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Alexander Disease

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Evaluation of Outcome Metrics in Alexander Disease

Participants will be asked to complete physical examinations including physical therapy, occupational therapy, speech and language therapy, and swallowing assessments. Patients (or caretakers) may be asked to complete questionnaires as well. The study asks for participants to return at least once yearly to repeat assessments.

Purpose of Study

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. The data obtained from this study will be used for the design of future treatment trials.

Eligibility

Sex:
Female Icon + Male Icon

Key Inclusion / Exclusion Criteria

Inclusion Criteria:

  • Diagnosed with Alexander Disease

Exclusion Criteria:

  • Other Leukodystrophies will not be enrolled

Conditions:

Alexander Disease

Status:

Recruiting

Study Type:

Observational

Clinical Trials ID

NCT02714764

Read Detailed Summary:

Start Date / End Date:

01 2016 - 12 2026

Enrollment:

100

Full Details:

https://clinicaltrials.gov/ct2/show/NCT02714764

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