Interventional Study of Expiratory Muscle Strength Training as a Treatment in Neuromuscular Disorders
试验速览
- 阶段
- 不适用
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Global Swallowing Function
研究概览
简要总结
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).
详细描述
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:
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
盲法说明
Participants will not be informed until the end of study whether they received active intervention or sham intervention
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.)
排除标准
- •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)
结局指标
主要结局
Global Swallowing Function
时间窗: 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.
次要结局
- 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.)
- 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.)
- Biomarker analyses(Baseline measurement (week 0))