Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

9 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-05-31

Study Completion Date

2023-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Effect PEEP on Diaphragm

NCT03539640

Diaphragm Disease

COMPLETED NA

Percutaneous Temporary Placement of a Transvenous Phrenic Nerve Stimulator for Diaphragm Pacing Using Jugular Access

NCT03659552

Ventilator-Induced Lung Injury

COMPLETED NA

Does Diaphragm Electrical Activity Monitoring Predict Extubation Success in Children?

NCT06163183

Diaphragm Electrical Activity Neurally Adjusted Ventilatory Assist Pediatrics

COMPLETED

Change In Diaphragmatic Thickness Predicts Extubation Success

NCT02656199

Mechanical Ventilation Complication

TERMINATED

Assesment of New Devices for the Diagnostic Evaluation of Diaphragmatic Dysfunction

NCT03463798

Diaphragmatic Paralysis Healthy Volunteers

COMPLETED

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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)

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pompe Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Patients With Pompe Disease Eligible for Diaphragm Pacing

Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.

Diaphragm conditioning via phrenic nerve stimulation

Intervention Type OTHER

Diaphragm pacing will be prescribed by the patient's clinical team.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Diaphragm conditioning via phrenic nerve stimulation

Diaphragm pacing will be prescribed by the patient's clinical team.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

2 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No