Ignite Creation Date:
2025-12-24 @ 11:51 PM
Ignite Modification Date:
2026-06-16 @ 7:43 PM
Study NCT ID:
NCT02354651
Status:
COMPLETED
Last Update Posted:
2023-08-01
First Post:
2015-01-21
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Response to Diaphragmatic Pacing in Subjects With Pompe Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006009', 'term': 'Glycogen Storage Disease Type II'}], 'ancestors': [{'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D006008', 'term': 'Glycogen Storage Disease'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'bksmith@ufl.edu', 'phone': '3522945315', 'title': 'Dr. Barbara Smith', 'organization': 'University of Florida'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'This was a pilot observational study that tracked an unusual clinical intervention in a rare disease; thus, generalizability is difficult to ascertain. Consistent measurement times were difficult to access some patients who resided great distances from the study center.'}}, 'adverseEventsModule': {'timeFrame': 'Longitudinal observational data through January 2019. Post-operative observation time frame ranged from 89 months in subject 1, to 45 months in subject 6..', 'eventGroups': [{'id': 'EG000', 'title': 'Pompe Subjects Receiving NeuRx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team.", 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 6, 'seriousNumAtRisk': 6, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Skin irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Respiratory infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Occipital pressure ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Damaged infusion port', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Product Issues', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Breakage of pacemaker external wires', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'Product Issues', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Scoliosis requiring spinal fusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Seizures', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pompe Subjects Receiving NeuRx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '0.52', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '1.5'}]}]}, {'title': 'Day 180', 'categories': [{'measurements': [{'value': '4.72', 'groupId': 'OG000', 'lowerLimit': '.04', 'upperLimit': '12'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180', 'description': '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.', 'unitOfMeasure': 'hours per day', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Full sample'}, {'type': 'SECONDARY', 'title': 'Maximal Inspiratory Pressure (MIP) at Baseline and Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pompe Subjects Receiving NeuRx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '35.6', 'groupId': 'OG000', 'lowerLimit': '12.8', 'upperLimit': '65'}]}]}, {'title': '180 Days', 'categories': [{'measurements': [{'value': '32.7', 'groupId': 'OG000', 'lowerLimit': '8.3', 'upperLimit': '61'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 180', 'description': 'MIP will be measured with a manometer connected to the airway opening of the subject, which occludes the airway and prevents airflow. A nose clip will be used to prevent leakage and the subject is instructed to suck air from the tube with as much force as possible. Measurements will be obtained with the subject sitting upright. Transdiaphragmatic pressure (Pdi) may be calculated with esophageal and gastric manometry during MIP. The measurement will be taken three times and the best value will be reported.', 'unitOfMeasure': 'cm H2O', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Subjects 4 and 6 were unavailable for MIP testing at baseline; Subjects 1 and 3 were unavailable for MIP testing at 6 months.'}, {'type': 'SECONDARY', 'title': 'Change in Resting Breathing Pattern', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pompe Subjects Receiving NeuRx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'classes': [{'title': 'Without diaphragm pacer', 'categories': [{'measurements': [{'value': '5.05', 'spread': '1.73', 'groupId': 'OG000'}]}]}, {'title': 'With diaphragm pacer', 'categories': [{'measurements': [{'value': '5.41', 'spread': '3.54', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 180', 'description': 'Resting ventilatory flow and timing will be recorded with a pneumotachograph and pressure transducer connected to the airway opening, to calculate minute ventilation. If a patient routinely uses MV throughout the day, the sensing equipment can be placed in series to the breathing tubes. Breathing pattern will be measured for a minimum of ten minutes.', 'unitOfMeasure': 'L/min', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Minute ventilation during off-ventilator breathing was assessed with and without diaphragm pacing at approximately 6 months post-implant.'}, {'type': 'SECONDARY', 'title': 'Diaphragm Electromyogram (EMG) at Day 180', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pompe Subjects Receiving NeuRx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'classes': [{'categories': [{'measurements': [{'value': '57.3', 'spread': '38.9', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 180', 'description': 'This activity reflects the activity of the diaphragm and phrenic nerve. The wires that are implanted for diaphragm pacemaker will be connected to a computer and recording equipment to monitor the activity of the diaphragm muscle during breathing at the lowest level of ventilator assistance. This EMG data is measured in units of microvolts per second and expressed as a percentage of the level of diaphragm EMG activity recorded during MIP maneuvers.', 'unitOfMeasure': 'RMS diaphragm (% of MIP)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Hours Per Day of Diaphragm Pacing', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Pompe Disease Eligible for Diaphragm Pacing', 'description': "Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm conditioning will be prescribed by the patient's clinical team."}], 'classes': [{'categories': [{'measurements': [{'value': '20.3', 'groupId': 'OG000', 'lowerLimit': '2', 'upperLimit': '24'}]}]}], 'paramType': 'MEAN', 'timeFrame': '180 days', 'description': 'Number of hours used per day diaphragm pacing occurred by subjects', 'unitOfMeasure': 'hours per day of diaphragm pacing', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients with Pompe disease who received diaphragm pacing'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Magnetic Stimulation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Pompe Disease Eligible for Diaphragm Pacing', 'description': "Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'timeFrame': '180 Days', 'description': 'EMG response to supramaximal bilateral stimulation of the phrenic nerves.', 'reportingStatus': 'POSTED', 'populationDescription': 'Magnetic stimulation was used as assessment for determining clinical eligibility for diaphragm pacing. It was not available at follow-up observational visits.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Severe Respiratory Insufficiency Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Patients With Pompe Disease Eligible for Diaphragm Pacing', 'description': "Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm conditioning will be prescribed by the patient's clinical team."}], 'timeFrame': '180 days', 'description': 'Patient reported measure of impact of respiratory insufficiency on functional mobility', 'reportingStatus': 'POSTED', 'populationDescription': 'The Severe Respiratory Insufficiency Questionnaire was validated only in adults. Adults who participated did not complete the questionnaire at Day 180.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Forced Expiratory Flow', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pompe Subjects Receiving NeuDx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'classes': [{'categories': [{'measurements': [{'value': '42.65', 'groupId': 'OG000', 'lowerLimit': '36.4', 'upperLimit': '48.9'}]}]}], 'paramType': 'MEAN', 'timeFrame': '180 days', 'description': 'Peak expiratory flow obtained during voluntary cough', 'unitOfMeasure': 'L/min', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Only 2 patients were able to complete voluntary cough during a 180-day visit'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Patients With Pompe Disease Eligible for Diaphragm Pacing', 'description': "Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm conditioning will be prescribed by the patient's clinical team."}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Subjects were eligible to receive a diaphragmatic pacemaker as part of their clinical care. Some subjects who enrolled decided not to include pacing as part of their clinical care (n=2), or were found to have medical conditions that made them ineligible (n=1).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Pompe Subjects Receiving NeuDx DPS', 'description': "Patients will receive resting breathing pattern evaluation, phrenic nerve stimulation, maximal inspiratory pressure (MIP) testing, forced expiratory testing, and EMG.\n\nDiaphragm conditioning via phrenic nerve stimulation: Diaphragm pacing will be prescribed by the patient's clinical team."}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '24.5', 'groupId': 'BG000', 'lowerLimit': '3', 'upperLimit': '53'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ventilator-Free Breathing', 'classes': [{'categories': [{'measurements': [{'value': '0.52', 'groupId': 'BG000', 'lowerLimit': '0', 'upperLimit': '1.5'}]}]}], 'paramType': 'MEAN', 'description': 'Hours of daily ventilation unassisted by positive pressure ventilation.', 'unitOfMeasure': 'hours of unassisted ventilation per day', 'dispersionType': 'FULL_RANGE'}], 'populationDescription': 'Participants who underwent implantation of a diaphragm pacing system.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-12-05', 'size': 321411, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-03-17T16:59', 'hasProtocol': True}, {'date': '2017-02-15', 'size': 130851, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-04-13T13:52', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-07', 'completionDateStruct': {'date': '2023-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-07-28', 'studyFirstSubmitDate': '2015-01-21', 'resultsFirstSubmitDate': '2023-03-20', 'studyFirstSubmitQcDate': '2015-01-29', 'lastUpdatePostDateStruct': {'date': '2023-08-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-05-08', 'studyFirstPostDateStruct': {'date': '2015-02-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2023-06-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Hours Per Day of Diaphragm Pacing', 'timeFrame': '180 days', 'description': 'Number of hours used per day diaphragm pacing occurred by subjects'}, {'measure': 'Magnetic Stimulation', 'timeFrame': '180 Days', 'description': 'EMG response to supramaximal bilateral stimulation of the phrenic nerves.'}, {'measure': 'Severe Respiratory Insufficiency Questionnaire', 'timeFrame': '180 days', 'description': 'Patient reported measure of impact of respiratory insufficiency on functional mobility'}, {'measure': 'Forced Expiratory Flow', 'timeFrame': '180 days', 'description': 'Peak expiratory flow obtained during voluntary cough'}], 'primaryOutcomes': [{'measure': 'Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180', 'timeFrame': 'Baseline, Day 180', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Maximal Inspiratory Pressure (MIP) at Baseline and Day 180', 'timeFrame': 'Baseline, Day 180', 'description': 'MIP will be measured with a manometer connected to the airway opening of the subject, which occludes the airway and prevents airflow. A nose clip will be used to prevent leakage and the subject is instructed to suck air from the tube with as much force as possible. Measurements will be obtained with the subject sitting upright. Transdiaphragmatic pressure (Pdi) may be calculated with esophageal and gastric manometry during MIP. The measurement will be taken three times and the best value will be reported.'}, {'measure': 'Change in Resting Breathing Pattern', 'timeFrame': 'Day 180', 'description': 'Resting ventilatory flow and timing will be recorded with a pneumotachograph and pressure transducer connected to the airway opening, to calculate minute ventilation. If a patient routinely uses MV throughout the day, the sensing equipment can be placed in series to the breathing tubes. Breathing pattern will be measured for a minimum of ten minutes.'}, {'measure': 'Diaphragm Electromyogram (EMG) at Day 180', 'timeFrame': 'Day 180', 'description': 'This activity reflects the activity of the diaphragm and phrenic nerve. The wires that are implanted for diaphragm pacemaker will be connected to a computer and recording equipment to monitor the activity of the diaphragm muscle during breathing at the lowest level of ventilator assistance. This EMG data is measured in units of microvolts per second and expressed as a percentage of the level of diaphragm EMG activity recorded during MIP maneuvers.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Pompe disease', 'mechanical ventilation', 'diaphragm pacing'], 'conditions': ['Pompe Disease']}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': '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:\n\n1. Forced expiratory tests\n2. Maximal Inspiratory Pressure (MIP)\n3. Resting Breathing Pattern\n4. Phrenic Nerve Function Evaluation with EMG\n5. Severe Respiratory Insufficiency Questionnaire\n6. Magnetic stimulation\n7. Diaphragm conditioning consisting of:\n\n 1. The external stimulator settings\n 2. The duration of diaphragm stimulation\n 3. Off-ventilator spontaneous breathing (SB)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects with Pompe disease who are eligible for NeuRx DPS implantation', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 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\n* 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.\n* 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\n\nExclusion Criteria:\n\n* Current participation in another treatment study (other than enzyme replacement therapy) directed at improving diaphragm function\n* Unable to complete pulmonary function testing'}, 'identificationModule': {'nctId': 'NCT02354651', 'briefTitle': 'Response to Diaphragmatic Pacing in Subjects With Pompe Disease', 'organization': {'class': 'OTHER', 'fullName': 'University of Florida'}, 'officialTitle': 'Response to Diaphragmatic Pacing in Subjects With Pompe Disease', 'orgStudyIdInfo': {'id': 'IRB201600161-N'}, 'secondaryIdInfos': [{'id': '003-2013', 'type': 'OTHER', 'domain': 'University of Florida pre-conversion'}, {'id': '1R21HD090752-01', 'link': 'https://reporter.nih.gov/quickSearch/1R21HD090752-01', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Patients With Pompe Disease Eligible for Diaphragm Pacing', 'description': 'Patients will receive tests of breathing pattern, phrenic nerve stimulation, maximal inspiratory pressure (MIP), forced expiration, and EMG.', 'interventionNames': ['Other: Diaphragm conditioning via phrenic nerve stimulation']}], 'interventions': [{'name': 'Diaphragm conditioning via phrenic nerve stimulation', 'type': 'OTHER', 'description': "Diaphragm pacing will be prescribed by the patient's clinical team.", 'armGroupLabels': ['Patients With Pompe Disease Eligible for Diaphragm Pacing']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}], 'overallOfficials': [{'name': 'Barbara K Smith, PT, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Florida'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Florida', 'class': 'OTHER'}, 'collaborators': [{'name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR'}}}}