Ignite Creation Date:
2025-12-24 @ 6:43 PM
Ignite Modification Date:
2025-12-24 @ 6:43 PM
Study NCT ID:
NCT03985657
Status:
COMPLETED
Last Update Posted:
2020-12-28
First Post:
2019-06-11
Is Possible Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Hemodynamics Response to Upper Airway Obstruction in Marfan Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012891', 'term': 'Sleep Apnea Syndromes'}, {'id': 'D012913', 'term': 'Snoring'}], 'ancestors': [{'id': 'D001049', 'term': 'Apnea'}, {'id': 'D012120', 'term': 'Respiration Disorders'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D020919', 'term': 'Sleep Disorders, Intrinsic'}, {'id': 'D020920', 'term': 'Dyssomnias'}, {'id': 'D012893', 'term': 'Sleep Wake Disorders'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012135', 'term': 'Respiratory Sounds'}, {'id': 'D012818', 'term': 'Signs and Symptoms, Respiratory'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'msowho1@jhmi.edu', 'phone': '4105506264', 'title': 'Mudiaga Sowho', 'organization': 'Johns Hopkins University'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Over the two night sessions.', 'eventGroups': [{'id': 'EG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography involved the monitoring of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.', 'otherNumAtRisk': 30, 'deathsNumAtRisk': 30, 'otherNumAffected': 0, 'seriousNumAtRisk': 30, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'CPAP Sleep Study', 'description': 'Participants were treated with continuous positive airway pressure (CPAP) on a separate night. Room air at pressures between 6-9 centimeters of water (cmH2O) were delivered via heated humidified tubing and a nasal mask.', 'otherNumAtRisk': 31, 'deathsNumAtRisk': 31, 'otherNumAffected': 0, 'seriousNumAtRisk': 31, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Pleural Pressure (Pes)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.'}, {'id': 'OG001', 'title': 'CPAP Sleep Study', 'description': 'Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.'}], 'classes': [{'categories': [{'measurements': [{'value': '9.3', 'spread': '3.4', 'groupId': 'OG000'}, {'value': '4.4', 'spread': '1.6', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Overnight on both CPAP and No CPAP nights', 'description': 'Pleural pressure (Pes) in mmHg monitored using an esophageal catheter.', 'unitOfMeasure': 'mmHg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with upper airway obstruction (apneas, hypopneas, and flow limitation) during the Baseline sleep study, who had pleural pressure and blood pressure data collected during both Baseline and CPAP sleep studies.'}, {'type': 'PRIMARY', 'title': 'Mean Arterial Blood Pressure (MAP)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.'}, {'id': 'OG001', 'title': 'CPAP Sleep Study', 'description': 'Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.'}], 'classes': [{'categories': [{'measurements': [{'value': '93.8', 'spread': '14.0', 'groupId': 'OG000'}, {'value': '88.3', 'spread': '12.4', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Overnight on both Baseline and CPAP studies', 'description': 'Continuous blood pressure monitored using a non-invasive finger cuff. Data captured in mmHg.', 'unitOfMeasure': 'mmHg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with upper airway obstruction (apneas, hypopneas, and flow limitation) during the Baseline sleep study, who had pleural pressure and blood pressure data collected during both Baseline and CPAP sleep studies.'}, {'type': 'SECONDARY', 'title': 'Augmentation Index (AI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.'}, {'id': 'OG001', 'title': 'CPAP Sleep Study', 'description': 'Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.'}], 'classes': [{'categories': [{'measurements': [{'value': '11.0', 'spread': '3.8', 'groupId': 'OG000'}, {'value': '2.4', 'spread': '3.7', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '15 minutes in the morning post Baseline and CPAP studies', 'description': 'The AI (measured as "percentage of pulse pressure") was assessed in the morning after both Baseline and CPAP studies. It is a measure of arterial stiffness that represents the degree of and can range from -10% to +10% in healthy individuals, with values generally higher in females and increases with age.', 'unitOfMeasure': 'percentage of pulse pressure', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with adequate sleep time (\\>2hrs) on both Baseline and CPAP studies and AI measurements after both Baseline and CPAP studies.'}, {'type': 'SECONDARY', 'title': 'Reactive Hyperemia Index (RHI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP.'}, {'id': 'OG001', 'title': 'CPAP Sleep Study', 'description': 'Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. CPAP: Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.2', 'spread': '0.1', 'groupId': 'OG000'}, {'value': '2.2', 'spread': '0.2', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '15 minutes', 'description': 'The RHI is a measure of endothelial function that was measured in the morning after both Baseline and CPAP studies. It is unitless and scored on a range of 1 to 3 in healthy individuals with lower values indicating poor endothelial function.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants with adequate sleep time (\\>2hrs) on both Baseline and CPAP studies and morning RHI measurements after both Baseline and CPAP studies.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography involved the monitoring of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP. Participants in this arm would switch to CPAP within one week of the Baseline Sleep Study.'}, {'id': 'FG001', 'title': 'CPAP Sleep Study', 'description': 'In a randomized cross-over fashion, participants were treated with continuous positive airway pressure (CPAP) on a separate night. Room air at pressures between 6-9 centimeters of water (cmH2O) were delivered via heated humidified tubing and a nasal mask. Participants in this arm would switch to Baseline Sleep Study within one week of the CPAP Sleep Study.'}], 'periods': [{'title': 'Intervention 1 (One Night)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '11'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Washout (Up to 1 Week)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Intervention 2 (One Night)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': '193 persons with Marfan syndrome were screened. Persons were recruited from the Johns Hopkins Marfan Clinic and the external Marfan Community via the Marfan foundation. Inclusion was based on a clinical diagnosis of Marfan syndrome (Ghent Criteria) or genetic testing. Persons were excluded if they had any chronic lung disease, other sleep disorders aside from sleep apnea, were on blood thinners, or had uncontrolled blood pressure. Recruitment occurred between 09/2018 and 11/2019.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants', 'description': 'All participants enrolled in this study.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '37.5', 'spread': '15.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '17', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-12-16', 'size': 334945, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-11-27T16:59', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER', 'interventionModelDescription': 'Participants will be assigned to a Baseline polysomnography and CPAP polysomnography in a randomized fashion. Participants will then be switched to the second polysomnography within 14 days of the first. The baseline polysomnography represents the exposure to sleep disordered breathing and the CPAP polysomnography represents the relief of the exposure. Markers of hemodynamic stress will be assessed in the morning after both studies.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 31}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-06-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-12', 'completionDateStruct': {'date': '2019-12-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-12-02', 'studyFirstSubmitDate': '2019-06-11', 'resultsFirstSubmitDate': '2020-12-02', 'studyFirstSubmitQcDate': '2019-06-11', 'lastUpdatePostDateStruct': {'date': '2020-12-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-12-02', 'studyFirstPostDateStruct': {'date': '2019-06-14', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2020-12-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-12-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Pleural Pressure (Pes)', 'timeFrame': 'Overnight on both CPAP and No CPAP nights', 'description': 'Pleural pressure (Pes) in mmHg monitored using an esophageal catheter.'}, {'measure': 'Mean Arterial Blood Pressure (MAP)', 'timeFrame': 'Overnight on both Baseline and CPAP studies', 'description': 'Continuous blood pressure monitored using a non-invasive finger cuff. Data captured in mmHg.'}], 'secondaryOutcomes': [{'measure': 'Augmentation Index (AI)', 'timeFrame': '15 minutes in the morning post Baseline and CPAP studies', 'description': 'The AI (measured as "percentage of pulse pressure") was assessed in the morning after both Baseline and CPAP studies. It is a measure of arterial stiffness that represents the degree of and can range from -10% to +10% in healthy individuals, with values generally higher in females and increases with age.'}, {'measure': 'Reactive Hyperemia Index (RHI)', 'timeFrame': '15 minutes', 'description': 'The RHI is a measure of endothelial function that was measured in the morning after both Baseline and CPAP studies. It is unitless and scored on a range of 1 to 3 in healthy individuals with lower values indicating poor endothelial function.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': True}, 'conditionsModule': {'conditions': ['Sleep-disordered Breathing', 'Snoring']}, 'descriptionModule': {'briefSummary': 'Upper airway obstruction (UAO) is an unrecognized source of hemodynamic stress that may contribute to aortic adverse events in persons with Marfan Syndrome (MFS). UAO occurs during snoring and sleep apnea and is characterized by repetitive partial or complete obstruction of the upper airway during sleep. These obstructive breathing events lead to intermittent surges in blood pressure (BP) REF and large decreases in pleural pressure (Pes), thereby increasing the trans-mural aortic pressure (TMP) and imposing mechanical stress on the aorta during sleep. Although UAO is known to increase mechanical stress on the aorta, the magnitude of the increase is not known for persons with MFS.\n\nIn this project, therefore, the investigators will also examine the changes in Pes and BP responses in periods of obstructed breathing and compare the diurnal markers or vascular stress between Baseline and CPAP studies in MFS persons.', 'detailedDescription': 'Research Objective/Significance:\n\nSpecific Aim 1a: To quantify the Pes and BP during periods with and without UAO during sleep in persons with MFS.\n\nSpecific Aim 1b: To examine the effect of CPAP treatment of UAO on Pes and BP in MFS persons.\n\nSpecific Aim 2: To examine the effect of CPAP treatment of UAO on daytime markers of hemodynamic stress (augmentation index, reactive hyperemia index) in MFS persons.\n\nPrimary Outcomes:\n\n* Overnight measure of hemodynamic stress (blood pressure, pleural pressure swings)\n\nSecondary Outcomes:\n\n* Changes in diurnal markers of hemodynamic stress (augmentation index, reactive hyperemia index)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\nPeople with Marfan syndrome.\n\n* Age ≥ 18yrs\n* Able and willing to provide informed consent\n* Willing to sleep connected to research apparatus\n\nExclusion Criteria:\n\n* Unstable cardiovascular disease (CHF, myocardial infarction or revascularization procedures, and unstable arrhythmias)\n* Uncontrolled hypertension (BP \\> 190/110)\n* Underlying obstructive or other intrinsic lung disease\n* Renal failure on dialysis\n* Cirrhosis\n* Pregnancy'}, 'identificationModule': {'nctId': 'NCT03985657', 'acronym': 'MSB', 'briefTitle': 'Hemodynamics Response to Upper Airway Obstruction in Marfan Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Johns Hopkins University'}, 'officialTitle': 'Sleep Disordered Breathing in Marfan Syndrome: Susceptibility and Hemodynamics', 'orgStudyIdInfo': {'id': 'IRB00157403'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Baseline Sleep Study', 'description': 'Baseline sleep polysomnography will involve the collection of electroencephalogram, electromyogram, electrocardiogram, airflow, heart rate, blood pressure, and pleural pressure during sleep with no CPAP. Participants in this arm would switch to CPAP within one week of the study.', 'interventionNames': ['Device: CPAP']}, {'type': 'EXPERIMENTAL', 'label': 'CPAP Sleep Study', 'description': 'Participants will be treated with continuous positive airway pressure to relieve sleep-disordered breathing. Participants in this arm would switch to Baseline study within one week of the study.', 'interventionNames': ['Device: CPAP']}], 'interventions': [{'name': 'CPAP', 'type': 'DEVICE', 'description': 'Continuous positive airway pressure (CPAP). Room air at pressures between 6-8 centimeters of water (cmH2O) delivered via heated humidified tubing and a nasal mask.', 'armGroupLabels': ['Baseline Sleep Study', 'CPAP Sleep Study']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21224', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Mudiaga Sowho', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Johns Hopkins University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Johns Hopkins University', 'class': 'OTHER'}, 'collaborators': [{'name': 'The Marfan Foundation', 'class': 'OTHER'}, {'name': 'National Institutes of Health (NIH)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR'}}}}