Natural History and Outcome Measures in Alexander Disease
| Status: | Recruiting |
|---|---|
| Healthy: | No |
| Age Range: | Any |
| Updated: | 10/10/2018 |
| Start Date: | January 2016 |
| End Date: | December 2026 |
| Contact: | Amy Waldman, MD |
| Phone: | 215-590-1719 |
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.
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.
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.
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.
Inclusion Criteria:
- Diagnosed with Alexander Disease
Exclusion Criteria:
- Other Leukodystrophies will not be enrolled
We found this trial at
1
site
South 34th Street
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
215-590-1000
Principal Investigator: Amy T Waldman, MD, MSCE
Phone: 267-425-2063
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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