Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders

Sponsor

University of Calgary (Other)

Overall Status

Enrolling by invitation

CT.gov ID

NCT04009408

Collaborator

Muscular Dystrophy Canada (Other)

Enrollment

Location

Arms

Anticipated Duration (Months)

2.8

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Oculopharyngeal Muscular Dystrophy Muscular Dystrophies Myopathy; Hereditary	Device: Expiratory muscle strength therapy (EMST150, Aspire LLC)	N/A

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Parallel group, double blind, sham controlled studyParallel group, double blind, sham controlled study

Masking:

Double (Participant, Outcomes Assessor)

Masking Description:

Participants will not be informed until the end of study whether they received active intervention or sham intervention

Primary Purpose:

Treatment

Official Title:

Interventional Study of Expiratory Muscle Strength Training as a Treatment in Neuromuscular Disorders

Anticipated Study Start Date :

May 1, 2022

Anticipated Primary Completion Date :

Dec 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: EMST therapy Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer.	Device: Expiratory muscle strength therapy (EMST150, Aspire LLC) Active therapy calibrated to the participant's maximum expiratory pressure
Sham Comparator: Sham EMST therapy Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance.	Device: Expiratory muscle strength therapy (EMST150, Aspire LLC) Active therapy calibrated to the participant's maximum expiratory pressure

Arm

Intervention/Treatment

Experimental: EMST therapy

Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer.

Device: Expiratory muscle strength therapy (EMST150, Aspire LLC)

Active therapy calibrated to the participant's maximum expiratory pressure

Sham Comparator: Sham EMST therapy

Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance.

Device: Expiratory muscle strength therapy (EMST150, Aspire LLC)

Active therapy calibrated to the participant's maximum expiratory pressure

Outcome Measures

Primary Outcome Measures

Global Swallowing Function [Change in score from week 0 to week 5]
Global swallowing function is rated from videofluoroscopy swallowing studies (VFSS), using the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST), a validated 5-point scale. Global swallowing function is rated from 0-4: 0 = no pharyngeal dysphagia; 1 = mild; 2 = moderate; 3 = severe; 4 = life-threatening. A lower score is a better outcome.

Secondary Outcome Measures

Global Swallowing Function [Change in score from week 0 to week 15; change in score from week 5 to week 15.]
Global swallowing function is rated from videofluoroscopy swallowing studies (VFSS), using the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST), a validated 5-point scale. Global swallowing function is rated from 0-4: 0 = no pharyngeal dysphagia; 1 = mild; 2 = moderate; 3 = severe; 4 = life-threatening.
Maximum expiratory pressure (MEP) [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.]
MEP is a measure of respiratory muscle strength and is assessed with a handheld manometer, measured in centimetres of water (cmH2O). A higher score is a better outcome.
Volitional cough strength (peak cough flow) [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 of cough strength that is assessed using a spirometer, measured in litres per minute (L/min). A higher score is a better outcome.
Forced vital capacity (FVC) [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 of how much air is exhaled during forced exhalation and is assessed with a spirometer, measured in litres. A higher score is a better outcome.
Oral Intake [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.]
A measure daily nutritional and hydration consumption. Oral intake is assessed using the Functional Oral Intake Scale (FOIS), a validated 7-point ordinal scale (1 = no oral intake; 2 = tube dependent with minimal/inconsistent oral intake; 3 = tube supplements with consistent oral intake; 4 = total oral intake in single consistency; 5 = total oral intake of multiple consistencies requiring special preparation; 6 = total oral intake with no special preparation, but must avoid specific foods or liquid items; 7 = total oral intake with no restrictions). A higher score is a better outcome.
Self-perceived swallowing impairment [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.]
Will be measured using the Eating Assessment Tool-10 (EAT-10), a self-administered, symptom-specific outcome instrument for dysphagia. The EAT-10 allows patients to rate their swallowing symptoms on scale of 0 = no problem to 4 = severe problem. A lower score is a better outcome.
Biomarker analyses [Baseline measurement (week 0)]
An optional blood sample will be collected for biomarker analysis, to identify correlations with clinical response. We will measure genetic biomarkers associated with swallowing function including rs6265, rs165599, rs10835211, rs17601696, and APOE4 genotype status. For these 5 genetic biomarkers, participants will be scored as having zero, one, or two alleles. This information will be used in subgroup analyses for the primary and secondary outcomes.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)