimal radiological progression from Baseline to Week 52
Measured: Baseline and Week 52
To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Core Study
- Correlation between brain volume loss and Montreal Cognitive Assessment (MoCA) changes from Baseline to Week 52
- Correlation between brain volume loss and Cortical basal ganglia functional scale (CBFS) changes from Baseline to Week 52
Measured: Baseline and Week 52
To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Core Study
- Percentage of subjects with minimal progression in the MoCA scale, from Baseline to Week 52
- Percentage of subjects with minimal progression in CBFS from Baseline to Week 52
- Percentage of subjects who did not worsen in the Clinical Global Impression - Change (CGI-C) scale at Week 52
Measured: Baseline and Week 52
To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Core Study
Change from Baseline to Week 52 in neurofilament light chain (NfL) in and blood
Measured: Baseline and Week 52
To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Core Study
Serum and cerebrospinal fluid (CSF) concentrations of iluzanebart
Measured: Through Week 52
To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Long-Term Extension
Change from Baseline to Weeks 76, 100, 124, and 148 in brain volume loss
Measured: Baseline and Weeks 76, 100, 124, and 148
To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Long-Term Extension
- Correlation between brain volume loss and MoCA changes from Baseline to Weeks 76, 100, 124, and 148
- Correlation between brain volume loss and CBFS changes from Baseline to Weeks 76, 100, 124, and 148
Measured: Baseline and Weeks 76, 100, 124, and 148
To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Long-Term Extension
- Change from baseline to Weeks 64, 76, 88, 100, 112, 124, 136, and 148 in the MoCA and CBFS
- Mean CGI-C score at Weeks 64, 76, 88, 100, 112, 124, 136, and 148
Measured: Baseline and Weeks 64, 76, 88, 100, 112, 124, 136, and 148
To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Long-Term Extension
Change from Baseline to Weeks 76, 100, 124, and 148 in NfL in blood
Measured: Baseline and Weeks 76, 100, 124, and 148
To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Long-Term Extension
Serum and CSF concentrations of iluzanebart
Measured: Week 52 through Week 148
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2023
- Study start14 Dec 2022
- Primary completion14 Feb 2025
- Study completion4 Jun 2025
Why it stopped: No beneficial effects on biomarker or clinical efficacy endpoints
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSPStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vigil Neuroscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05677659
- Sponsor's protocol ID
- VGL101-01.201
- Status last verified
- Jun 2025
- First submitted
- 30 Nov 2022
- Last updated
- 11 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05677659
- Acronym / sponsor ID
- VGL101-01.201
- Conditions
- ALSP
- Keywords
- ALSP, CSF1R, leukoencephalopathy, Hereditary Diffuse Leukoencephalopathy with Spheroids, HDLS, CSF1R-related Leukoencephalopathy, Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia, CSF1R gene mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: No beneficial effects on biomarker or clinical efficacy endpoints
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Vigil Neuroscience, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Dec 2022 (actual)
- Primary completion
- 14 Feb 2025 (actual)
- Study completion
- 4 Jun 2025 (actual)
- First posted
- 10 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGL101 | Experimental | Drug: VGL101Solution administered via Intravenous Infusion (IV) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Participants who have documentation of a gene mutation in the CSF1R gene
- Participants fulfill both (Parts A and B) of the following criteria:
- 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 (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VGL101Solution administered via Intravenous Infusion (IV) | Drug | VGL101 |
Primary outcome
Core Study Adverse Events
Time frame: Through 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 Events
Time frame: Week 52 through Week 148
To evaluate the safety and tolerability of iluzanebart for the treatment of ALSP by adverse events in the Long-Term Extension
10 secondary outcomes
To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Core Study
Time frame: Baseline and Week 52
To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Core Study
Time frame: Baseline and Week 52
To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Core Study
Time frame: Baseline and Week 52
To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Core Study
Time frame: Baseline and Week 52
To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Core Study
Time frame: Through Week 52
To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Long-Term Extension
Time frame: Baseline and Weeks 76, 100, 124, and 148
To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Long-Term Extension
Time frame: Baseline and Weeks 76, 100, 124, and 148
To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Long-Term Extension
Time frame: Baseline and Weeks 64, 76, 88, 100, 112, 124, 136, and 148
To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Long-Term Extension
Time frame: Baseline and Weeks 76, 100, 124, and 148
To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Long-Term Extension
Time frame: Week 52 through Week 148
Results and safety
The study was stopped early (reason given: No beneficial effects on biomarker or clinical efficacy endpoints). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VGL101 in people with ALSP, run by Vigil Neuroscience, Inc. at 10 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Germany, France, Netherlands, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: No beneficial effects on biomarker or clinical efficacy endpoints). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Core Study Adverse Events (time frame: Through Week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2025.