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Evaluation of Outcome Metrics in Alexander Disease

NCT02714764 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2026-01-15

No results posted yet for this study

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.

Conditions

  • Alexander Disease

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • University of Wisconsin, Madison

    collaborator OTHER

  • Pennsylvania Department of Health

    collaborator OTHER_GOV

  • Children's Hospital of Philadelphia

    lead OTHER

Principal Investigators

  • Amy Waldman, MD · Children's Hospital of Philadelphia

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-26
Primary Completion
2030-12-31
Completion
2030-12-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02714764 on ClinicalTrials.gov