Ignite Creation Date:
2025-12-24 @ 7:58 PM
Ignite Modification Date:
2025-12-30 @ 3:37 AM
Study NCT ID:
NCT01194804
Status:
COMPLETED
Last Update Posted:
2019-09-17
First Post:
2010-08-30
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C481642', 'term': 'eculizumab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alexion.com', 'title': 'Alexion Pharmaceuticals, Inc.', 'organization': 'Alexion Pharmaceuticals, Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From baseline (after first dose) to end of study (1 year).', 'eventGroups': [{'id': 'EG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.', 'otherNumAtRisk': 27, 'deathsNumAtRisk': 27, 'otherNumAffected': 26, 'seriousNumAtRisk': 27, 'deathsNumAffected': 0, 'seriousNumAffected': 12}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Conjunctival haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Cataract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Enterocolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyperbilirubinaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 21}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood alkaline phosphatase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood bilirubin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper respiratory tract inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Jaundice cholestatic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Herpes virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Animal bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Subdural haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Skin disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Lactate Dehydrogenase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'classes': [{'categories': [{'measurements': [{'value': '-15.9', 'spread': '1.20', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<0.001', 'groupIds': ['OG000'], 'statisticalMethod': 'Sign test', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'unitOfMeasure': 'Units/Liter', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in FACIT-Fatigue Scale Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'classes': [{'categories': [{'measurements': [{'value': '8.0', 'spread': '1.95', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'description': 'The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PNH Red Blood Cell (RBC) Count', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.51', 'spread': '0.129', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'unitOfMeasure': 'cellsx10^12/L', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Number of Units of Packed RBCs Transfused', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.5', 'spread': '1.43', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'description': 'Baseline is defined as the number of units transfused in 3 months prior to baseline', 'unitOfMeasure': 'units', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma Free Hemoglobin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'classes': [{'categories': [{'measurements': [{'value': '-13.5', 'spread': '8.55', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with paroxysmal nocturnal hemoglobinuria who have successfully completed the C07-001 protocol (NCT01192399)\n\nEculizumab: 900 mg intravenous every 14 days.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '27'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '26'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol\n\n900 mg intravenous every 14 days.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '48.3', 'spread': '12.35', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Japanese', 'categories': [{'measurements': [{'value': '27', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Japan', 'categories': [{'measurements': [{'value': '27', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 27}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-09', 'completionDateStruct': {'date': '2011-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-09-13', 'studyFirstSubmitDate': '2010-08-30', 'resultsFirstSubmitDate': '2017-11-06', 'studyFirstSubmitQcDate': '2010-09-02', 'lastUpdatePostDateStruct': {'date': '2019-09-17', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-04-30', 'studyFirstPostDateStruct': {'date': '2010-09-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-07-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2010-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Lactate Dehydrogenase', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in FACIT-Fatigue Scale Total Score', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'description': 'The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.'}, {'measure': 'Change From Baseline in PNH Red Blood Cell (RBC) Count', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)'}, {'measure': 'Change From Baseline in Number of Units of Packed RBCs Transfused', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)', 'description': 'Baseline is defined as the number of units transfused in 3 months prior to baseline'}, {'measure': 'Change From Baseline in Plasma Free Hemoglobin', 'timeFrame': '52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hemolytic Paroxysmal Nocturnal Hemoglobinuria', 'Eculizumab', 'PNH'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'referencesModule': {'references': [{'pmid': '23934275', 'type': 'RESULT', 'citation': 'Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11.'}]}, 'descriptionModule': {'briefSummary': 'The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients must have fully completed C07-001 study 12-week treatment period\n\nExclusion Criteria:\n\n* Patients who terminated early from the C07-001 study'}, 'identificationModule': {'nctId': 'NCT01194804', 'briefTitle': 'E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol', 'orgStudyIdInfo': {'id': 'E07-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Eculizumab', 'description': 'Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol', 'interventionNames': ['Drug: Eculizumab']}], 'interventions': [{'name': 'Eculizumab', 'type': 'DRUG', 'otherNames': ['Soliris'], 'description': 'Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.', 'armGroupLabels': ['Eculizumab']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'CMIC Co, Ltd. Japan', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}