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Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders

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ClinicalTrials.gov Identifier: NCT04009408
Recruitment Status : Not yet recruiting
First Posted : July 5, 2019
Last Update Posted : March 24, 2020
Sponsor:
Collaborator:
Muscular Dystrophy Canada
Information provided by (Responsible Party):
University of Calgary

Study Description
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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) Not Applicable

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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Parallel 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
Estimated Study Start Date : July 1, 2021
Estimated Primary Completion Date : July 1, 2022
Estimated Study Completion Date : July 1, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04009408


Contacts
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Contact: Robyn Wells, MSc 403-483-2277 robyn.wells@ucalgary.ca
Contact: Gerald Pfeffer, MD, PhD 403-210-3926 gerald.pfeffer@ucalgary.ca

Sponsors and Collaborators
University of Calgary
Muscular Dystrophy Canada
Investigators
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Principal Investigator: Gerald Pfeffer, MD, PhD University of Calgary
More Information
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Publications:

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Responsible Party: University of Calgary
ClinicalTrials.gov Identifier: NCT04009408    
Other Study ID Numbers: REB18-1121
First Posted: July 5, 2019    Key Record Dates
Last Update Posted: March 24, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by University of Calgary:
Dysphagia
Myopathy
Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Diseases
Muscular Dystrophy, Oculopharyngeal
Neuromuscular Diseases
 Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Genetic Diseases, Inborn