NCT05020743

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

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

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 56 (actual)
• Sponsor: Vigil Neuroscience, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• ALSP

Interventions

Type	Name	Description
OTHER	No intervention	Not applicable for a Natural History Study

Timeline

• Start date: 2021-09-13
• Primary completion: 2025-05-30
• Completion: 2025-05-30
• First posted: 2021-08-25
• Last updated: 2025-08-13
• Results posted: 2025-08-13

Locations

12 sites across 6 countries: United States, Brazil, Canada, Germany, Netherlands, United Kingdom