Respiratory Muscle Training in Late-Onset Pompe Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 2
- 主要终点
- Change in respiratory strength
研究概览
简要总结
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles. The results of this study will help design future research studies about RMT in LOPD. The goals of this study are to decide if sham-RMT is a useful control condition for RMT and to choose the best ways to measure the health benefits of RMT in LOPD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Double (Participant, Investigator)
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
- •Diagnosis of LOPD
- •On enzyme replacement therapy for ≥ 26 weeks at pretest
- •Able to follow directions for study participation
- •Able to complete a home-based RMT regimen
排除标准
- •Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic condition that would prevent meaningful study participation as determined at the discretion of the principle investigator
- •Inability to give legally effective consent
- •Inability to read and understand English
研究组 & 干预措施
Respiratory muscle training (RMT)
Subjects in the experimental arm will be given an inspiratory and expiratory RMT device to use during Duke-based and home-based RMT therapy.
干预措施: RMT therapy using modified RMT device (Device)
Sham-RMT
Subjects in control arm will be given an inspiratory and expiratory sham-device and will complete Duke-based and home-based sham-RMT therapy.
干预措施: Sham-RMT therapy using modified RMT device (Device)
结局指标
主要结局
Change in respiratory strength
时间窗: baseline, 12 weeks, 6 months, 9 months
RMT to sham-RMT comparison of maximum inspiratory pressures (MIP). Change pretest to posttest is primary measure (baseline to 12 weeks).
Feasibility of sham-RMT, as measured by program adherence
时间窗: 12 weeks
As measured by program adherence (good is considered greater than or equal to 80%). Helps determines if a future efficacy trial is warranted and if sham-RMT may be a good control condition for a future trial.
次要结局
- Change in maximum expiratory pressures (MEP)(baseline, 12 weeks, 6 months, 9 months)
- Change in 6-minute walk test (6MWT)(baseline, 12 weeks, 6 months, 9 months)
- Change in Gait, Stairs, Gower, and Chair (GSGC) scale(baseline, 12 weeks, 6 months, 9 months)
- Change in peak cough flow (PCF)(baseline, 12 weeks, 6 months, 9 months)
- Change in Rasch-built Pompe-specific Activity Score (R-PAct)(baseline, 12 weeks, 6 months, 9 months)
- Change in diaphragm thickness(baseline, 12 weeks, 6 months, 9 months)