Clinical trial NCT04009408: Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
Official title: Interventional Study of Expiratory Muscle Strength Training as a Treatment in Neuromuscular Disorders
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Oculopharyngeal Muscular Dystrophy
Muscular Dystrophies
Myopathy; Hereditary
Keywords: Dysphagia
Myopathy
Muscular Dystrophy
Lead sponsor: University of Calgary
Sponsor class: OTHER
Collaborators: name: Muscular Dystrophy Canada
First posted: 2019-07-05
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel group, double blind, sham controlled study
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy (OPMD).

Detailed description: Outline:

Twenty participants with OPMD, with dysphagia, will be recruited from Neuromuscular clinics within Calgary. The investigators will enrol patients in a parallel group, sham-controlled, randomized clinical trial, with 10 participants in each group (active EMST and sham EMST).

Participants will have baseline measurements of: (i) global swallowing function via modified barium swallow study, (ii) maximum expiratory pressure, (iii) voluntary cough spirometry, (iv) forced vital capacity, (v) functional oral intake, (vi) patient report of self-perceived swallowing impairment (EAT-10 Questionnaire), and (vii) biomarker analyses.

Participants will undergo 5-weeks of EMST (active or sham). All baseline measurements will be repeated after 5-weeks of EMST and 10-weeks post-EMST to measure durability of effect.

Outcomes:

The end-goal of the current research is to obtain preliminary data for the benefit of EMST in a new study population, and direct future studies that may provide evidence for a new standard of care in treating neuromuscular diagnoses.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of oculopharyngeal muscular dystrophy (OPMD)
* 18 years of age or older
* Must be capable of providing informed consent
* Must be able to undergo respiratory function testing and swallowing studies
* Must have a forced vital capacity (FVC) greater than 60%
* A score of 3 or greater on the Eating Assessment Tool-10 (EAT-10; self-administered, symptom-specific outcome instrument for dysphagia. A score of 3 or greater indicates increased stress around eating)
* A score of 26 or greater on the Montreal Cognitive Assessment (MoCA; 30-point screening assessment used for detecting cognitive impairment. A score of 26 or greater is considered to be within functional limits.)

Exclusion Criteria:

* Severe coronary artery disease
* Acute myocardial infarction
* Moderate to severe hypovolemia
* Acute neurological events
* Unstable cardiac status
* Recent hernia
* Severe chronic obstructive pulmonary disease (COPD)
* Uncontrolled reflux issues
* Women who are pregnant, or who suspect they may be pregnant
* Cognitive impairment that would prevent comprehension of instructions and adherence to intervention guidelines (a score of less than 26 points on the MoCA)

Primary outcomes:
measure: Global Swallowing Function
timeFrame: Change in score from week 0 to week 5
Secondary outcomes:
measure: Global Swallowing Function
timeFrame: Change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Maximum expiratory pressure (MEP)
timeFrame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Volitional cough strength (peak cough flow)
timeFrame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Forced vital capacity (FVC)
timeFrame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Oral Intake
timeFrame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Self-perceived swallowing impairment
timeFrame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
measure: Biomarker analyses
timeFrame: Baseline measurement (week 0)
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Neuromuscular Clinic, South Health Campus
state: Alberta
Study officials:
affiliation: University of Calgary
name: Gerald Pfeffer, MD, PhD
role: PRINCIPAL_INVESTIGATOR
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