Oculopharyngeal Muscular DystrophyNCT02877784
Screening in Oculopharyngeal Muscular Dystrophy
An observational study of Oculopharyngeal Muscular Dystrophy, run by University of Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy
- What is being tested?
- Videofluoroscopic swallowing study, Functional Oral Intake Scale and Eating Assessment Tool-10
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2016to Aug 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The sample population will consist of individuals with a diagnosis of oculopharyngeal muscular dystrophy.
Who may be able to join
- diagnosis of oculopharyngeal muscular dystrophy
- no allergies to barium or capsaicin,
- no tracheotomy or mechanical ventilation
- no significant concurrent respiratory disease (e.g., COPD).
Who cannot join
- Pregnant Women
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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:
- To identify accurate, reliable and non-invasive clinical markers of swallowing impairment
- To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.
Read the detailed description
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.
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Being tested
- Videofluoroscopic swallowing study, Functional Oral Intake Scale, Eating Assessment Tool-10
- Keywords
- screening, OPMD, dysphagia, dysarthria
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal, Deglutition Disorders, Dysarthria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Videofluoroscopic swallowing studyProcedure or surgery
Also called VFSS, modified barium swallow (MBS)
Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.
Functional Oral Intake ScaleOther
Also called FOIS
The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.
Eating Assessment Tool-10Other
Also called EAT-10
The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.
| Study group | Role | What they receive |
|---|---|---|
| Screening | Participants enrolled will undergo testing of the swallowing mechanism Receives: Procedure: Videofluoroscopic swallowing study; Other: Functional Oral Intake Scale; Other: Eating Assessment Tool-10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dynamic Imaging Grade of Swallowing Toxicity
Global Metric of swallowing Safety and Efficiency
Measured: Baseline
Secondary outcomes (2)
Dietary intake as confirmed by Functional Oral Intake Scale (FOIS)
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
Measured: Baseline
Participant perception of swallowing related symptoms as confirmed by The Eating Assessment Tool 10 (EAT-10)
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.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2016 (estimated)
- Study startOct 2016
- Primary completionAug 2017
- Study completionAug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oculopharyngeal Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emily Plowman, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02877784
- Sponsor's protocol ID
- IRB201601374
- Status last verified
- Mar 2020
- First submitted
- 19 Aug 2016
- Last updated
- 27 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02877784
- Acronym / sponsor ID
- IRB201601374
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Keywords
- screening, OPMD, dysphagia, dysarthria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Study start
- Oct 2016
- Primary completion
- Aug 2017 (actual)
- Study completion
- Aug 2017 (actual)
- First posted
- 24 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- To identify accurate, reliable and non-invasive clinical markers of swallowing impairment
- To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Screening | – | Procedure: Videofluoroscopic swallowing study, Other: Functional Oral Intake Scale, Other: Eating Assessment Tool-10Participants enrolled will undergo testing of the swallowing mechanism |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The sample population will consist of individuals with a diagnosis of oculopharyngeal muscular dystrophy.
Key inclusion criteria (4 listed)
- diagnosis of oculopharyngeal muscular dystrophy
- no allergies to barium or capsaicin,
- no tracheotomy or mechanical ventilation
- no significant concurrent respiratory disease (e.g., COPD).
Key exclusion criteria (1 listed)
- Pregnant Women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Videofluoroscopic swallowing studyAlso: VFSS, modified barium swallow (MBS)Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing. | Procedure or surgery | Screening |
| Functional Oral Intake ScaleAlso: FOISThe Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits. | Other | Screening |
| Eating Assessment Tool-10Also: EAT-10The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity. | Other | Screening |
Primary outcome
Dynamic Imaging Grade of Swallowing Toxicity
Time frame: Baseline
Global Metric of swallowing Safety and Efficiency
2 secondary outcomes
Dietary intake as confirmed by Functional Oral Intake Scale (FOIS)
Time frame: Baseline
Participant perception of swallowing related symptoms as confirmed by The Eating Assessment Tool 10 (EAT-10)
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emily Plowman, PhD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Oculopharyngeal Muscular Dystrophy, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dynamic Imaging Grade of Swallowing Toxicity (time frame: Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2020.