Evaluation of Outcome Metrics in Alexander Disease

Status: Recruiting
Location: See location...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Diagnosed with Alexander Disease

Locations
United States
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Amy Waldman, MD
215-590-1719
Backup
Geraldine Liu, MA
liug@chop.edu
267-425-2063
Time Frame
Start Date: 2016-01-26
Estimated Completion Date: 2030-12
Participants
Target number of participants: 200
Related Therapeutic Areas
Sponsors
Leads: Children's Hospital of Philadelphia
Collaborators: Ionis Pharmaceuticals, Inc., University of Wisconsin, Madison, Pennsylvania Department of Health

This content was sourced from clinicaltrials.gov