Ignite Creation Date:
2025-12-25 @ 3:45 AM
Ignite Modification Date:
2025-12-26 @ 2:32 AM
Study NCT ID:
NCT04320602
Status:
COMPLETED
Last Update Posted:
2024-09-05
First Post:
2020-03-23
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
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'}, {'id': 'C000629409', 'term': 'ravulizumab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alexion.com', 'phone': '855-752-2356', 'title': 'Alexion Pharmaceuticals Inc.', 'organization': 'Alexion Pharmaceuticals Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline through Day 351', 'description': 'Safety set included all participants who received at least 1 dose of ravulizumab.', 'eventGroups': [{'id': 'EG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen.", 'otherNumAtRisk': 18, 'deathsNumAtRisk': 18, 'otherNumAffected': 15, 'seriousNumAtRisk': 18, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Peripheral swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Upper respiratory tract congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Herpes zoster', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'SARS-CoV-2 test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Body temperature abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Haemoglobin decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Nail ridging', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Neurodermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Skin lesion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Chromaturia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Extravascular haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}], 'seriousEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Retinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants Who Experienced Free C5-associated BTH', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'spread': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '18.5'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 351', 'description': 'Free C5-associated BTH was defined as BTH concurrent with free C5 concentrations ≥0.5 micrograms (μg)/milliliter (mL). BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \\[dyspnea\\], anemia \\[hemoglobin \\<10 grams {g}/deciliter {dL}\\], major adverse vascular event \\[MAVE\\], including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) ≥2 \\* upper limit of normal (ULN).', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all participants who received at least 1 dose of ravulizumab.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Experienced BTH', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'classes': [{'categories': [{'measurements': [{'value': '5.6', 'spread': '0.1', 'groupId': 'OG000', 'lowerLimit': '0.1', 'upperLimit': '27.3'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 351', 'description': 'BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \\[dyspnea\\], anemia \\[hemoglobin \\<10 g/dL\\], MAVE, including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 \\* ULN.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all participants who received at least 1 dose of ravulizumab.'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in LDH at Day 351', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'classes': [{'categories': [{'measurements': [{'value': '-0.81', 'spread': '6.132', 'groupId': 'OG000', 'lowerLimit': '6.132'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Day 351', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': "The FAS included all participants who received at least 1 dose of ravulizumab. Here, 'Overall number of participants analyzed' = participants evaluable for this endpoint."}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Received a Red Blood Cell (RBC) Transfusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'classes': [{'categories': [{'measurements': [{'value': '33.3', 'spread': '13.3', 'groupId': 'OG000', 'lowerLimit': '13.3', 'upperLimit': '59.0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 351', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all participants who received at least 1 dose of ravulizumab.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Stabilized Hemoglobin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'classes': [{'categories': [{'measurements': [{'value': '61.1', 'spread': '35.7', 'groupId': 'OG000', 'lowerLimit': '35.7', 'upperLimit': '82.7'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 351', 'description': 'Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion from Baseline to Day 351.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all participants who received at least 1 dose of ravulizumab.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'The study consisted of a Screening Period of approximately 3 months and a Treatment Period of 351 days. Eligible participants were administered eculizumab 1200 milligrams (mg) every 2 weeks (q2w) optionally at home at the discretion of the Investigator, and preference of the participant during the Screening Period.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ravulizumab', 'description': "Participants received a loading dose of ravulizumab on Day 1 and maintenance treatment with ravulizumab on Day 15 and q8w thereafter until Day 351. Ravulizumab loading and maintenance doses were based on the participant's body weight measured at the prior visit, per approved dosing regimen."}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '55.7', 'spread': '11.34', 'groupId': 'BG000', 'lowerLimit': '11.34'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'categories': [{'title': 'In utero', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Preterm newborn infants (gestational age < 37 wks)', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Newborns (0-27 days)', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Infants and toddlers (28 days-23 months)', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Children (2-11 years)', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Adolescents (12-17 years)', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Adults (18-64 years)', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'From 65-84 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '85 years and over', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The full analysis set (FAS) included all participants who received at least 1 dose of ravulizumab.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-05-07', 'size': 3708479, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-03-12T15:04', 'hasProtocol': True}, {'date': '2023-01-20', 'size': 2715267, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-03-12T15:04', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 18}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-04-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-07', 'completionDateStruct': {'date': '2022-12-20', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-08-12', 'studyFirstSubmitDate': '2020-03-23', 'resultsFirstSubmitDate': '2024-03-12', 'studyFirstSubmitQcDate': '2020-03-23', 'lastUpdatePostDateStruct': {'date': '2024-09-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-03-12', 'studyFirstPostDateStruct': {'date': '2020-03-25', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-08-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-20', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants Who Experienced Free C5-associated BTH', 'timeFrame': 'Baseline through Day 351', 'description': 'Free C5-associated BTH was defined as BTH concurrent with free C5 concentrations ≥0.5 micrograms (μg)/milliliter (mL). BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \\[dyspnea\\], anemia \\[hemoglobin \\<10 grams {g}/deciliter {dL}\\], major adverse vascular event \\[MAVE\\], including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) ≥2 \\* upper limit of normal (ULN).'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants Who Experienced BTH', 'timeFrame': 'Baseline through Day 351', 'description': 'BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \\[dyspnea\\], anemia \\[hemoglobin \\<10 g/dL\\], MAVE, including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 \\* ULN.'}, {'measure': 'Percent Change From Baseline in LDH at Day 351', 'timeFrame': 'Baseline, Day 351'}, {'measure': 'Percentage of Participants Who Received a Red Blood Cell (RBC) Transfusion', 'timeFrame': 'Baseline through Day 351'}, {'measure': 'Percentage of Participants With Stabilized Hemoglobin', 'timeFrame': 'Baseline through Day 351', 'description': 'Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion from Baseline to Day 351.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Paroxysmal Nocturnal Hemoglobinuria', 'PNH', 'Ravulizumab', 'Ultomiris', 'Eculizumab', 'Soliris'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://filehosting-v2.pharmacm.com/api/Attachment/Download?tenantId=80217111&parentIdentifier=ALXN1210-PNH-401&attachmentIdentifier=f4f63037-1e40-4456-96df-06b466412244&fileName=ALXN1210-PNH-401-_CSR_synopsis_redacted.pdf&versionIdentifier=', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Documented diagnosis of PNH, confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells, with granulocyte or monocyte clone size of ≥ 5%.\n2. Received 1200 mg eculizumab every 12 to 16 days (every 2 weeks) for at least 3 months prior to Screening.\n3. LDH ≤ 2 x upper limit of normal (ULN) according to central laboratory, at Screening.\n4. To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infections within 3 years prior to initiating study drug.\n5. Body weight ≥ 40 kilograms.\n\nKey Exclusion Criteria:\n\n1. History of major adverse vascular events within 6 months of Day 1.\n2. History of bone marrow transplantation.\n3. Lymphoma, leukemia, myelodysplastic syndrome, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.\n4. Concomitant use of anticoagulants is prohibited if not on a stable regimen for at least 2 weeks prior to Day 1.\n5. Concomitant use of any of the following medications and not on a stable regimen (as judged by the Investigator) for the time period indicated prior to Screening:\n\n * Erythropoietin or immunosuppressants for at least 8 weeks\n * Systemic corticosteroids for at least 4 weeks\n * Vitamin K antagonists (for example, warfarin) with a stable international normalized ratio level for at least 4 weeks\n * Iron supplements or folic acid for 4 weeks\n6. Live vaccine(s) within 1 month prior to Screening or plans to receive such vaccines during the study.\n7. More than 1 LDH value \\> 2 × ULN within the 6 months prior to Day 1.\n8. Platelet count \\< 30,000/cubic millimeter (30 × 10\\^9/Liter \\[L\\]) at Screening.\n9. Absolute neutrophil count \\< 500/microliter (0.5 × 10\\^9/L) at Screening.'}, 'identificationModule': {'nctId': 'NCT04320602', 'briefTitle': 'Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab', 'orgStudyIdInfo': {'id': 'ALXN1210-PNH-401'}, 'secondaryIdInfos': [{'id': '2019-003440-74', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ravulizumab', 'description': 'Participants will receive eculizumab during the 3-month Screening Period. Participants will then switch over to and receive weight-based doses of ravulizumab for the duration of the study Treatment Period (351 days).', 'interventionNames': ['Biological: Eculizumab', 'Biological: Ravulizumab']}], 'interventions': [{'name': 'Eculizumab', 'type': 'BIOLOGICAL', 'otherNames': ['Soliris'], 'description': 'Participants must have been prescribed and be receiving a stable dose of eculizumab 1200 milligrams (mg) every 2 weeks (q2w) for at least 3 months prior to the Screening Period. During the Screening Period, participants will continue to receive eculizumab 1200 mg q2w.', 'armGroupLabels': ['Ravulizumab']}, {'name': 'Ravulizumab', 'type': 'BIOLOGICAL', 'otherNames': ['ALXN1210', 'Ultomiris'], 'description': "During the Treatment Period, participants will receive a loading dose of ravulizumab on Day 1, followed by maintenance doses on Day 15 and every 8 weeks, administered by intravenous infusion. Ravulizumab loading and maintenance doses will be based on participants' body weight per approved dose regimen.", 'armGroupLabels': ['Ravulizumab']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'LS9 7TF', 'city': 'Leeds', 'country': 'United Kingdom', 'facility': 'Clinical Study Site', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'zip': 'LS9 7TF', 'city': 'Leeds', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'zip': 'SE5 9RS', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Clinical Study Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE5 9RS', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}