Evaluation of Outcome Metrics in Alexander Disease

Conditions

• Alexander Disease

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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 Dates

Start date

Jan 26, 2016

Status verified

Jan 2026

Primary completion

Dec 31, 2030

Completion

Dec 31, 2030

Study Design

Enrollment

200 participants (estimated)

Primary Outcome Measure

Change in Gross Motor Function Over Time [ Time Frame: Up to 10 years ]

Central Contacts

• Amy Waldman, MD
  215-590-1719
• Geraldine Liu, MA
  267-425-2063
  [email protected]

Locations (1)

Find similar trials in Philadelphia, PA

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