Ignite Creation Date:
2025-12-24 @ 5:16 PM
Ignite Modification Date:
2026-02-18 @ 1:22 PM
Study NCT ID:
NCT02834650
Status:
COMPLETED
Last Update Posted:
2022-05-10
First Post:
2016-07-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003287', 'term': 'Contrast Media'}, {'id': 'D006403', 'term': 'Hematologic Tests'}, {'id': 'D006339', 'term': 'Heart Rate'}, {'id': 'D012143', 'term': 'Respiratory Physiological Phenomena'}, {'id': 'D005820', 'term': 'Genetic Testing'}], 'ancestors': [{'id': 'D064907', 'term': 'Diagnostic Uses of Chemicals'}, {'id': 'D020228', 'term': 'Pharmacologic Actions'}, {'id': 'D020164', 'term': 'Chemical Actions and Uses'}, {'id': 'D020313', 'term': 'Specialty Uses of Chemicals'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D055986', 'term': 'Vital Signs'}, {'id': 'D010808', 'term': 'Physical Examination'}, {'id': 'D006439', 'term': 'Hemodynamics'}, {'id': 'D002320', 'term': 'Cardiovascular Physiological Phenomena'}, {'id': 'D002943', 'term': 'Circulatory and Respiratory Physiological Phenomena'}, {'id': 'D005821', 'term': 'Genetic Techniques'}, {'id': 'D033142', 'term': 'Genetic Services'}, {'id': 'D006296', 'term': 'Health Services'}, {'id': 'D005159', 'term': 'Health Care Facilities Workforce and Services'}, {'id': 'D003954', 'term': 'Diagnostic Services'}, {'id': 'D011314', 'term': 'Preventive Health Services'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'BASIC_SCIENCE', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 89}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-02-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-05', 'completionDateStruct': {'date': '2022-03-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-05-04', 'studyFirstSubmitDate': '2016-07-13', 'studyFirstSubmitQcDate': '2016-07-13', 'lastUpdatePostDateStruct': {'date': '2022-05-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-07-15', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2022-03-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Myocardial Tissue Characterization', 'timeFrame': '6 months', 'description': 'Focal and diffuse fibrosis, intra myocardial fat, edema plus water mobility'}, {'measure': 'Myocardial Functional Characterization', 'timeFrame': '6 months', 'description': 'Strain imaging and rotational mechanics'}, {'measure': 'Genomic Analysis', 'timeFrame': '4 years', 'description': 'Proposing mechanisms of cardiac dysfunction or protective phenotypes using genomic analysis'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Muscular Dystrophy, Duchenne']}, 'descriptionModule': {'briefSummary': 'This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD.\n\nCurrently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD.\n\nConsequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.', 'detailedDescription': 'The second objective is to use their well-characterized cardiac MRI biomarkers and define their sensitivity for detecting early cardiac involvement. The final objective is to use these validated cardiac MRI biomarkers to better understand the genotype-phenotype correlation in boys with DMD, which to date remain tenuous. The investigators propose a pilot study to explore cardiac genotype-phenotype correlations in boys with DMD and outlier phenotypes using approaches they have pioneered for skeletal muscle.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '7 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Healthy boys or pediatric patients with DMD age 7 to 21\n* Able \\& willing to complete an approximately 75-minute (or less) MRI exam without sedation or mechanical ventilation\n* Drug regimen (if applicable) stable for at least 3 months prior to participation\n\nExclusion Criteria:\n\n* Renal insufficiency (GFR\\<40 mL/min/m2)\n* Non-MRI compatible implants (e.g. neurostimulator, pacemaker, implanted cardioverter defibrillator)\n* Claustrophobia that prevents an MRI exam\n* Known allergy to MRI contrast agents\n* Serum potassium level of \\>5.0 mmol/L\n* Signs and symptoms of heart failure'}, 'identificationModule': {'nctId': 'NCT02834650', 'briefTitle': 'Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD', 'organization': {'class': 'OTHER', 'fullName': 'Stanford University'}, 'officialTitle': 'Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for Duchenne Muscular Dystrophy (DMD)', 'orgStudyIdInfo': {'id': 'DMD2016'}, 'secondaryIdInfos': [{'id': '1R01HL131975-01', 'link': 'https://reporter.nih.gov/quickSearch/1R01HL131975-01', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group 1a', 'description': "Group 1a comprises healthy volunteers who will complete a Cardiac MRI without contrast. A subset of healthy volunteers will have a repeat MRI at Children's Hospital of Orange County.", 'interventionNames': ['Other: Cardiac MRI without contrast', 'Other: Repeat MRI scan']}, {'type': 'EXPERIMENTAL', 'label': 'Group 1b', 'description': "Group 1b comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test.\n\nA subset of boys with DMD will have a repeat MRI with contrast at Children's Hospital of Orange County.", 'interventionNames': ['Other: Cardiac MRI with contrast', 'Other: Blood Test', 'Other: Heart Rate', 'Other: Pulmonary Function Test', 'Other: Genetic Testing', 'Other: Repeat MRI scan']}, {'type': 'EXPERIMENTAL', 'label': 'Group 2', 'description': 'Group 2 comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test and a repeat MRI scan with contrast at 6 Months.', 'interventionNames': ['Other: Cardiac MRI with contrast', 'Other: Blood Test', 'Other: Heart Rate', 'Other: Pulmonary Function Test', 'Other: Genetic Testing', 'Other: Repeat MRI scan']}, {'type': 'EXPERIMENTAL', 'label': 'Group 3', 'description': 'Group 3 comprises boys with DMD who will complete a Cardiac MRI with contrast, a blood test, a heart rate test and a pulmonary function test and a genetic testing.', 'interventionNames': ['Other: Cardiac MRI with contrast', 'Other: Blood Test', 'Other: Heart Rate', 'Other: Pulmonary Function Test', 'Other: Genetic Testing']}], 'interventions': [{'name': 'Cardiac MRI with contrast', 'type': 'OTHER', 'description': 'Cardiac MRI', 'armGroupLabels': ['Group 1b', 'Group 2', 'Group 3']}, {'name': 'Cardiac MRI without contrast', 'type': 'OTHER', 'description': 'Cardiac MRI', 'armGroupLabels': ['Group 1a']}, {'name': 'Blood Test', 'type': 'OTHER', 'description': 'Hematocrit, Creatinine, Troponin, BNP', 'armGroupLabels': ['Group 1b', 'Group 2', 'Group 3']}, {'name': 'Heart Rate', 'type': 'OTHER', 'description': 'Holter Monitor', 'armGroupLabels': ['Group 1b', 'Group 2', 'Group 3']}, {'name': 'Pulmonary Function Test', 'type': 'OTHER', 'description': 'Pulmonary Function Test', 'armGroupLabels': ['Group 1b', 'Group 2', 'Group 3']}, {'name': 'Genetic Testing', 'type': 'OTHER', 'description': 'Genetic Testing', 'armGroupLabels': ['Group 1b', 'Group 2', 'Group 3']}, {'name': 'Repeat MRI scan', 'type': 'OTHER', 'description': 'Repeat MRI scan', 'armGroupLabels': ['Group 1a', 'Group 1b', 'Group 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90024', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Orange County", 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}], 'overallOfficials': [{'name': 'Daniel Ennis, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Stanford University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Stanford University', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institutes of Health (NIH)', 'class': 'NIH'}, {'name': "Children's Hospital of Orange County", 'class': 'OTHER'}, {'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}, {'name': 'University of California, Los Angeles', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Daniel Ennis', 'investigatorAffiliation': 'Stanford University'}}}}