Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders

NCT04009408 | Unknown FDA Device

• 1 other identifier: REB18-1121
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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).

Conditions

Oculopharyngeal Muscular Dystrophy; Muscular Dystrophies; Myopathy; Hereditary

Keywords

Dysphagia; Myopathy; Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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.

  Change in score from week 0 to week 5

Secondary Outcomes (7)

• Global Swallowing Function

  Change in score from week 0 to week 15; change in score from week 5 to week 15.

• 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.

• 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.

• 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.

• 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.

Study Arms (2)

EMST therapy

EXPERIMENTAL

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)

Sham EMST therapy

SHAM COMPARATOR

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)

Interventions

Expiratory muscle strength therapy (EMST150, Aspire LLC) DEVICE

Active therapy calibrated to the participant's maximum expiratory pressure

EMST therapy; Sham EMST therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of oculopharyngeal muscular dystrophy (OPMD)
• 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.)

You may not qualify if:

• 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)

Sponsors & Collaborators

• University of Calgary: lead
• Muscular Dystrophy Canada: collaborator

Study Sites (1)

Neuromuscular Clinic, South Health Campus

Calgary, Alberta, T3M 1M4, Canada

Location

Related Publications (23)

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MeSH Terms

Conditions

Muscular Dystrophy, Oculopharyngeal; Muscular Dystrophies; Muscular Diseases; Deglutition Disorders

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases

Study Officials

• Gerald Pfeffer, MD, PhD

  University of Calgary

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: Participants will not be informed until the end of study whether they received active intervention or sham intervention
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Parallel group, double blind, sham controlled study

Study Record Dates

• First Submitted: June 6, 2019
• First Posted: July 5, 2019
• Study Start: May 1, 2022
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: April 21, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)