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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05677659
Recruitment Status : Recruiting
First Posted : January 10, 2023
Last Update Posted : March 20, 2023
Sponsor:
Information provided by (Responsible Party):
Vigil Neuroscience, Inc.

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
ALSP Drug: VGL101 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : December 14, 2022
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : June 30, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: VGL101
Solution administered via Intravenous Infusion (IV)
 Drug: VGL101
Solution administered via Intravenous Infusion (IV)


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Week 24 ]
    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


Secondary Outcome Measures :
  1. To evaluate the effects of VGL101 on volumetric magnetic resonance imaging in participants with ALSP [ Time Frame: Week 24 ]
    Change from Baseline to Week 24 in volumetric magnetic resonance imaging (MRI). The unit of this measure is ml.

  2. To evaluate the effects of VGL101 on in the ALSP severity score based on MRI in participants with ALSP [ Time Frame: Week 24 ]
    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.

  3. To evaluate the effects of VGL101 on biomarkers of disease progression in participants with ALSP [ Time Frame: Week 24 ]
    Change from Baseline to Week 24 in neurofilament light chain (NfL) in cerebrospinal fluid (CSF) and blood

  4. To evaluate the effects of VGL101 on biomarkers of target engagement in participants with ALSP [ Time Frame: Week 24 ]
    Change from Baseline to Week 24 in soluble colony-stimulating factor 1 receptor (sCSF1R) in CSF


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05677659


Contacts
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Contact: Vigil Clinical Trials +1 857 254 4445 trials@vigilneuro.com

Locations
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United States, California
Investigative Site 3 Recruiting
San Francisco, California, United States, 94158
Contact       trials@vigilneuro.com   
United States, Colorado
Investigative Site 2 Recruiting
Englewood, Colorado, United States, 80113
Contact       trials@vigilneuro.com   
United States, Florida
Investigative Site 1 Recruiting
Jacksonville, Florida, United States, 32224
Contact       trials@vigilneuro.com   
Sponsors and Collaborators
Vigil Neuroscience, Inc.
More Information
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Responsible Party: Vigil Neuroscience, Inc.
ClinicalTrials.gov Identifier: NCT05677659    
Other Study ID Numbers: VGL101-01.201
First Posted: January 10, 2023    Key Record Dates
Last Update Posted: March 20, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Vigil Neuroscience, Inc.:
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
Additional relevant MeSH terms:
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Leukoencephalopathies
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases