Evaluation of Outcome Metrics in Alexander Disease
Recruiting
Observational Study
Alexander Disease
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 200 (estimated)
- Sponsor
- Children's Hospital of Philadelphia · Other
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About This Trial
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) …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Diagnosed with Alexander Disease
Exclusion Criteria:
* Other Leukodystrophies will not be enrolled