Natural History Study in Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia

Description

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

Study Overview

Study Details

Study overview

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.

A Natural History Study of Patients with Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP)

Natural History Study in Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia

Condition
ALSP
Intervention / Treatment

-

Contacts and Locations

San Francisco

Investigative Site 4, San Francisco, California, United States, 94158

Englewood

Investigative Site 5, Englewood, Colorado, United States, 80113

Boca Raton

Investigative Site 1, Boca Raton, Florida, United States, 33486

Jacksonville

Investigative Site 2, Jacksonville, Florida, United States, 32224

Boston

Investigative Site 11, Boston, Massachusetts, United States, 02114

Philadelphia

Investigative Site 10, 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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

    Ages Eligible for Study

    18 Years to

    Sexes Eligible for Study

    ALL

    Accepts Healthy Volunteers

    No

    Collaborators and Investigators

    Vigil Neuroscience, Inc.,

    Study Record Dates

    2027-12