Natural History Study of Oculopharyngeal Muscular Dystrophy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2016-12-12

Study Completion Date

2018-07-18

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Oculopharyngeal Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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OPMD Subjects

The study cohort consists of individuals with genetically confirmed OPMD who will be followed longitudinally using periodic standardized assessments of clinical status in an observational, non-interventional study.

Non-interventional study

Intervention Type OTHER

Non-interventional study

Interventions

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Non-interventional study

Intervention Type OTHER

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No