N/A
Completed N=9
Response to Diaphragmatic Pacing in Subjects With Pompe Disease
Pompe Disease
Source: ClinicalTrials.gov NCT02354651 ↗
Enrolled (actual)
9
Serious AEs
50.0%
Results posted
Jun 2023
Primary outcomePrimary: Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180 — 0.52; 4.72 hours per day
Summary
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180 |
0.52; 4.72 | — |
| SECONDARY Maximal Inspiratory Pressure (MIP) at Baseline and Day 180 |
35.6; 32.7 | — |
| SECONDARY Change in Resting Breathing Pattern |
5.05; 5.41 | — |
| SECONDARY Diaphragm Electromyogram (EMG) at Day 180 |
57.3 | — |
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- Current participation in another treatment study (other than enzyme replacement therapy) directed at improving diaphragm function
- Unable to complete pulmonary function testing
Data sourced from ClinicalTrials.gov (NCT02354651). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.