Ignite Creation Date:
2025-12-24 @ 9:33 PM
Ignite Modification Date:
2025-12-31 @ 12:15 AM
Study NCT ID:
NCT00867932
Status:
COMPLETED
Last Update Posted:
2018-10-31
First Post:
2009-03-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
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': 'First dose of study drug (Day 0) to End of Follow-up (Week 20 [8 weeks after EOT])', 'eventGroups': [{'id': 'EG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.', 'otherNumAtRisk': 7, 'otherNumAffected': 7, 'seriousNumAtRisk': 7, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Lymph node pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Chromaturia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Vaginal discharge', 'notes': 'Includes only female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Vulvovaginal pruritus', 'notes': 'Includes only female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Petechiae', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Rash papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Skin hyperpigmentation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Swelling face', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Aplastic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Menorrhagia', 'notes': 'Includes only female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Vaginal haemorrhage', 'notes': 'Includes only female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Acute sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Catheter site cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.0)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Peak And Trough Concentrations Of Eculizumab In Serum At Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'classes': [{'title': 'Trough concentration', 'categories': [{'measurements': [{'value': '192.5', 'groupId': 'OG000', 'lowerLimit': '124.2', 'upperLimit': '321.1'}]}]}, {'title': 'Peak concentration', 'categories': [{'measurements': [{'value': '425.4', 'groupId': 'OG000', 'lowerLimit': '220.5', 'upperLimit': '566.1'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Pre-infusion and 1 hour post-infusion at End of Treatment (EOT) (Day 84 [Week 12]) or ET', 'description': 'Serum concentrations of eculizumab were measured by using a validated enzyme-linked immunosorbent assay (ELISA) method developed at Alexion Pharmaceuticals Bioanalytical Laboratory. The range of the analytical assay was 10 to 600 microgram per milliliter (μg/mL). Peak concentrations were not measured at the early termination (ET) visit.', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Number Of Participants With Treatment-emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'classes': [{'title': 'Participants with at least 1 TEAE', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any serious TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any mild TEAE', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any moderate TEAE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any severe TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any unrelated TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any possibly related TEAE', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any probably related TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Participants with any definitely related TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'First dose of study drug (Day 0) to End of Follow-up (Week 20 [8 weeks after EOT])', 'description': 'A TEAE was defined as any adverse event (AE) not present prior to exposure to eculizumab or any event already present that worsened in either intensity or frequency following exposure to eculizumab. A serious TEAE was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. Related TEAEs were considered by investigators to be definitely, probably, or possibly related to administration of the study drug. Relationship is ordered as follows: unrelated, possibly related, probably related, or definitely related. TEAEs and TEAE severity were classified in accordance with the Medical Dictionary for Regulatory Activities (MedDRA) 13.0 dictionary. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Area Under The Curve (AUC) Of The Change From Baseline To Week 12 In Levels Of Lactate Dehydrogenase (LDH)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'classes': [{'categories': [{'measurements': [{'value': '-60634', 'spread': '72916', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, EOT (Day 84 [Week 12]) or ET', 'description': 'The AUC of LDH was calculated by using the change of LDH from baseline values for each participant up to Week 12. For those participants with missing LDH values, the last observation carried forward method (LOCF) was used to impute missing values. Individual AUC values of LDH were summarized and tabulated.', 'unitOfMeasure': 'Units * Days per Liter (U*Day/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Concentration Of Plasma-free Hemoglobin At Baseline And Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '17.70', 'spread': '19.091', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'categories': [{'measurements': [{'value': '7.44', 'spread': '3.145', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, EOT (Day 84 [Week 12]) or ET', 'description': 'Plasma-free hemoglobin was determined for each participant by using standard laboratory assays. The values of plasma-free hemoglobin were summarized by visit.', 'unitOfMeasure': 'mg per deciliter (mg/dL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline In LDH Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '1020', 'spread': '967', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 1', 'categories': [{'measurements': [{'value': '-672', 'spread': '837', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 2', 'categories': [{'measurements': [{'value': '-763', 'spread': '916', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 3', 'categories': [{'measurements': [{'value': '-752', 'spread': '934', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 4', 'categories': [{'measurements': [{'value': '-761', 'spread': '924', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 8', 'categories': [{'measurements': [{'value': '-747', 'spread': '904', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline at Week 12', 'categories': [{'measurements': [{'value': '-771', 'spread': '914', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 1 to 12 or ET', 'description': 'Levels of LDH were determined by using standard laboratory assays. LDH values and the change of LDH from baseline were summarized by visit.', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an intravenous (IV) infusion for 12 weeks. All participants weighed more than 45 kilograms (kg) and received the following weight-based dosing regimen: induction/loading = 600 milligram (mg) weekly x 4; maintenance = 900 mg at Week (Wk) 5; 900 mg every 2 weeks (Q2W).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'Induction Phase', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'Maintenance Phase', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'Intent-to-Treat (ITT) Population', 'comment': 'Participants who received at least 1 dose of study drug.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'The recruitment period lasted from October 2009 to January 2011. Three sites in the United States of America enrolled a total of 7 participants with paroxysmal nocturnal hemoglobinuria (PNH).', 'preAssignmentDetails': "Participants who completed this study could have continued treatment with commercially available eculizumab (Soliris®) and been followed in the Soliris® PNH Registry. Those who didn't complete the study or who didn't continue with Soliris® treatment after the study were followed for 8 weeks and monitored for signs and symptoms of serious hemolysis."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 mg weekly x 4; maintenance = 900 mg at Wk 5; 900 mg Q2W.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '15.01', 'spread': '2.28', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': 'Children (2 to 11 years)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Adolescents (12 to 17 years)', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'ITT Population: Participants who received at least 1 dose of study drug.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 7}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-10-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-10', 'dispFirstSubmitDate': '2017-03-09', 'completionDateStruct': {'date': '2011-05-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-10-03', 'studyFirstSubmitDate': '2009-03-23', 'dispFirstSubmitQcDate': '2017-03-09', 'resultsFirstSubmitDate': '2018-10-03', 'studyFirstSubmitQcDate': '2009-03-23', 'dispFirstPostDateStruct': {'date': '2017-03-10', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2018-10-31', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-10-03', 'studyFirstPostDateStruct': {'date': '2009-03-24', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-10-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2011-05-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Peak And Trough Concentrations Of Eculizumab In Serum At Week 12', 'timeFrame': 'Pre-infusion and 1 hour post-infusion at End of Treatment (EOT) (Day 84 [Week 12]) or ET', 'description': 'Serum concentrations of eculizumab were measured by using a validated enzyme-linked immunosorbent assay (ELISA) method developed at Alexion Pharmaceuticals Bioanalytical Laboratory. The range of the analytical assay was 10 to 600 microgram per milliliter (μg/mL). Peak concentrations were not measured at the early termination (ET) visit.'}], 'secondaryOutcomes': [{'measure': 'Number Of Participants With Treatment-emergent Adverse Events (TEAEs)', 'timeFrame': 'First dose of study drug (Day 0) to End of Follow-up (Week 20 [8 weeks after EOT])', 'description': 'A TEAE was defined as any adverse event (AE) not present prior to exposure to eculizumab or any event already present that worsened in either intensity or frequency following exposure to eculizumab. A serious TEAE was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. Related TEAEs were considered by investigators to be definitely, probably, or possibly related to administration of the study drug. Relationship is ordered as follows: unrelated, possibly related, probably related, or definitely related. TEAEs and TEAE severity were classified in accordance with the Medical Dictionary for Regulatory Activities (MedDRA) 13.0 dictionary. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.'}, {'measure': 'Area Under The Curve (AUC) Of The Change From Baseline To Week 12 In Levels Of Lactate Dehydrogenase (LDH)', 'timeFrame': 'Baseline, EOT (Day 84 [Week 12]) or ET', 'description': 'The AUC of LDH was calculated by using the change of LDH from baseline values for each participant up to Week 12. For those participants with missing LDH values, the last observation carried forward method (LOCF) was used to impute missing values. Individual AUC values of LDH were summarized and tabulated.'}, {'measure': 'Concentration Of Plasma-free Hemoglobin At Baseline And Week 12', 'timeFrame': 'Baseline, EOT (Day 84 [Week 12]) or ET', 'description': 'Plasma-free hemoglobin was determined for each participant by using standard laboratory assays. The values of plasma-free hemoglobin were summarized by visit.'}, {'measure': 'Change From Baseline In LDH Levels', 'timeFrame': 'Baseline, Weeks 1 to 12 or ET', 'description': 'Levels of LDH were determined by using standard laboratory assays. LDH values and the change of LDH from baseline were summarized by visit.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True}, 'conditionsModule': {'keywords': ['Alexion', 'Eculizumab', 'Paroxysmal Nocturnal Hemoglobinuria', 'PNH', 'Soliris'], 'conditions': ['Hemoglobinuria, Paroxysmal']}, 'referencesModule': {'references': [{'pmid': '24777716', 'type': 'BACKGROUND', 'citation': 'Reiss UM, Schwartz J, Sakamoto KM, Puthenveetil G, Ogawa M, Bedrosian CL, Ware RE. Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria. Pediatr Blood Cancer. 2014 Sep;61(9):1544-50. doi: 10.1002/pbc.25068. Epub 2014 Apr 29.'}], 'seeAlsoLinks': [{'url': 'http://www.ncbi.nlm.nih.gov/pubmed/24777716', 'label': 'PubMed'}]}, 'descriptionModule': {'briefSummary': 'The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.', 'detailedDescription': "This was an open-label, multi-center study of eculizumab administered to approximately 6 to 8 pediatric and adolescent participants aged 2 to 17 years with PNH. There were 3 periods in this study (screening, treatment, and post-treatment) with the treatment period having 2 dosing phases (induction and maintenance). If all screening criteria were met, the participant was eligible to enter the treatment period of the study after receiving Neisseria meningitidis (N. men), Streptococcus pneumoniae (S. pneumo), and Haemophilus influenzae (H. influ) vaccinations at least 14 days prior to first dose of study drug, or was vaccinated and received treatment with appropriate antibiotics until 14 days after the vaccinations. Participants received eculizumab intravenously (IV) based on their weight. Eculizumab was administered via an IV infusion at a rate of 5 to 10 milliliters (mL) per kilogram (kg) per hour (hr) (mL/kg/hr) for at least 25 minutes. The planned duration of treatment was 12 weeks with a 4-week induction phase and an 8-week maintenance phase. At the Investigator's and parents/legal guardian's discretion, participants who completed this study with eculizumab could continue treatment with commercially available eculizumab (Soliris®) and were followed in the Soliris® PNH Registry. Participants who stopped study participation before study completion or who did not continue with Soliris® treatment at the completion of the study were followed for 8 weeks and monitored for signs and symptoms of serious hemolysis."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Participants between 2 and 17 years of age;\n* Diagnosed with PNH;\n* Participants with ≥ 5% glycosylphosphatidylinositol-deficient red blood cells or granulocytes as confirmed by flow cytometry;\n* Participants must have shown evidence of hemolytic anemia as documented by lactate dehydrogenase greater than the upper limit of normal or at least 1 transfusion in the past 2 years for anemia or anemia related symptoms;\n* Written informed consent from a parent/guardian;\n* Negative pregnancy test for females of child bearing potential at screening;\n* Sexually active females must have documented a reliable and medically approved method of contraception;\n* Participant must have been vaccinated against N. men, S. pneumo, and H. influ at least 14 days prior to study drug initiation or received antibiotics for 14 days after the vaccinations.\n\nExclusion Criteria:\n\n* Prior eculizumab treatment;\n* Presence or suspicion of active bacterial infection at baseline;\n* Participation in another concurrent clinical study within at least 30 days prior to screening;\n* History of meningococcal/pneumococcal/gonococcal disease;\n* Pregnant, breast feeding, or intending to conceive during the study including the safety follow-up visits;\n* Any other condition that could increase the participant's risk or confound the outcome of the study."}, 'identificationModule': {'nctId': 'NCT00867932', 'briefTitle': 'Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria', 'orgStudyIdInfo': {'id': 'M07-005'}, 'secondaryIdInfos': [{'id': '2009-010402-11', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Eculizumab', 'description': 'Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 milligram (mg) weekly x 4; maintenance = 900 mg at Week 5; 900 mg every 2 weeks.', 'interventionNames': ['Drug: Eculizumab']}], 'interventions': [{'name': 'Eculizumab', 'type': 'DRUG', 'otherNames': ['Soliris®'], 'description': '5 mg/mL solution in 5% Dextrose', 'armGroupLabels': ['Eculizumab']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '32504', 'city': 'Pensacola', 'state': 'Florida', 'country': 'United States', 'geoPoint': {'lat': 30.42131, 'lon': -87.21691}}, {'zip': '38105', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}