Clinical Trial: Natural History Study of Oculopharyngeal Muscular Dystrophy
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Observational
Official Title: Natural History Study of Oculopharyngeal Muscular Dystrophy
Brief Summary: The objective of this study is to test a set of clinical outcome measures longitudinally in a cohort of OPMD patients to identify ones that show quantifiable change over time as the disease progresses. The investigators' goal is to delineate the natural history of OPMD.
Detailed Summary: OPMD patients will undergo a screening evaluation and testing to confirm the participants carry the OPMD mutation. Subjects fulfilling the inclusion/exclusion criteria will be enrolled and followed prospectively at regular intervals to determine the natural history of this disease. Measures of muscle function and swallowing will be made at baseline and at follow-up visits to measure natural clinical progression.
Sponsor: University of New Mexico
Current Primary Outcome: Muscle Strength over time [ Time Frame: Baseline and every 9 months for 3 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Dysphagia severity over time [ Time Frame: Baseline and every 9 months for 3 years ]
Original Secondary Outcome: Same as current
Information By: University of New Mexico
Dates:
Date Received: May 16, 2017
Date Started: July 2017
Date Completion: December 2024
Last Updated: May 18, 2017
Last Verified: May 2017