Ignite Creation Date:
2025-12-24 @ 5:37 PM
Ignite Modification Date:
2025-12-30 @ 4:25 PM
Study NCT ID:
NCT00430768
Status:
COMPLETED
Last Update Posted:
2016-12-15
First Post:
2007-01-31
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}], 'ancestors': [{'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013352', 'term': 'Subcutaneous Emphysema'}, {'id': 'D004646', 'term': 'Emphysema'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Terry.Flotte@umassmed.edu', 'phone': '(508) 856-2107', 'title': 'Dr. Terry Flotte', 'organization': 'UMASS Medical School'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Study results based on small number of subjects No statistical analysis.'}}, 'adverseEventsModule': {'timeFrame': '1 year active study; 4 years long term follow up', 'eventGroups': [{'id': 'EG000', 'title': 'Group 1 Low Dose', 'description': '6.9 x10e12 vector genomes\n\ne. Group 1 receives rAAV1-CB-hAAT 6.9 x10e12 vg (vector genomes)', 'otherNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Group 2 Middle Dose', 'description': '2.2 x 10e13 vector genomes\n\nGroup 2 receives rAAV1-CB-hAAT 2.1 x10e13 vg', 'otherNumAtRisk': 3, 'otherNumAffected': 2, 'seriousNumAtRisk': 3, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Group 3 High Dose', 'description': 'rAAV1-CB-hAAT 6.0 x10e13 vg\n\nGroup 3 receives rAAV1-CB-hAAT 6.0 x10e13 vg', 'otherNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Lacrimation increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site hematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Injection site warmth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Escherichia urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Herpes zoster', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Tooth infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Skin injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Alkaline phosphatase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Blood cholesterol increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Gamma-glutamyl transferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Anorexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Dry throat', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Nasal mucosal disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Rhinorrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Sinus Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Sinus headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Throat Irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Palpitations', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Pharyngoesophageal diverticulum', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Bronchitis', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Tooth infection', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Gastrointestinal anastomotic leak', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Road Traffic Accident', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Wrist Fracture', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Hematuria', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Prostate Cancer', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Chest Pain', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Cough', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Dyspnea', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Subcutaneous abscess', 'notes': 'Long Term Follow up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}], 'seriousEvents': [{'term': 'E. coli Epididymitis', 'notes': '69 y/o man low dose of study drug 14MAR2006. 6 Apr 2006 diagnosed with E coli epididymitis confirmed by testicular ultrasound and urine culture. Event considered not related to study agent.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}, {'term': 'Pharyngoesophageal diverticulum repair with complications', 'notes': 'Subject received study agent 14Mar2006.Surgical repair 20Jan2010 for pharyngoesophageal diverticulum.Hospitalized 27Jan2010 with GI anastomotic leak following surgery, treated with feeding tube, resolved, considered not related to study agent.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'vocabulary,MedDRA'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Adverse Events Possibly, Probably or Definitely Related to Study Drug', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Group 1 Low Dose'}, {'id': 'OG001', 'title': 'Group 2 Middle Dose'}, {'id': 'OG002', 'title': 'Group 3 High Dose'}], 'classes': [{'title': 'Number with one or more related AE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'injection site erythema mild', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Inject site hematoma mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Inject site induration mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Inject site pain mild', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Inject site swelling mild', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Injection site warmth', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'During 1 year after study agent administration', 'description': 'Adverse events considered possibly, probably or definitely related to study drug/study drug procedure Criteria to evaluate severity according to Attachment 2 of the Protocol\n\n1. Mild toxicity, usually transient, requiring no special treatment and generally not interfering with usual daily activities\n2. Moderate toxicity which may be ameliorated by simple therapeutic maneuvers, and impairs usual activities\n3. Severe toxicity which requires therapeutic intervention and interrupts usual activities. Hospitalization may or may not be required\n4. Life-threatening toxicity which requires hospitalization', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'subjects in the group reporting the event'}, {'type': 'SECONDARY', 'title': 'hAAT Expression in Blood Measured Using M-specific Allele ELISA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}, {'value': '1', 'groupId': 'OG003'}, {'value': '1', 'groupId': 'OG004'}, {'value': '1', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': '201 (Medium Dose)', 'description': 'Subject 201 M-specific AAT Level'}, {'id': 'OG001', 'title': '202 (Medium Dose)', 'description': 'Subject 202 M-specific AAT Level'}, {'id': 'OG002', 'title': '203 (Medium Dose)', 'description': 'Subject 203 M-specific AAT Level'}, {'id': 'OG003', 'title': '301 (High Dose)', 'description': 'Subject 301M-specific AAT Level'}, {'id': 'OG004', 'title': '302 (High Dose)', 'description': 'Subject 302 M-specific AAT Level'}, {'id': 'OG005', 'title': '303 (High Dose)', 'description': 'Subject 303 M-specific AAT Level'}], 'classes': [{'title': 'Baseline Level', 'categories': [{'measurements': [{'value': '33', 'groupId': 'OG000'}, {'value': '10.8', 'groupId': 'OG001'}, {'value': '11.2', 'groupId': 'OG002'}, {'value': '9.6', 'groupId': 'OG003'}, {'value': '7.1', 'groupId': 'OG004'}, {'value': '18.3', 'groupId': 'OG005'}]}]}, {'title': 'Day 14 Level', 'categories': [{'measurements': [{'value': '7.5', 'groupId': 'OG000'}, {'value': '12.7', 'groupId': 'OG001'}, {'value': '12.0', 'groupId': 'OG002'}, {'value': '13.2', 'groupId': 'OG003'}, {'value': '10.3', 'groupId': 'OG004'}, {'value': '7.8', 'groupId': 'OG005'}]}]}, {'title': 'Day 30 Level', 'categories': [{'measurements': [{'value': '7.5', 'groupId': 'OG000'}, {'value': '17.8', 'groupId': 'OG001'}, {'value': '14.7', 'groupId': 'OG002'}, {'value': '24.7', 'groupId': 'OG003'}, {'value': '16.6', 'groupId': 'OG004'}, {'value': '10.5', 'groupId': 'OG005'}]}]}, {'title': 'Day 45 Level', 'categories': [{'measurements': [{'value': '6.2', 'groupId': 'OG000'}, {'value': '16.7', 'groupId': 'OG001'}, {'value': '14.5', 'groupId': 'OG002'}, {'value': '22.6', 'groupId': 'OG003'}, {'value': '18.5', 'groupId': 'OG004'}, {'value': '41.5', 'groupId': 'OG005'}]}]}, {'title': 'Day 60 Level', 'categories': [{'measurements': [{'value': '15.1', 'groupId': 'OG000'}, {'value': '16.8', 'groupId': 'OG001'}, {'value': '20.7', 'groupId': 'OG002'}, {'value': '20.8', 'groupId': 'OG003'}, {'value': '10.5', 'groupId': 'OG004'}, {'value': '37.7', 'groupId': 'OG005'}]}]}, {'title': 'Day 75 Level', 'categories': [{'measurements': [{'value': '14.6', 'groupId': 'OG000'}, {'value': '14.7', 'groupId': 'OG001'}, {'value': '12.9', 'groupId': 'OG002'}, {'value': '21.8', 'groupId': 'OG003'}, {'value': '14.3', 'groupId': 'OG004'}, {'value': '45.9', 'groupId': 'OG005'}]}]}, {'title': 'Day 90 Level', 'categories': [{'measurements': [{'value': '26.8', 'groupId': 'OG000'}, {'value': '12.6', 'groupId': 'OG001'}, {'value': '14.6', 'groupId': 'OG002'}, {'value': '42.6', 'groupId': 'OG003'}, {'value': '6.7', 'groupId': 'OG004'}, {'value': '48.5', 'groupId': 'OG005'}]}]}, {'title': 'Day 180 Level', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG000'}, {'value': '9.8', 'groupId': 'OG001'}, {'value': '10.8', 'groupId': 'OG002'}, {'value': '41.7', 'groupId': 'OG003'}, {'value': '26.4', 'groupId': 'OG004'}, {'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG005'}]}]}, {'title': 'Day 270 Level', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG000'}, {'value': '8.0', 'groupId': 'OG001'}, {'value': '7.0', 'groupId': 'OG002'}, {'value': '47.9', 'groupId': 'OG003'}, {'value': '33.5', 'groupId': 'OG004'}, {'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG005'}]}]}, {'title': 'Day 365 Level', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG000'}, {'value': 'NA', 'comment': 'blood hemolyzed', 'groupId': 'OG001'}, {'value': '24.2', 'groupId': 'OG002'}, {'value': '50.9', 'groupId': 'OG003'}, {'value': '33.4', 'groupId': 'OG004'}, {'value': 'NA', 'comment': 'resumed AAT protein therapy', 'groupId': 'OG005'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, Days 14, 30, 45, 60, 90, (180, 270, and 365 if not on protein replacement therapy)', 'description': '4 subjects received prior AAT augmentation therapy; 2 subjects from Group 1 having only washed out for only 28 days complicated the measurement of M-specific levels 2 subjects from group 1 and the other subject did not have an appreciable change in M-specific AAT levels. Thus reporting only Cohorts 2 and 3. After day 90 patients were able to resume AAT protein therapy and thus levels were not collected following commencement of therapy on 201 and 303. 202, Day 365 blood hemolyzed; level not determinable.', 'unitOfMeasure': 'nM', 'reportingStatus': 'POSTED', 'populationDescription': 'AAT Levels of each subject at various time points Baseline and Days following administration. 202 Day 365 blood hemolyzed; not determinable, 201 and 303 went back on AAT protein augmentation therapy after day 90, unable to collect M-specific levels on day 180, 270 and 303.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Group 1 Low Dose', 'description': '6.9 x10e12 vector genomes (vg)\n\nGroup 1 receives rAAV1-CB-hAAT 6.9 x10e12 vg.'}, {'id': 'FG001', 'title': 'Group 2 Middle Dose', 'description': '2.1 x 10e13 vector genomes; 2.2 x 10e13 vector genomes\n\nGroup 2, 1 subject received rAAV1-CB-hAAT 2.1 x10e13 vg; 202 and 203 received rAAV1-CB-hAAT 2.2 x10e13 vg'}, {'id': 'FG002', 'title': 'Group 3 High Dose', 'description': 'rAAV1-CB-hAAT 6.0 x10e13 vg\n\nGroup 3 receives rAAV1-CB-hAAT 6.0 x10e13 vg'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Subjects were recruited to the University of Florida, Clinical Research Center for the active study; long term follow up was completed at the University of Massachusetts, Medical School.', 'preAssignmentDetails': 'Subjects on alpha-1 antitrypsin (AAT) protein replacement product prior to study, discontinued treatment 4 weeks (Group 1) and 8 weeks (Groups 2 and 3) prior to study agent administration and were able to resume treatment 11 weeks after study agent had been administered. The group was determined by when the subject joined the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Group 1 Low Dose', 'description': '6.9 x10e12 vector genomes\n\nGroup 1 receives rAAV1-CB-hAAT 6.9 x1012 vg (vector genomes), e'}, {'id': 'BG001', 'title': 'Group 2 Middle Dose', 'description': '2.1 x 10e13 vector genomes\n\nGroup 2 receives rAAV1-CB-hAAT 2.1 x1013 vg'}, {'id': 'BG002', 'title': 'Group 3 High Dose', 'description': 'rAAV1-CB-hAAT 6.0 x10e13 vg\n\nGroup 2 receives rAAV1-CB-hAAT 2.1 x1013 vg'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'categories': [{'measurements': [{'value': '69', 'groupId': 'BG000', 'lowerLimit': '66', 'upperLimit': '73'}, {'value': '54', 'groupId': 'BG001', 'lowerLimit': '38', 'upperLimit': '59'}, {'value': '47', 'groupId': 'BG002', 'lowerLimit': '35', 'upperLimit': '61'}, {'value': '54', 'groupId': 'BG003', 'lowerLimit': '35', 'upperLimit': '73'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'FEV1 (% predicted)', 'classes': [{'categories': [{'measurements': [{'value': '86.7', 'groupId': 'BG000', 'lowerLimit': '53.6', 'upperLimit': '92.1'}, {'value': '50.8', 'groupId': 'BG001', 'lowerLimit': '40.5', 'upperLimit': '93.0'}, {'value': '58.0', 'groupId': 'BG002', 'lowerLimit': '54.9', 'upperLimit': '97.6'}, {'value': '58.0', 'groupId': 'BG003', 'lowerLimit': '40.5', 'upperLimit': '97.6'}]}]}], 'paramType': 'MEDIAN', 'description': 'forced expiratory volume in 1 second; percent predicted for race, height, and weight', 'unitOfMeasure': 'percent predicted', 'dispersionType': 'FULL_RANGE'}, {'title': 'Weight (kg)', 'classes': [{'categories': [{'measurements': [{'value': '66.5', 'groupId': 'BG000', 'lowerLimit': '54.6', 'upperLimit': '73.7'}, {'value': '83.0', 'groupId': 'BG001', 'lowerLimit': '72.7', 'upperLimit': '89.0'}, {'value': '72.6', 'groupId': 'BG002', 'lowerLimit': '69.3', 'upperLimit': '134.6'}, {'value': '72.6', 'groupId': 'BG003', 'lowerLimit': '54.6', 'upperLimit': '134.6'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'kilograms', 'dispersionType': 'FULL_RANGE'}, {'title': 'Prior AAT Protein Augmentation Therapy', 'classes': [{'title': 'Yes', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}, {'title': 'No', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-12', 'completionDateStruct': {'date': '2015-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-12-13', 'studyFirstSubmitDate': '2007-01-31', 'resultsFirstSubmitDate': '2016-09-21', 'studyFirstSubmitQcDate': '2007-01-31', 'lastUpdatePostDateStruct': {'date': '2016-12-15', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2016-12-13', 'studyFirstPostDateStruct': {'date': '2007-02-02', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-12-15', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse Events Possibly, Probably or Definitely Related to Study Drug', 'timeFrame': 'During 1 year after study agent administration', 'description': 'Adverse events considered possibly, probably or definitely related to study drug/study drug procedure Criteria to evaluate severity according to Attachment 2 of the Protocol\n\n1. Mild toxicity, usually transient, requiring no special treatment and generally not interfering with usual daily activities\n2. Moderate toxicity which may be ameliorated by simple therapeutic maneuvers, and impairs usual activities\n3. Severe toxicity which requires therapeutic intervention and interrupts usual activities. Hospitalization may or may not be required\n4. Life-threatening toxicity which requires hospitalization'}], 'secondaryOutcomes': [{'measure': 'hAAT Expression in Blood Measured Using M-specific Allele ELISA', 'timeFrame': 'Baseline, Days 14, 30, 45, 60, 90, (180, 270, and 365 if not on protein replacement therapy)', 'description': '4 subjects received prior AAT augmentation therapy; 2 subjects from Group 1 having only washed out for only 28 days complicated the measurement of M-specific levels 2 subjects from group 1 and the other subject did not have an appreciable change in M-specific AAT levels. Thus reporting only Cohorts 2 and 3. After day 90 patients were able to resume AAT protein therapy and thus levels were not collected following commencement of therapy on 201 and 303. 202, Day 365 blood hemolyzed; level not determinable.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Gene Transfer Techniques', 'Gene Therapy', 'AAV', 'AAT', 'Phase I', 'Intramuscular transfer'], 'conditions': ['Alpha 1-Antitrypsin Deficiency']}, 'referencesModule': {'references': [{'pmid': '9826709', 'type': 'BACKGROUND', 'citation': 'Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998 Nov 24;95(24):14384-8. doi: 10.1073/pnas.95.24.14384.'}, {'pmid': '16518879', 'type': 'BACKGROUND', 'citation': 'Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, Flotte TR, Song S. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med. 2006 Jun;8(6):730-5. doi: 10.1002/jgm.896.'}, {'pmid': '17115945', 'type': 'BACKGROUND', 'citation': 'Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther. 2006 Dec;17(12):1177-86. doi: 10.1089/hum.2006.17.1177.'}]}, 'descriptionModule': {'briefSummary': 'Individuals with a deficiency of the alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells with the expectation that the AAT protein may be produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency. The study will also determine what dose may be required to achieve normal levels of AAT.', 'detailedDescription': 'AAT deficiency is a genetic disorder in which individuals have inadequate levels of the AAT protein. AAT protects the lungs from white blood cell enzymes that can damage air sacs within the lungs, potentially leading to emphysema. Experimental gene transfer procedures, in which normal copies of genes are inserted into cells, are being developed to treat many genetic diseases, including AAT deficiency. In this study, a modified virus, adeno-associated virus (AAV), has been genetically engineered to contain a normal copy of the AAT gene. When AAV is combined with the AAT gene, the resulting agent, Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector with a chicken beta actin promoter (CB), may be able to carry normal copies of the AAT gene into muscle cells with the expectation that additional AAT would be produced. The purpose of this study is to evaluate the safety of injecting rAAV1-CB-hAAT into individuals with AAT deficiency.\n\nThis 14-month study will enroll individuals with AAT deficiency. Participants currently using AAT protein replacement will discontinue its use for 19 weeks during the study. Participants will first attend a baseline study visit, which will include a medical history review; a physical examination; an electrocardiogram (ECG) to record heart activity; blood, urine, and semen collection; pulmonary function tests; and chest and arm scans. Participants will then attend a 5-day inpatient visit, during which they will receive a series of injections consisting of one of four different doses of rAAV1-CB-hAAT. Physical examinations will occur on all 5 inpatient days; pulmonary function testing, arm circumference measurements, and collection of blood, urine, and semen will occur on selected days of the inpatient stay. Follow-up study visits, with possible overnight stays, will occur on Days 14 and 90. On Days 30, 45, 60, 75, 180, 270, and 365, participants will have blood drawn at a local clinic. On these same days, study staff will contact participants by telephone to review their medical history and symptoms. Unused blood and semen samples will be frozen and stored for future research purposes. Participants will have yearly follow-up evaluations by either telephone or mail for a total of 5 years.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed with AAT deficiency\n* Forced expiratory volume in one second (FEV1) greater than 24% of predicted value (post bronchodilator)\n* Willing to discontinue AAT protein replacement 4 weeks (Group 1) and 8 weeks (Groups 2 and 3) prior to study entry, and to resume 11 weeks after rAAV1-CB-hAAT has been administered\n* Willing to discontinue aspirin, aspirin-containing products, and other drugs that may alter platelet function 7 days prior to study entry, and to resume 24 hours after rAAV1-CB-hAAT has been administered\n* Willing to use contraception throughout the study\n\nExclusion Criteria:\n\n* Required antibiotic therapy for a respiratory infection in the 28 days prior to rAAV1-CB-hAAT administration\n* Required oral or systemic corticosteroids in the 28 days prior to rAAV1-CB-hAAT administration\n* Liver disease\n* Currently receiving or has received an investigational study agent in the 30 days prior to study entry\n* Received gene transfer agents in the 6 months prior to study entry\n* Currently smokes cigarettes or uses illegal drugs\n* History of immune response to human AAT replacement\n* History of platelet dysfunction\n* Abnormal ECG, heart disease, pulmonary edema, or embolism in the 6 months prior to study entry\n* Current or recent facial or chest trauma that makes it medically impossible to perform pulmonary function tests (PFTs)\n* Any other medical condition that the investigator deems unsuitable for study participation\n* Pregnant or breastfeeding'}, 'identificationModule': {'nctId': 'NCT00430768', 'acronym': 'AAT', 'briefTitle': 'Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency', 'organization': {'class': 'OTHER', 'fullName': 'University of Massachusetts, Worcester'}, 'officialTitle': 'Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults', 'orgStudyIdInfo': {'id': '438'}, 'secondaryIdInfos': [{'id': '5R01HL069877', 'link': 'https://reporter.nih.gov/quickSearch/5R01HL069877', 'type': 'NIH'}, {'id': 'NIH-OBA 0404-638', 'type': 'REGISTRY', 'domain': 'Gene Therapy Protocol Number'}, {'id': 'NCRR-supported GCRC# 611', 'type': 'OTHER', 'domain': 'University of Florida'}, {'id': 'UF IBC RD 2630', 'type': 'OTHER', 'domain': 'University of Florida'}, {'id': 'AGTC-AAV1-001', 'type': 'OTHER', 'domain': 'Applied Genetics Technologies C'}, {'id': 'WIRB # 20052374', 'type': 'OTHER', 'domain': 'Western Institutional Review B'}, {'id': 'BB-IND 12728', 'type': 'OTHER', 'domain': 'FDA'}, {'id': 'RR00032, RR00082', 'type': 'OTHER_GRANT', 'domain': 'NCRR'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group 1 Low Dose', 'description': 'rAAV1-CB-hAAT 6.9 x10e12 vector genomes (vg) administered in a 9.9 ml volume of study agent in nine separate 1.1 mL injections in the deltoid muscle of the "non-dominant" side under ultrasound guidance', 'interventionNames': ['Biological: rAAV1-CB-hAAT']}, {'type': 'EXPERIMENTAL', 'label': 'Group 2 Middle Dose', 'description': 'rAAV1-CB-hAAT 2.2 x 10e13 vg administered in a 9.9 ml volume of study agent in nine separate 1.1 mL injections in the deltoid muscle of the "non-dominant" side under ultrasound guidance', 'interventionNames': ['Biological: rAAV1-CB-hAAT']}, {'type': 'EXPERIMENTAL', 'label': 'Group 3 High Dose', 'description': 'rAAV1-CB-hAAT 6.0 x10e13 vg administered in a 9.9 ml volume of study agent in nine separate 1.1 mL injections in the deltoid muscle of the "non-dominant" side under ultrasound guidance', 'interventionNames': ['Biological: rAAV1-CB-hAAT']}], 'interventions': [{'name': 'rAAV1-CB-hAAT', 'type': 'BIOLOGICAL', 'armGroupLabels': ['Group 1 Low Dose', 'Group 2 Middle Dose', 'Group 3 High Dose']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida, College of Medicine, Department of Pediatrics', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '01655', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'University of Massachusetts School of Medicine', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}], 'overallOfficials': [{'name': 'Terence R. Flotte, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'UMass Medical School'}, {'name': 'Mark L Brantly, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Florida'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'Laboratory information shared as received; copy of manuscript given to patients; at 5 year follow up Principal investigator explained results to patients'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Massachusetts, Worcester', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}, {'name': 'Beacon Therapeutics', 'class': 'INDUSTRY'}, {'name': 'Alpha-1 Foundation', 'class': 'OTHER'}, {'name': 'University of Florida', 'class': 'OTHER'}, {'name': 'National Center for Research Resources (NCRR)', 'class': 'NIH'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Study Principle Investigator', 'investigatorFullName': 'Terence Flotte', 'investigatorAffiliation': 'University of Massachusetts, Worcester'}}}}