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Trial record 13 of 88 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

Screening in Oculopharyngeal Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02877784
Recruitment Status : Recruiting
First Posted : August 24, 2016
Last Update Posted : December 25, 2017
Sponsor:
Information provided by (Responsible Party):

August 19, 2016
August 24, 2016
December 25, 2017
October 2016
October 2018   (Final data collection date for primary outcome measure)
  • Kinematic Swallowing Measurements will be used to analyze a normal movement of swallowing [ Time Frame: Baseline ]
    Kinematic swallowing measurements is an objective analysis of videofluoroscopy. This involves capturing and manipulating digital images with computer technology to make exact timing measures of bolus flow and movement of structures, as well as spatial measurements of distance and area against reference points.
  • The IOPI will be used to measure Lingual strength and endurance [ Time Frame: Baseline ]
    The Iowa Oral Pressure Instrument device will measure the peak performance of lingual strength a via the action of a bulb placed on the hard palate. To assess maximum anterior isometric pressure, the bulb will be placed on the roof of the mouth and the participant will raise his or her tongue to apply pressure to this bulb and then quickly release the tongue back into a neutral position. This repetitive tongue movement will be performed three times and values will be recorded.
  • The nebulizer with cough protocol will be used to measure reflexive cough [ Time Frame: Baseline ]
    Reflexive cough will be assessed using an oral pneumotachograph, differential pressure transducer, and have a side port with a one-way inspiratory valve for nebulizer connection. The nebulizer will deliver three randomized blocks of 0, 50, 100, 200, and 500 μM capsaicin.
Same as current
Complete list of historical versions of study NCT02877784 on ClinicalTrials.gov Archive Site
  • Global disease progression as confirmed by the ALS Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: Baseline ]
    The ALSFRS-R includes 12 questions. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best.
  • Participant perception of swallowing impairment as confirmed by Swallowing Quality of Life Questionnaire (SWAL-QOL) [ Time Frame: Baseline ]
    SWAL-QOL is a standardized psychometric scale measuring swallow-related QOL and containing 44-items relating to swallowing specific quality of life measured across 10 domains. Individual domain scores and a total SWAL-QOL score will be derived. Scores range from 0 to 100, with a score of 100 representing no impairment (the most favorable state)
  • Dietary intake as confirmed by Functional Oral Intake Scale (FOIS) [ Time Frame: Baseline ]

    FOIS is a validated 7-point scale indexing an individual's ability to intake food on a day-to-day basis.

    TUBE DEPENDENT (levels 1-3) No oral intake Tube dependent with minimal/inconsistent oral intake Tube supplements with consistent oral intake TOTAL ORAL INTAKE (levels 4-7) Total oral intake of a single consistency Total oral intake of multiple consistencies requiring special preparation Total oral intake with no special preparation, but must avoid specific foods or liquid items Total oral intake with no restrictions

  • Participant perception of swallowing related symptoms as confirmed by The Eating Assessment Tool 10 (EAT-10) [ Time Frame: Baseline ]
    EAT-10 is a 10-item validated self-administered dysphagia severity symptom survey. Score is 0 - 40 with 0 being the best possible score and 40 the worst possible score.
  • Participant perception of communication abilities as confirmed by Communication Effectiveness Survey [ Time Frame: Baseline ]
    A measure of self-rated communication abilities across a variety of settings as perceived by the patients and/or their caregivers will be measured using the 8-point Communication Effectiveness Survey (CES)
  • Bulbar function confirmed by the Center for Neurologic Studies Bulbar Function Scale (CNS-BFS) [ Time Frame: Baseline ]
Same as current
Not Provided
Not Provided
 
Screening in Oculopharyngeal Muscular Dystrophy
Determination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular Dystrophy

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. To identify accurate, reliable and non-invasive clinical markers of swallowing impairment
  2. To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.
Participants will be recruited from the Neurology clinic at the University of Florida. The single evaluation will occur in the PIs research laboratory at the University of Florida, Gainesville, Florida.
Interventional
Early Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Oculopharyngeal Muscular Dystrophy
  • Device: Iowa Oral Performance Instrument
    The Iowa Oral Performance Instrument (IOPI) is a device that measures the peak pressure performance of lingual strength and endurance via the action of a bulb placed on the hard palate.
    Other Name: IOPI
  • Drug: Capsaicin
    A capsaicin challenge with three randomized blocks of 0, 50, 100, 200, and 500 μM capsaicin. The capsaicin will be dissolved in a vehicle solution consisting of 80% physiological saline, and 20% ethanol. Participants will be given the instruction "cough if you need to" prior to capsaicin delivery.The solution will be administered automatically upon detection of an inspired breath and there will be a minimum of one minute between each trial. This is to test the reflexive cough testing for upper airway sensitivity and motor thresholds.
    Other Name: Hot pepper
  • Device: Electrical Impedance Myography (EIM)
    The SFB7 Bioimpedance device measures the function of the tongue by calculating measures of reactance, resistance and phase once a custom electrode array is placed on the midline of the subject's tongue for approximately 2 seconds.
    Other Name: EIM
  • Procedure: Videofluoroscopic swallowing study
    Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.
    Other Names:
    • VFSS
    • modified barium swallow (MBS)
  • Other: Swallowing Related Quality of Life Questionnaire
    Swallowing Quality of Life Questionnaire (SWAL-QOL) will be used for participant reporting of swallow-related quality of life.
    Other Name: SWAL-QOL
  • Other: Functional Oral Intake Scale
    The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.
    Other Name: FOIS
  • Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised
    The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) will be used to track global disease progression in participants.
    Other Name: ALSFRS-R
  • Other: Eating Assessment Tool-10
    The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.
    Other Name: EAT-10
  • Other: Communicative Effectiveness Survey
    The Communication Effectiveness Survey (CES) will be used for participant reporting of communication abilities across different speech contexts.
    Other Name: CES
  • Other: The Center for Neurologic Studies Bulbar Function Scale
    The Center for Neurologic Studies Bulbar Function Scale (CNS-BFS) will be used to report bulbar function in study participants.
    Other Name: CNS-BFS
Experimental: Screening
Participants enrolled will have the following test: Micro Mouth Pressure Meter, reflexive cough testing (with capsaicin used in blocks), lingual strength and endurance trials using the Iowa Oral Performance Instrument, Electrical Impedance Myography of the tongue, Pulmonary Function Testing, and a Videofluoroscopic Swallowing Study (VFSS). In addition, the patient will complete the following surveys: Swallowing Related Quality of Life Questionnaire (SWAL-QOL), Eating Assessment Tool-10 (EAT-10), Functional Oral Intake Scale (FOIS), Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R), The Center for Neurologic Studies Bulbar Function Scale (CNS-BFS), and the Communicative Effectiveness Survey (CES).
Interventions:
  • Device: Iowa Oral Performance Instrument
  • Drug: Capsaicin
  • Device: Electrical Impedance Myography (EIM)
  • Procedure: Videofluoroscopic swallowing study
  • Other: Swallowing Related Quality of Life Questionnaire
  • Other: Functional Oral Intake Scale
  • Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised
  • Other: Eating Assessment Tool-10
  • Other: Communicative Effectiveness Survey
  • Other: The Center for Neurologic Studies Bulbar Function Scale
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
15
October 2018
October 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

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

Exclusion Criteria:

  • Pregnant Women
Sexes Eligible for Study: All
18 Years to 80 Years   (Adult, Senior)
No
Contact: Kelby Magennis, MPH 352-273-8632 kmagennis@phhp.ufl.edu
Contact: Emily Plowman 3522739215 eplowman@phhp.ufl.edu
United States
 
 
NCT02877784
IRB201601374
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
University of Florida
University of Florida
Not Provided
Principal Investigator: Emily Plowman, PhD University of Florida
University of Florida
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP