Back to resultsExpiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
Primary Purpose
Oculopharyngeal Muscular Dystrophy, Muscular Dystrophies, Myopathy; Hereditary
Status
Enrolling by invitation
Phase
Not Applicable
Locations
Canada
Study Type
Interventional
Intervention
Expiratory muscle strength therapy (EMST150, Aspire LLC)
Sponsored by
About this trial
This is an interventional treatment trial for Oculopharyngeal Muscular Dystrophy focused on measuring Dysphagia, Myopathy, Muscular Dystrophy
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)
Sites / Locations
- Neuromuscular Clinic, South Health Campus
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Sham Comparator
Arm Label
EMST therapy
Sham EMST therapy
Arm Description
Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer.
Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance.
Outcomes
Primary Outcome Measures
Global Swallowing Function
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
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)
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)
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)
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
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
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
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.
Full Information
NCT ID
NCT04009408
First Posted
June 6, 2019
Last Updated
April 19, 2022
Sponsor
University of Calgary
Collaborators
Muscular Dystrophy Canada
1. Study Identification
Unique Protocol Identification Number
NCT04009408
Brief Title
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
2. Study Status
Record Verification Date
April 2022
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 2022 (Anticipated)
Primary Completion Date
December 1, 2022 (Anticipated)
Study Completion Date
December 1, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Calgary
Collaborators
Muscular Dystrophy Canada
4. Oversight
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
Data Monitoring Committee
No
5. 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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Oculopharyngeal Muscular Dystrophy, Muscular Dystrophies, Myopathy; Hereditary
Keywords
Dysphagia, Myopathy, Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Parallel group, double blind, sham controlled study
Masking
ParticipantOutcomes Assessor
Masking Description
Participants will not be informed until the end of study whether they received active intervention or sham intervention
Allocation
Randomized
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
EMST therapy
Arm Type
Experimental
Arm Description
Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer.
Arm Title
Sham EMST therapy
Arm Type
Sham Comparator
Arm Description
Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance.
Intervention Type
Device
Intervention Name(s)
Expiratory muscle strength therapy (EMST150, Aspire LLC)
Intervention Description
Active therapy calibrated to the participant's maximum expiratory pressure
Primary Outcome Measure Information:
Title
Global Swallowing Function
Description
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.
Time Frame
Change in score from week 0 to week 5
Secondary Outcome Measure Information:
Title
Global Swallowing Function
Description
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.
Time Frame
Change in score from week 0 to week 15; change in score from week 5 to week 15.
Title
Maximum expiratory pressure (MEP)
Description
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.
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.
Title
Volitional cough strength (peak cough flow)
Description
Measure of cough strength that is assessed using a spirometer, measured in litres per minute (L/min). A higher score is a better outcome.
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.
Title
Forced vital capacity (FVC)
Description
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.
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.
Title
Oral Intake
Description
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.
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.
Title
Self-perceived swallowing impairment
Description
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.
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.
Title
Biomarker analyses
Description
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.
Time Frame
Baseline measurement (week 0)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gerald Pfeffer, MD, PhD
Organizational Affiliation
University of Calgary
Official's Role
Principal Investigator
Facility Information:
Facility Name
Neuromuscular Clinic, South Health Campus
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3M 1M4
Country
Canada
12. IPD Sharing Statement
Plan to Share IPD
No
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Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
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