Actively Recruiting
Evaluation of Outcome Metrics in Alexander Disease
Led by Children's Hospital of Philadelphia · Updated on 2026-01-15
200
Participants Needed
1
Research Sites
774 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Philadelphia
Lead Sponsor
I
Ionis Pharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Evaluation of Outcome Metrics in Alexander Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with Alexander Disease
You will not qualify if you...
- Other Leukodystrophies will not be enrolled
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
A
Amy Waldman, MD
CONTACT
G
Geraldine Liu, MA
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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