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Evaluation of Outcome Metrics in Alexander Disease
Sponsor: Children's Hospital of Philadelphia
Summary
The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. Specimens are collected to measure glial fibrillary acidic protein (GFAP) levels in cerebrospinal fluid (CSF) and blood. The data obtained from this study will be used for the design of future treatment trials.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
200
Start Date
2016-01-26
Completion Date
2030-12
Last Updated
2026-01-15
Healthy Volunteers
No
Conditions
Locations (1)
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States