Gainesville, Florida 32610


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).

Study summary:

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: 1. Forced expiratory tests 2. Maximal Inspiratory Pressure (MIP) 3. Resting Breathing Pattern 4. Phrenic Nerve Function Evaluation with EMG 5. Severe Respiratory Insufficiency Questionnaire 6. Magnetic stimulation 7. Diaphragm conditioning consisting of: 1. The external stimulator settings 2. The duration of diaphragm stimulation 3. Off-ventilator spontaneous breathing (SB)


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



Primary Contact:

Principal Investigator
Barbara K Smith, PT, PhD
University of Florida

Barbara K Smith, PT, PhD
Phone: 352-294-5315

Backup Contact:


Location Contact:

Gainesville, Florida 32610
United States

Barbara K Smith, PT, PhD
Phone: 352-294-5315

Site Status: Recruiting

Data Source:

Date Processed: March 16, 2018

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