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 ]

Change in Manual muscle testing over time


Original Primary Outcome: Same as current

Current Secondary Outcome: Dysphagia severity over time [ Time Frame: Baseline and every 9 months for 3 years ]

Videofluoroscopic Swallow Studies


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