Major study tracks rare brain disease to pave way for future treatments

NCT ID NCT02714764

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 200 people with Alexander Disease, a rare brain disorder, to learn how the disease changes over time. Researchers measure movement, speech, swallowing, and quality of life, and collect blood and spinal fluid samples. The goal is to gather information that will help design future treatment trials.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.