Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

January 2, 2024 updated by: Vigil Neuroscience, Inc.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
  • Germany
  • Netherlands
  • United Kingdom
  • United States
    • California
      • San Francisco, California, United States, 94158
    • Colorado
      • Englewood, Colorado, United States, 80113
    • Florida
      • Boca Raton, Florida, United States, 33486
        • Active, not recruiting
        • Investigative Site 1
      • Jacksonville, Florida, United States, 32224
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Investigative Site 11
        • Contact:
          • trials@vigilneuro.com
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with Definitive ALSP and Prodromal ALSP.

Description

Key Inclusion Criteria for both Definitive ALSP patients and Prodromal ALSP patients:

1. Subjects who have documentation of a gene mutation in the CSF1R gene

Key Inclusion Criteria for Definitive ALSP patients, only:

  1. Subjects who fulfill both of the following criteria (a and b):

    a. 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 b. 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.

  2. Subjects who, in the investigator's opinion, have demonstrated clinical progression of their ALSP within the past year.
  3. Subjects who meet the criteria for definitive ALSP must have a designated caregiver who spends at least 4 hours per week with them. The caregiver 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 caregiver ICF.

Key Inclusion Criteria for Prodromal ALSP patients, only:

1. MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum. Prodromal subjects may have none or up to and including 2 ALSP-related clinical signs or symptoms (i.e., they do not meet the clinical criteria outlined in the Definitive ALSP as "more than two").

Key Exclusion Criteria (for all study participants):

  1. 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
  2. Subjects who are unable to undergo MRI
  3. 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.
  4. Subjects who have previously undergone HSCT or plan to undergo HSCT within 12 months of the Screening/Baseline visit.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with ALSP
Other: No intervention
Not applicable for a Natural History Study
Patients with Prodromal ALSP
Other: No intervention
Not applicable for a Natural History Study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Magnetic Resonance Imaging (MRI)
Time Frame: Months 6, 12, 18, 24, 30, and 36 Months
Change from Baseline in structural and volumetric MRI
Months 6, 12, 18, 24, 30, and 36 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vigil Neuroscience, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 13, 2021

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

June 1, 2027

Study Registration Dates

First Submitted

August 18, 2021

First Submitted That Met QC Criteria

August 18, 2021

First Posted (Actual)

August 25, 2021

Study Record Updates

Last Update Posted (Estimated)

January 3, 2024

Last Update Submitted That Met QC Criteria

January 2, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VGL101-01.002

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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