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Evaluation of Outcome Metrics in Alexander Disease
Who can join
All ages · All sexes
Full eligibility criteria
Inclusion Criteria: * Diagnosed with Alexander Disease Exclusion Criteria: * Other Leukodystrophies will not be enrolled
About the study
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.
Sponsor: Children's Hospital of Philadelphia · Participants: 200 · Started: Jan 26, 2016
Contact the study team
- Amy Waldman, MD · Phone: 215-590-1719
- Geraldine Liu, MA · Phone: 267-425-2063
Official record on ClinicalTrials.gov — NCT02714764
Locations in the U.S.
| Pennsylvania | Children's Hospital of Philadelphia, Philadelphia |
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.