Response to Diaphragmatic Pacing in Subjects With Pompe Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180
研究概览
简要总结
The purpose of this study is to determine the effect of diaphragm pacing on respiratory function in subjects with Pompe disease by evaluating the duration and pattern of spontaneous respiratory function versus paced ventilation respiratory function in patients with Pompe disease who have received the NeuDx Diaphragm pacer (DPS).
详细描述
Subjects with Pompe disease who are scheduled to receive a NeuRx Diaphragm pacer (DPS) will be invited to participate in this study. The following tests will be performed during participation in the study:
-
Forced expiratory tests
-
Maximal Inspiratory Pressure (MIP)
-
Resting Breathing Pattern
-
Phrenic Nerve Function Evaluation with EMG
-
Severe Respiratory Insufficiency Questionnaire
-
Magnetic stimulation
-
Diaphragm conditioning consisting of:
-
The external stimulator settings
-
The duration of diaphragm stimulation
-
Off-ventilator spontaneous breathing (SB)
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Pompe disease as defined by mutational analysis, GAA enzyme activity assay in blood spot, and/or fibroblast culture less than 40% of control value
- •Medically and functionally eligible for NeuRx DPS implantation, as determined by a clinical surgical evaluation. The surgeon will determine eligibility through pulmonary function tests, ability to stimulate your diaphragm, and arterial blood gas levels. Eligibility may also be determined by your response to traditional respiratory strengthening exercises without a pacemaker.
- •History of mechanical ventilation dependence (six or more hours of invasive or non-invasive mechanical ventilation support daily for at least 21 days in duration
排除标准
- •Current participation in another treatment study (other than enzyme replacement therapy) directed at improving diaphragm function
- •Unable to complete pulmonary function testing
结局指标
主要结局
Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180
时间窗: Baseline, Day 180
The patient will undergo an initial trial of unassisted breathing with the diaphragm pacer turned on. The duration of SB with pacing will be increased to tolerance, using a protocol. If the patient cannot initially complete at least 30-minute bouts of paced SB, then the focus of breathing trials will be to breathe with reduced support from the mechanical ventilator. These measures were generated utilizing ventilator chip data for SB that has been averaged.
次要结局
- Maximal Inspiratory Pressure (MIP) at Baseline and Day 180(Baseline, Day 180)
- Diaphragm Electromyogram (EMG) at Day 180(Day 180)
- Change in Resting Breathing Pattern(Day 180)