Evaluation of Outcome Metrics in Alexander Disease

Recruiting now

Conditions studied: Alexander Disease

In brief

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 facts

Study ID

NCT02714764

Run by

Children's Hospital of Philadelphia

People needed

200

Starts

2016-01-26

Expected to finish

2030-12-01

Last updated by the study team

2026-01-15

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosed with Alexander Disease

You may not qualify if…

• Other Leukodystrophies will not be enrolled

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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