Screening in Oculopharyngeal Muscular Dystrophy

NCT02877784 | Completed

• 1 other identifier: IRB201601374
• Study Type: observational
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

Oculopharyngeal muscular dystrophy (OPMD) is a rare myopathic disease that results in progressive degeneration of the oral and pharyngeal muscular, resulting in severe dysphagia and dysarthria. OPMD is considered a rare disease; therefore, limited research is available on the natural progression of the disease or the utility of biomarkers to identify swallowing impairment. The aim of this study is:

1. 1.To identify accurate, reliable and non-invasive clinical markers of swallowing impairment
2. 2.To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.

Conditions

Oculopharyngeal Muscular Dystrophy

Keywords

screening; OPMD; dysphagia; dysarthria

Outcome Measures

Primary Outcomes (1)

• Dynamic Imaging Grade of Swallowing Toxicity

  Global Metric of swallowing Safety and Efficiency

  Baseline

Secondary Outcomes (2)

• Dietary intake as confirmed by Functional Oral Intake Scale (FOIS)

  Baseline

• Participant perception of swallowing related symptoms as confirmed by The Eating Assessment Tool 10 (EAT-10)

  Baseline

Study Arms (1)

Screening

Participants enrolled will undergo testing of the swallowing mechanism

Procedure: Videofluoroscopic swallowing study; Other: Functional Oral Intake Scale; Other: Eating Assessment Tool-10

Interventions

Videofluoroscopic swallowing study PROCEDURE

Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.

Also known as: VFSS, modified barium swallow (MBS)

Screening

Functional Oral Intake Scale OTHER

The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.

Also known as: FOIS

Screening

Eating Assessment Tool-10 OTHER

The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.

Also known as: EAT-10

Screening

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The sample population will consist of individuals with a diagnosis of oculopharyngeal muscular dystrophy.

You may qualify if:

• diagnosis of oculopharyngeal muscular dystrophy
• no allergies to barium or capsaicin,
• no tracheotomy or mechanical ventilation
• no significant concurrent respiratory disease (e.g., COPD).

You may not qualify if:

• Pregnant Women

Sponsors & Collaborators

• University of Florida: lead

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Oculopharyngeal; Deglutition Disorders; Dysarthria

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases; Articulation Disorders; Speech Disorders; Language Disorders; Communication Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Emily Plowman, PhD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 19, 2016
• First Posted: August 24, 2016
• Study Start: October 1, 2016
• Primary Completion: August 1, 2017
• Study Completion: August 1, 2017
• Last Updated: March 27, 2020

Record last verified: 2020-03

Locations

US (1)