A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
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|ClinicalTrials.gov Identifier: NCT05677659|
Recruitment Status : Recruiting
First Posted : January 10, 2023
Last Update Posted : March 20, 2023
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|Condition or disease||Intervention/treatment||Phase|
|ALSP||Drug: VGL101||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Solution administered via Intravenous Infusion (IV)
Solution administered via Intravenous Infusion (IV)
- 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
- 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.
- 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.
- 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
- 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
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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:
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.
- 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.
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
|Contact: Vigil Clinical Trials||+1 857 254 email@example.com|
|United States, California|
|Investigative Site 3||Recruiting|
|San Francisco, California, United States, 94158|
|United States, Colorado|
|Investigative Site 2||Recruiting|
|Englewood, Colorado, United States, 80113|
|United States, Florida|
|Investigative Site 1||Recruiting|
|Jacksonville, Florida, United States, 32224|
|Responsible Party:||Vigil Neuroscience, Inc.|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Hereditary Diffuse Leukoencephalopathy with Spheroids
Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia
CSF1R gene mutation
Central Nervous System Diseases
Nervous System Diseases