Natural History Study of Oculopharyngeal Muscular Dystrophy

Oculopharyngeal Muscular Dystrophy Clinical Trial

— NH-OPMD  

Official title:

Natural History Study of Oculopharyngeal Muscular Dystrophy

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03161847
• Other study ID #: 16-326
• Status: Withdrawn
• Start date: December 12, 2016
• Est. completion date: July 18, 2018

Study information

• Verified date: May 2017
• Source: University of New Mexico
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 18, 2018
• Est. primary completion date: July 18, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

Inclusion Criteria:

• OPMD by genetic criteria
• = 18 years old
• English-speaking

Exclusion Criteria:

• Another medical condition that precludes safe completion of study tasks (such as severe cardiac or respiratory disease)
• Another medical condition that causes symptoms similar to OPMD (i.e., ptosis, dysphagia [trouble swallowing] or limb weakness).
• History of head or neck cancer, or history of radiation to the head or neck
• A videofluoroscopic swallow study within the 12 months prior to study enrollment

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Oculopharyngeal
• Oculopharyngeal Muscular Dystrophy

Intervention

Other:
• Non-interventional study
Non-interventional study

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
University of New Mexico	University of Florida

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Muscle Strength over time	Change in Manual muscle testing over time	Baseline and every 9 months for 3 years
Secondary	Dysphagia severity over time	Videofluoroscopic Swallow Studies	Baseline and every 9 months for 3 years