Clinical Trial: Natural History and Outcome Measures in Alexander Disease
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: Natural History and Outcome Measures in Alexander Disease
Brief 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. The data obtained from this study will be used for the design of future treatment trials.
Detailed Summary: 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.
Sponsor: Children's Hospital of Philadelphia
Current Primary Outcome: Change in Gross Motor Function Over Time [ Time Frame: Up to 10 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Change in the Bruininks-Oseretsky Test of Motor Proficiency Over Time [ Time Frame: Up to 10 years ]
Original Secondary Outcome: Same as current
Information By: Children's Hospital of Philadelphia
Dates:
Date Received: February 19, 2016
Date Started: January 2016
Date Completion: December 2026
Last Updated: September 19, 2016
Last Verified: September 2016