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

Recruiting

• Conditions: ALSP
• Interventions: Other: No intervention

• 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

Study Timeline

• Study First Submitted: 2021-08-18
• Study First Submitted QC: 2021-08-18
• Study First Posted: 2021-08-25
• Study Start: 2021-09-13
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-13
• Last Update Posted: 2025-01-15
• Primary Completion: 2028-06
• Study Completion: 2028-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with ALSP	No intervention	-
Patients with Prodromal ALSP	No intervention	-

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

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

Investigative Site 6; 🇳🇱 Amsterdam, Netherlands

Investigative Site 7; 🇬🇧 London, United Kingdom