ALSPALSPNCT05020743
Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
An observational study of ALSP, run by Vigil Neuroscience, Inc. at 12 sites in 6 countries.
Trial summary
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative disease for which no definitive treatment options and limited information on the natural history of the disease are available. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ALSP
- How many people
- 56 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 13 Sep 2021to 30 May 2025
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
Study population: Patients with Definitive ALSP and Prodromal ALSP.
Who may be able to join
- Subjects who have documentation of a gene mutation in the CSF1R gene (prior to enrollment)
- Subjects who fulfill both of the following criteria (a and b):
- More than two findings of clinical signs or symptoms in the following categories: i. Cognitive impairment or psychiatric problem ii. Pyramidal signs on neurological examination iii. Extrapyramidal signs, such as rigidity, tremor, abnormal gait, or bradykinesia iv. Epilepsy
- MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum NOTE: Subjects with other causes of leukoencephalopathy, including vascular dementia, multiple sclerosis, or leukodystrophy (e.g., adrenoleukodystrophy, Krabbe disease, metachromatic leukodystrophy), will be excluded.
- Subjects who, in the investigator's opinion, have demonstrated clinical progression of their ALSP within the past year.
- Subjects who meet the criteria for definitive ALSP must have a designated study partner (i.e caregiver) who spends at least 4 hours per week with them. The study partner must be able and willing to assist the subject in complying with the study requirements, be able to provide information during study visits, and be willing to sign a study partner ICF. Subjects who do not have a study partner may be enrolled at the investigator's discretion if they are able to comply with protocol requirements.
Who cannot join
- Subjects with any neurological or psychiatric diseases that can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but not limited to, Alzheimer's disease, frontotemporal dementia, ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's disease, and Down syndrome, or with active alcohol/drug abuse
- Subjects who are unable to undergo MRI
- Subjects 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 subject's participation in the study.
- Subjects who have previously undergone HSCT or plan to undergo HSCT within 12 months of the Screening/Baseline visit.
Trial locations and contacts
12 sites in 6 countries.
12 sites in 6 countries
United States6 sites
Investigative Site 1
Boca Raton, Florida, 33486
Investigative Site 11
Boston, Massachusetts, 02114
Investigative Site 5
Englewood, Colorado, 80113
Investigative Site 2
Jacksonville, Florida, 32224
Investigative Site 10
Philadelphia, Pennsylvania, 19104
Investigative Site 4
San Francisco, California, 94158
Germany2 sites
Investigative Site 8
Leipzig, 04103
Investigative Site 9
Tübingen, 72076
About this study
Official title: A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative disease for which no definitive treatment options and limited information on the natural history of the disease are available. The structural, genetic, and neuropathophysiological abnormalities of ALSP lead to the onset of neurologic symptoms, such as moderate to severe motor and neuropsychiatric impairments. This natural history study will collect data to contribute to the development of future novel therapies that focus on the neuropathophysiological features that underlie ALSP and that are essential to reverse, delay, or stop progression of this debilitating disorder.
- Conditions
- ALSP
- Being tested
- No intervention
- 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, POLD, Pigmentary Orthochromatic Leukodystrophy
- 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.
No interventionOther
Not applicable for a Natural History Study
| Study group | Role | What they receive |
|---|---|---|
| Patients with ALSP | Not described Receives: Other: No intervention | |
| Patients with Prodromal ALSP | Not described Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 6
Measured: Month 6
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 12
Measured: Month 12
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 18
Measured: Month 18
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 24
Measured: Month 24
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 30
Measured: Month 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2021
- Study start13 Sep 2021
- Primary completion30 May 2025
- Study completion30 May 2025
Why it stopped: No beneficial effects on biomarker or clinical efficacy endpoints in the VGL101-01.201 Study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 56 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
- NCT05020743
- Sponsor's protocol ID
- VGL101-01.002
- Acronym
- ALSP
- Status last verified
- Jul 2025
- First submitted
- 18 Aug 2021
- Last updated
- 13 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)
- ClinicalTrials.gov ID
- NCT05020743
- Acronym / sponsor ID
- ALSP / VGL101-01.002
- 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
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: No beneficial effects on biomarker or clinical efficacy endpoints in the VGL101-01.201 Study
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Vigil Neuroscience, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Sep 2021 (actual)
- Primary completion
- 30 May 2025 (actual)
- Study completion
- 30 May 2025 (actual)
- First posted
- 25 Aug 2021 (actual)
- Results
- Posted 13 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative disease for which no definitive treatment options and limited information on the natural history of the disease are available. The structural, genetic, and neuropathophysiological abnormalities of ALSP lead to the onset of neurologic symptoms, such as moderate to severe motor and neuropsychiatric impairments. This natural history study will collect data to contribute to the development of future novel therapies that focus on the neuropathophysiological features that underlie ALSP and that are essential to reverse, delay, or stop progression of this debilitating disorder.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with ALSP | – | Other: No intervention |
| Patients with Prodromal ALSP | – | Other: No intervention |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Definitive ALSP and Prodromal ALSP.
Key inclusion criteria (6 listed)
- Subjects who have documentation of a gene mutation in the CSF1R gene (prior to enrollment)
- Subjects who fulfill both of the following criteria (a and b):
- Subjects who, in the investigator's opinion, have demonstrated clinical progression of their ALSP within the past year.
- Subjects who meet the criteria for definitive ALSP must have a designated study partner (i.e caregiver) who spends at least 4 hours per week with them. The study partner must be able and willing to assist the subject in complying with the study requirements, be able to provide information during study visits, and be willing to sign a study partner ICF. Subjects who do not have a study partner may be enrolled at the investigator's discretion if they are able to comply with protocol requirements.
Key exclusion criteria (4 listed)
- Subjects with any neurological or psychiatric diseases that can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but not limited to, Alzheimer's disease, frontotemporal dementia, ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's disease, and Down syndrome, or with active alcohol/drug abuse
- Subjects who are unable to undergo MRI
- Subjects 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 subject's participation in the study.
- Subjects who have previously undergone HSCT or plan to undergo HSCT within 12 months of the Screening/Baseline visit.
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 |
|---|---|---|
| No interventionNot applicable for a Natural History Study | Other | Patients with ALSP, Patients with Prodromal ALSP |
Primary outcome
Magnetic Resonance Imaging (MRI) Ventricle Volume
Time frame: Month 6
Change from Baseline in ventricle volume at Month 6
Magnetic Resonance Imaging (MRI) Ventricle Volume
Time frame: Month 12
Change from Baseline in ventricle volume at Month 12
Magnetic Resonance Imaging (MRI) Ventricle Volume
Time frame: Month 18
Change from Baseline in ventricle volume at Month 18
Magnetic Resonance Imaging (MRI) Ventricle Volume
Time frame: Month 24
Change from Baseline in ventricle volume at Month 24
Magnetic Resonance Imaging (MRI) Ventricle Volume
Time frame: Month 30
Change from Baseline in ventricle volume at Month 30
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for ALSP
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Enrolling by invitationObservational study
A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation
CSF1R-related Leukoencephalopathy, ALSP
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
RecruitingObservational study
The Myelin Disorders Biorepository Project
Leukodystrophy, White Matter Disease
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.
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of ALSP, run by Vigil Neuroscience, Inc. at 12 sites in 6 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 6 inclusion and 4 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 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Germany, Brazil, Canada, Netherlands and 1 more country. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Magnetic Resonance Imaging (MRI) Ventricle Volume (time frame: Month 6). The study lists 5 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 13 Aug 2025.