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 Type
OBSERVATIONAL
Enrollment
56
Not applicable for a Natural History Study
Investigative Site 4
San Francisco, California, United States
Investigative Site 5
Englewood, Colorado, United States
Investigative Site 1
Boca Raton, Florida, United States
Investigative Site 2
Jacksonville, Florida, United States
Investigative Site 11
Boston, Massachusetts, United States
Investigative Site 10
Philadelphia, Pennsylvania, United States
Investigative Site 12
São Paulo, Brazil
Investigative Site 3
London, Ontario, Canada
Investigative Site 8
Leipzig, Germany
Investigative Site 9
Tübingen, Germany
...and 2 more locations
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 6
Time frame: Month 6
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 12
Time frame: Month 12
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 18
Time frame: Month 18
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 24
Time frame: Month 24
Magnetic Resonance Imaging (MRI) Ventricle Volume
Change from Baseline in ventricle volume at Month 30
Time frame: Month 30
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