Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
- Conditions
- ALSP
- Registration Number
- NCT05020743
- Lead Sponsor
- Vigil Neuroscience, Inc.
- Brief 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. 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 56
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Magnetic Resonance Imaging (MRI) Months 6, 12, 18, 24, 30, and 36 Months Change from Baseline in structural and volumetric MRI
- Secondary Outcome Measures
Name Time Method
Related Research Topics
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Trial Locations
- Locations (12)
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
Scroll for more (2 remaining)Investigative Site 4🇺🇸San Francisco, California, United States