High-dose Inspiratory Muscle Training (IMT) in Late-onset Pompe Disease (LOPD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 2
- 主要终点
- Change in maximum inspiratory pressure (MIP)
研究概览
简要总结
Study Objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and 2) determine its effects on respiratory and patient-reported outcomes.
详细描述
This study aims to develop treatments that enhance respiratory strength and function to provide meaningful clinical improvements for people with LOPD. Identification of a cost-effective adjunctive intervention to address respiratory weakness remains critical to reduce disease burden, ease activity limitations and participation restrictions, and improve health-related quality of life. The proposed study will provide a high-dose inspiratory muscle training (IMT) stimulus to enhance treatment efficacy and efficiency. Our hypothesis is that high-dose IMT is necessary to produce meaningful changes in respiratory muscle strength and other outcomes in participants with LOPD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
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
- •Age ≥ 18 years
- •Confirmed diagnosis of LOPD
- •MIP >50% of predicted for sex and age
- •Stable on current Pompe disease treatment regimen >6 months
- •Able to follow directions for study participation
- •Access to computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies
排除标准
- •Presence of medical comorbidities that prevent meaningful study participation (e.g., COPD GOLD III-IV, significant mental illness, dementia)
- •Use of continuous invasive or non-invasive ventilation while awake
- •Prior history of gene therapy for LOPD
- •Inability to give legally effective consent
- •Inability to read and understand English
结局指标
主要结局
Change in maximum inspiratory pressure (MIP)
时间窗: Baseline, week 15, week 30
Change pre-test to post test, measured in cm H20
次要结局
- Change in peak expiratory flow (PEF)(Baseline, week 15, week 30)
- Change in inspiratory phase duration (IPD)(Baseline, week 15, week 30)
- Change in compression phase duration (CPD)(Baseline, week 15, week 30)
- Change in forced vital capacity (FVC)(Baseline, week 15, week 30)
- Change in impact of fatigue on quality of life (QOL)(Baseline, week 15, week 30)
- Change in voice quality(Baseline, week 15, week 30)
- Change in forced expiratory volume over 1 second (FEV1)(Baseline, week 15, week 30)
- Change in maximum expiratory pressure (MEP)(Baseline, week 15, week 30)
- Change in inspiratory peak flow (IPF)(Baseline, week 15, week 30)
- Change in expiratory phase rise time (EPRT)(Baseline, week 15, week 30)
- Change in fatigue(Baseline, week 15, week 30)
- Change in daytime sleepiness(Baseline, week 15, week 30)
- Change in respiratory symptoms(Baseline, week 15, week 30)
- Change in ability to communicate(Baseline, week 15, week 30)
- Change in cough volume acceleration (CVA)(Baseline, week 15, week 30)
- Change in fatigue index test score (FIT)(Baseline, week 15, week 30)
- Change in sleep quality(Baseline, week 15, week 30)
- Change in motor performance(Baseline, week 15, week 30)
- Change in health-related quality of life(Baseline, week 15, week 30)
- Change in swallowing symptoms(Baseline, week 15, week 30)
- Change in inspiratory power curve (IPC)(Baseline, week 15, week 30)
- Change in inspiratory duration (ID)(Baseline, week 15, week 30)