Ignite Creation Date:
2025-12-24 @ 11:10 PM
Ignite Modification Date:
2025-12-25 @ 8:43 PM
Study NCT ID:
NCT01379469
Status:
TERMINATED
Last Update Posted:
2021-10-12
First Post:
2011-06-15
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}, {'id': 'D008103', 'term': 'Liver Cirrhosis'}, {'id': 'D008107', 'term': 'Liver Diseases'}], 'ancestors': [{'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'}, {'id': 'D005355', 'term': 'Fibrosis'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D002220', 'term': 'Carbamazepine'}], 'ancestors': [{'id': 'D003984', 'term': 'Dibenzazepines'}, {'id': 'D006575', 'term': 'Heterocyclic Compounds, 3-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'rudnick_d@wustl.edu', 'phone': '314-286-2832', 'title': 'David Rudnick MD, PhD- Associate Professor', 'organization': 'Washington University School of Medicine'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The primary \\& secondary outcomes could not be assessed because the number of subjects with available pre \\& post biopsies was insufficient in subject number and sample quality to conduct the analyses. As described in more detail in Outcome Measures.\n\n.'}}, 'adverseEventsModule': {'timeFrame': 'Serious Adverse Events and Adverse Events were collected for 52 weeks.', 'eventGroups': [{'id': 'EG000', 'title': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nDrug-Carbamazepine (Tegretol XR): To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated, and the placebo group will receive encapsulated tablets without CBZ.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 13, 'seriousNumAtRisk': 13, 'deathsNumAffected': 0, 'seriousNumAffected': 6}, {'id': 'EG001', 'title': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nCarbamazepine (Tegretol XR) Placebo: Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.', 'otherNumAtRisk': 7, 'deathsNumAtRisk': 7, 'otherNumAffected': 7, 'seriousNumAtRisk': 7, 'deathsNumAffected': 2, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Cardiac', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Child-Pugh Score Increase', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Constitutional', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 63, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 17, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dermatologic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Endocrine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastrointestinal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 35, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 13, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Genitourological', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gynecological', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hematologic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 16, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Metabolic/Laboratory', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Musculoskeletal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 12, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Neurologic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 31, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 15, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pulmonary', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 9, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Gastrointestinal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pulmonary', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cardiac', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 4, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Constitutional', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 6, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hematologic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Metabolic/Laboratory', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nDrug-Carbamazepine (Tegretol XR): To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated, and the placebo group will receive encapsulated tablets without CBZ.'}, {'id': 'OG001', 'title': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nCarbamazepine (Tegretol XR) Placebo: Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.'}], 'timeFrame': '52 weeks', 'description': 'The effect of Carbamazepine on hepatic ATZ load will be measured by the number of hepatocytes with PAS+/diastase-resistant globules and/or steady state levels of ATZ by immunoblot analysis.', 'reportingStatus': 'POSTED', 'populationDescription': 'The primary outcome was not assessable because the number of subjects with available pre \\& post PAS+/diastase stained liver biopsies \\& tissue for immunoblot was insufficient in subject number and sample quality. To elaborate only 3 subjects had both pre \\& post biopsies stained for PAS, 2 from the placebo \\& 1 from the Carbamazepine arms \\& the available histology quality was insufficient for histomorphormetry. Frozen liver samples were also of insufficient number \\& mass for immunoblot.'}, {'type': 'SECONDARY', 'title': 'For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nDrug-Carbamazepine (Tegretol XR): To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated, and the placebo group will receive encapsulated tablets without CBZ.'}, {'id': 'OG001', 'title': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nCarbamazepine (Tegretol XR) Placebo: Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.'}], 'timeFrame': '52 weeks', 'description': 'For the secondary outcomes we will determine the effect of Carbamazepine treatment on hepatic fibrosis on the basis of sirius red staining and hydroxyproline concentration and whether Carbamazepine treatment changes portal pressure as determined by Hepatic Venous Pressure Gradient.', 'reportingStatus': 'POSTED', 'populationDescription': 'The secondary outcome was also not assessable because the number of subjects with available pre \\& post trichrome stained liver biopsies \\& tissue for hydroxyproline was also insufficient in subject number and sample quality. Again only 3 subjects had both pre \\& post biopsies stained for trichrome, 2 placebo- \\& 1 Carbamazepine treated. Available histology quality was also insufficient here for histomorphormetry \\&.frozen liver samples insufficient for hydroxyproline determination.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nDrug-Carbamazepine (Tegretol XR): To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated.'}, {'id': 'FG001', 'title': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nCarbamazepine (Tegretol XR) Placebo: Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '7'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '4'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nDrug-Carbamazepine (Tegretol XR): To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated, and the placebo group will receive encapsulated tablets without CBZ.'}, {'id': 'BG001', 'title': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.\n\nCarbamazepine (Tegretol XR) Placebo: Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '50.7', 'spread': '9.4', 'groupId': 'BG000'}, {'value': '53.7', 'spread': '19.6', 'groupId': 'BG001'}, {'value': '51.75', 'spread': '13.38', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}], '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'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-02-19', 'size': 1192116, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-10-20T10:46', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 20}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2012-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-10', 'completionDateStruct': {'date': '2017-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-10-07', 'studyFirstSubmitDate': '2011-06-15', 'resultsFirstSubmitDate': '2021-09-09', 'studyFirstSubmitQcDate': '2011-06-21', 'lastUpdatePostDateStruct': {'date': '2021-10-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-10-07', 'studyFirstPostDateStruct': {'date': '2011-06-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-10-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load.', 'timeFrame': '52 weeks', 'description': 'The effect of Carbamazepine on hepatic ATZ load will be measured by the number of hepatocytes with PAS+/diastase-resistant globules and/or steady state levels of ATZ by immunoblot analysis.'}], 'secondaryOutcomes': [{'measure': 'For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis.', 'timeFrame': '52 weeks', 'description': 'For the secondary outcomes we will determine the effect of Carbamazepine treatment on hepatic fibrosis on the basis of sirius red staining and hydroxyproline concentration and whether Carbamazepine treatment changes portal pressure as determined by Hepatic Venous Pressure Gradient.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Carbamazepine (Tegretol) use', 'severe liver disease', 'alpha-1-antitrypsin deficiency'], 'conditions': ['Alpha-1-antitrypsin Deficiency', 'Liver Cirrhosis']}, 'referencesModule': {'references': [{'type': 'BACKGROUND', 'citation': "Perlmutter D.H., Alpha-1-Antitrypsin Deficiency, in Schiff's Disease of Liver, Schiff E.R., Maddrey W.C., Editor. 2007; Lippincott-Raven: Philadelphia. 1063-1084."}, {'pmid': '16864711', 'type': 'BACKGROUND', 'citation': 'Perlmutter DH. Pathogenesis of chronic liver injury and hepatocellular carcinoma in alpha-1-antitrypsin deficiency. Pediatr Res. 2006 Aug;60(2):233-8. doi: 10.1203/01.pdr.0000228350.61496.90.'}, {'pmid': '18617899', 'type': 'BACKGROUND', 'citation': 'Perlmutter DH. Autophagic disposal of the aggregation-prone protein that causes liver inflammation and carcinogenesis in alpha-1-antitrypsin deficiency. Cell Death Differ. 2009 Jan;16(1):39-45. doi: 10.1038/cdd.2008.103. Epub 2008 Jul 11.'}, {'pmid': '16044402', 'type': 'BACKGROUND', 'citation': 'Rudnick DA, Perlmutter DH. Alpha-1-antitrypsin deficiency: a new paradigm for hepatocellular carcinoma in genetic liver disease. Hepatology. 2005 Sep;42(3):514-21. doi: 10.1002/hep.20815.'}, {'pmid': '16236857', 'type': 'BACKGROUND', 'citation': 'Piitulainen E, Carlson J, Ohlsson K, Sveger T. Alpha1-antitrypsin deficiency in 26-year-old subjects: lung, liver, and protease/protease inhibitor studies. Chest. 2005 Oct;128(4):2076-81. doi: 10.1378/chest.128.4.2076.'}, {'pmid': '1083485', 'type': 'BACKGROUND', 'citation': 'Sveger T. Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants. N Engl J Med. 1976 Jun 10;294(24):1316-21. doi: 10.1056/NEJM197606102942404.'}, {'pmid': '3485248', 'type': 'BACKGROUND', 'citation': 'Eriksson S, Carlson J, Velez R. Risk of cirrhosis and primary liver cancer in alpha 1-antitrypsin deficiency. N Engl J Med. 1986 Mar 20;314(12):736-9. doi: 10.1056/NEJM198603203141202.'}, {'pmid': '4541913', 'type': 'BACKGROUND', 'citation': 'Pugh RN, Murray-Lyon IM, Dawson JL, Pietroni MC, Williams R. Transection of the oesophagus for bleeding oesophageal varices. Br J Surg. 1973 Aug;60(8):646-9. doi: 10.1002/bjs.1800600817. No abstract available.'}, {'pmid': '10733541', 'type': 'BACKGROUND', 'citation': 'Malinchoc M, Kamath PS, Gordon FD, Peine CJ, Rank J, ter Borg PC. 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No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'The primary objective is to determine if the medication Carbamazepine, can be used as a therapy for patients with severe liver disease due to Alpha-1-Antitrypsin Deficiency .', 'detailedDescription': 'The primary objective is to determine if Carbamazepine therapy in patients with severe liver disease due to Alpha-1-Antitrypsin Deficiency leads to a significant reduction in the hepatic accumulation of ATZ.\n\nThe other objectives are:\n\nTo determine whether Carbamazepine treatment reduces hepatic fibrosis in alpha-1-antitrypsin deficient patients with severe liver disease. To determine whether Carbamazepine treatment reduces portal pressure in alpha-1-antitrypsin deficient patients with severe liver disease. To determine whether Carbamazepine treatment is safe and tolerated by patients with severe liver disease caused by alpha-1-deficiency. To determine whether Carbamazepine treatment leads to stabilization in disease severity as measured by the MELD scores.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '14 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age greater than or equal to 14 years to less than or equal to 80 years of age.\n* Alpha-1-Antitrypsin deficiency confirmed by ZZ or SZ phenotype \\& serum level\n* \\< 83mg/dl.\n* HVPG greater than or equal to 10 mmHg unless collateral vessels are visualized via transvenous biopsy.\n\nExclusion Criteria:\n\n* Child Pugh Score greater than or equal to 12. Serum total bilirubin \\> 5 mg/dl. INR \\> 2.2.'}, 'identificationModule': {'nctId': 'NCT01379469', 'acronym': 'CBZ', 'briefTitle': 'Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency', 'organization': {'class': 'OTHER', 'fullName': 'Washington University School of Medicine'}, 'officialTitle': 'A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency', 'orgStudyIdInfo': {'id': '201510060-PRO09070279'}, 'secondaryIdInfos': [{'id': '1R21DK092567-01', 'link': 'https://reporter.nih.gov/quickSearch/1R21DK092567-01', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Drug-Carbamazepine (Tegretol XR)', 'description': 'One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.', 'interventionNames': ['Drug: Drug-Carbamazepine (Tegretol XR)']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Drug-Carbamazepine (Tegretol XR) Placebo', 'description': 'One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.', 'interventionNames': ['Drug: Carbamazepine (Tegretol XR) Placebo']}], 'interventions': [{'name': 'Drug-Carbamazepine (Tegretol XR)', 'type': 'DRUG', 'otherNames': ['Tegretol-XR Carbamazepine extended release tablets.', 'NDC 0078-0510-05.'], 'description': 'To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated..', 'armGroupLabels': ['Drug-Carbamazepine (Tegretol XR)']}, {'name': 'Carbamazepine (Tegretol XR) Placebo', 'type': 'DRUG', 'otherNames': ['Carbamazepine (Tegretol-XR) placebo.'], 'description': 'Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.', 'armGroupLabels': ['Drug-Carbamazepine (Tegretol XR) Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University in St. Louis School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '15201', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh, UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center, Presbyterian Hospital', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}], 'overallOfficials': [{'name': 'David H. Perlmutter, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Washington University School of Medicine'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'We will share data and liver tissue with other researchers. They may be doing research in areas similar to this research or in other unrelated areas. These researchers may be at Washington University, at other research centers and institutions, or industry sponsors of research. We may also share research data with large data repositories (a repository is a database of information) for broad sharing with the research community. If individual research data is placed in one of these repositories only qualified researchers, who have received prior approval from individuals that monitor the use of the data, will be able to look at the information.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Washington University School of Medicine', 'class': 'OTHER'}, 'collaborators': [{'name': 'Novartis', 'class': 'INDUSTRY'}, {'name': 'National Institutes of Health (NIH)', 'class': 'NIH'}, {'name': 'National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)', 'class': 'NIH'}, {'name': 'University of Pittsburgh', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}