Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders

April 19, 2022 updated by: University of Calgary

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

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).

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3M 1M4
        • Neuromuscular Clinic, South Health Campus

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Global Swallowing Function
Time Frame: 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.
Change in score from week 0 to week 5

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Global Swallowing Function
Time Frame: 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.
Change in score from week 0 to week 15; change in score from week 5 to week 15.
Maximum expiratory pressure (MEP)
Time Frame: 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.
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.
Volitional cough strength (peak cough flow)
Time Frame: 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.
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.
Forced vital capacity (FVC)
Time Frame: 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.
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.
Oral Intake
Time Frame: 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.
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.
Self-perceived swallowing impairment
Time Frame: 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.
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.
Biomarker analyses
Time Frame: 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.
Baseline measurement (week 0)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Calgary

Collaborators

Muscular Dystrophy Canada

Investigators

  • Principal Investigator: Gerald Pfeffer, MD, PhD, University of Calgary

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

May 1, 2022

Primary Completion (Anticipated)

December 1, 2022

Study Completion (Anticipated)

December 1, 2022

Study Registration Dates

First Submitted

June 6, 2019

First Submitted That Met QC Criteria

July 2, 2019

First Posted (Actual)

July 5, 2019

Study Record Updates

Last Update Posted (Actual)

April 21, 2022

Last Update Submitted That Met QC Criteria

April 19, 2022

Last Verified

April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • REB18-1121

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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