Ignite Creation Date:
2025-12-24 @ 9:31 PM
Ignite Modification Date:
2026-01-04 @ 9:15 PM
Study NCT ID:
NCT04025632
Status:
TERMINATED
Last Update Posted:
2022-07-27
First Post:
2019-07-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
Sponsor:
Organization:
Raw JSON
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'seriousNumAffected': 8}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Sinus tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 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disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Faeces soft', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Lower gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 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'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Vaccination site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Vessel puncture site bruise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 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'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}], 'seriousEvents': [{'term': 'Ventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Rectal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Sinusitis bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Staphylococcal sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Rhinovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Liver function test increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 25, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (24.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-20.72', 'spread': '31.22', 'groupId': 'OG000'}, {'value': '-9.86', 'spread': '26.06', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.464', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Wilcoxon-Mann-Whitney odds', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.55', 'ciLowerLimit': '0.19', 'ciUpperLimit': '1.57', 'pValueComment': 'Conducted using a 2-sided Van Elteren test, which represents an extension of the Wilcoxon rank sum test for comparing 2 treatments in a stratified experiment using within-stratum ranks assigning greater weight to rank sums from smaller strata.', 'estimateComment': 'The magnitude of association between treatment groups was expressed as in Wilcoxon-Mann-Whitney odds followed by the 95% confidence intervals.', 'statisticalMethod': '2-sided Van Elteren test', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'All laboratory samples were obtained prior to administration of study drug at applicable visits. CK levels were measured by a central laboratory.', 'unitOfMeasure': 'percentage change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'PRIMARY', 'title': 'Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Main Portion: Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study.'}, {'id': 'OG001', 'title': 'Main Portion: Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) to end of Main Portion (Week 8)', 'description': 'A TEAE was defined as:\n\n* An adverse event (AE) that occurred after study treatment start that was not present at the time of treatment start.\n* An AE that increased in severity after treatment start if the event was present at the time of treatment start.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Population included all participants who have received at least 1 dose of study drug, with participants analyzed based on the actual study treatment received.'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.919', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.088', 'ciLowerLimit': '0.214', 'ciUpperLimit': '5.535', 'pValueComment': 'P-value for the comparison of treatment groups was calculated using logistic regression with investigational medicinal product and strata as fixed factors.', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The ACR/EULAR scale utilized a conjoint analysis-based continuous model using absolute percent change from Baseline in core set measures (physician, patient, and Myositis Disease Activity Assessment Tool (MDAAT); muscle strength; Health Assessment Questionnaire (HAQ); and muscle enzyme levels). A total improvement score (range 0-100) was determined by summing scores for each core set measure and comparing improvement in each respective core set measure. The threshold for minimal improvement was ≥20 in the total improvement score with higher scores indicating a better outcome.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.712', 'spread': '0.789', 'groupId': 'OG000'}, {'value': '-1.401', 'spread': '0.788', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.496', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Least squares (LS) mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.688', 'ciLowerLimit': '-2.781', 'ciUpperLimit': '1.404', 'pValueComment': 'Based on a linear model with treatment and strata (anti-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase \\[HMGCR\\]+/anti-signal recognition particle \\[SRP\\]+) as fixed factors with Baseline 3TUG as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The 3TUG test involved the ambulatory participant getting up from a seated position in a chair, walking at their normal pace for 3 meters, turning around, walking back to the chair, and sitting down. This sequence was repeated 3 times without rest, and the 3TUG test time is the average of the 3 lap times. A negative change from baseline indicated a better outcome.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8. The test was also only performed in participants who were ambulatory.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.18', 'spread': '3.44', 'groupId': 'OG000'}, {'value': '3.71', 'spread': '3.81', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.431', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.89', 'ciLowerLimit': '-6.18', 'ciUpperLimit': '13.95', 'pValueComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline proximal MMT as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The proximal MMT assessed muscle strength using manual muscle testing in 7 muscle groups (left and right sides assessed separately). The total MMT score for this study, inclusive of both sides, could range from 0-140, where 0 means no strength in any muscles and 140 means full strength in all the muscles examined. A negative change from Baseline indicated a worse outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.626', 'spread': '0.557', 'groupId': 'OG000'}, {'value': '-0.830', 'spread': '0.671', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.800', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.204', 'ciLowerLimit': '-1.855', 'ciUpperLimit': '1.448', 'pValueComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline Physician Global Activity VAS as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The Physician Global Activity VAS Score measured the treating physician\'s global evaluation of the participant\'s overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Physician Global Activity VAS Score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in Patient Global Activity VAS Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.685', 'spread': '0.707', 'groupId': 'OG000'}, {'value': '-1.966', 'spread': '0.854', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.221', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.281', 'ciLowerLimit': '-3.390', 'ciUpperLimit': '0.829', 'pValueComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline Patient Global Activity VAS as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The Patient Global Activity VAS Score measured the treating participant\'s global evaluation of their overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Patient Global Activity VAS score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in HAQ Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.022', 'spread': '0.151', 'groupId': 'OG000'}, {'value': '-0.125', 'spread': '0.183', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.508', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.147', 'ciLowerLimit': '-0.601', 'ciUpperLimit': '0.307', 'pValueComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline HAQ as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The HAQ had 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities with 2 to 3 questions for each section. Scoring within each section ranged from 0 (without any difficulty) to 3 (unable to do). The total HAQ score was then calculated by summing the scores and dividing by the number of categories answered. The total HAQ score for this study could range from 0-3, where 0 means no functional impairment and 3 means complete functional impairment. A negative change from Baseline indicated a better outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.144', 'spread': '0.336', 'groupId': 'OG000'}, {'value': '-0.287', 'spread': '0.398', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.765', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.143', 'ciLowerLimit': '-1.123', 'ciUpperLimit': '0.837', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline MDAAT as a covariate.'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The MDAAT extramuscular disease activity VAS score measured the degree of disease activity of extramuscular organ systems and muscle. The scoring was performed by the physician and ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.45', 'spread': '3.41', 'groupId': 'OG000'}, {'value': '8.98', 'spread': '4.08', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.265', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS mean difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '5.53', 'ciLowerLimit': '-4.49', 'ciUpperLimit': '15.55', 'pValueComment': 'Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline FACIT-Fatigue Scale as a covariate.', 'estimateComment': 'The difference presented is zilucoplan 0.3 mg/kg minus placebo.', 'statisticalMethod': 'Linear Mixed Effect Model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': "The FACIT-Fatigue Scale is a 13-item tool which measured an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue was measured on a 4-point Likert scale. The total FACIT-Fatigue Scale score for this study could range from 0-52, where 0 means the participants were very much fatigued during their usual daily activities and 52 means the participants were not at all fatigued during their usual daily activities. A negative change from Baseline indicated a worse outcome.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population with no missing observations at Baseline and Week 8.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'FG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'Completed Main Portion', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'Started Main Portion Safety Follow-up', 'comment': 'Refers to study participants who completed the 8-week blinded treatment period in the Main Portion of the study and had not entered the Extension Portion.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Completed Main Portion Safety Follow-up', 'comment': 'Refers to study participants who completed the 8-week blinded treatment period in the Main Portion of the study and had not entered the Extension Portion.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Started Extension Portion', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'comment': 'Completed Main Portion, Main Portion Safety Follow-up or Extension Portion.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '12'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Study Terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '9'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'This study was performed in 4 countries (France, the Netherlands, the United Kingdom, and the United States of America) between 07 November 2019 and 14 June 2021.', 'preAssignmentDetails': 'Of the 37 participants who were screened, 10 participants were deemed ineligible and were screen failures. 27 participants with immune-mediated necrotizing myopathy were randomized in a 1:1 ratio to receive zilucoplan 0.3 mg/kg or a matching placebo for the 8-week Treatment Period in the Main Portion of the study. All eligible participants were given the option to receive daily subcutaneous (SC) zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Participants were randomized to receive daily SC doses of matching placebo during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'BG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants were randomized to receive daily SC doses of zilucoplan 0.3 mg/kg during the 8-week Main Portion of the study. All eligible participants were given the option to receive daily SC zilucoplan 0.3 mg/kg in the Extension Portion of the study.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '52.8', 'spread': '13.6', 'groupId': 'BG000'}, {'value': '56.9', 'spread': '9.0', 'groupId': 'BG001'}, {'value': '54.6', 'spread': '11.8', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'American Indian/Alaska native', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Black or African American', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Native Hawaiian or other Pacific Islander', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'White', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}]}]}, {'title': 'Other/Mixed', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Unknown or Not Reported', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Missing', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Intent-to-Treat (ITT) Population included all randomized participants.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-02-16', 'size': 1927098, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-03-02T09:45', 'hasProtocol': True}, {'date': '2021-06-09', 'size': 1881303, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-03-02T09:46', 'hasProtocol': False}]}}, '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': 27}}, 'statusModule': {'whyStopped': 'The study was terminated based on the primary efficacy endpoint analysis after the first data lock.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-11-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-07', 'completionDateStruct': {'date': '2021-06-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-07-26', 'studyFirstSubmitDate': '2019-07-17', 'resultsFirstSubmitDate': '2022-03-03', 'studyFirstSubmitQcDate': '2019-07-17', 'lastUpdatePostDateStruct': {'date': '2022-07-27', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-04-21', 'studyFirstPostDateStruct': {'date': '2019-07-19', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2022-05-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-03-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'All laboratory samples were obtained prior to administration of study drug at applicable visits. CK levels were measured by a central laboratory.'}, {'measure': 'Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)', 'timeFrame': 'Baseline (Day 1) to end of Main Portion (Week 8)', 'description': 'A TEAE was defined as:\n\n* An adverse event (AE) that occurred after study treatment start that was not present at the time of treatment start.\n* An AE that increased in severity after treatment start if the event was present at the time of treatment start.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The ACR/EULAR scale utilized a conjoint analysis-based continuous model using absolute percent change from Baseline in core set measures (physician, patient, and Myositis Disease Activity Assessment Tool (MDAAT); muscle strength; Health Assessment Questionnaire (HAQ); and muscle enzyme levels). A total improvement score (range 0-100) was determined by summing scores for each core set measure and comparing improvement in each respective core set measure. The threshold for minimal improvement was ≥20 in the total improvement score with higher scores indicating a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The 3TUG test involved the ambulatory participant getting up from a seated position in a chair, walking at their normal pace for 3 meters, turning around, walking back to the chair, and sitting down. This sequence was repeated 3 times without rest, and the 3TUG test time is the average of the 3 lap times. A negative change from baseline indicated a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The proximal MMT assessed muscle strength using manual muscle testing in 7 muscle groups (left and right sides assessed separately). The total MMT score for this study, inclusive of both sides, could range from 0-140, where 0 means no strength in any muscles and 140 means full strength in all the muscles examined. A negative change from Baseline indicated a worse outcome.'}, {'measure': 'Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The Physician Global Activity VAS Score measured the treating physician\'s global evaluation of the participant\'s overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Physician Global Activity VAS Score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in Patient Global Activity VAS Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The Patient Global Activity VAS Score measured the treating participant\'s global evaluation of their overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Patient Global Activity VAS score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in HAQ Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The HAQ had 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities with 2 to 3 questions for each section. Scoring within each section ranged from 0 (without any difficulty) to 3 (unable to do). The total HAQ score was then calculated by summing the scores and dividing by the number of categories answered. The total HAQ score for this study could range from 0-3, where 0 means no functional impairment and 3 means complete functional impairment. A negative change from Baseline indicated a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': 'The MDAAT extramuscular disease activity VAS score measured the degree of disease activity of extramuscular organ systems and muscle. The scoring was performed by the physician and ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.'}, {'measure': 'Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score', 'timeFrame': 'Baseline (Day 1) and end of Main Portion (Week 8)', 'description': "The FACIT-Fatigue Scale is a 13-item tool which measured an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue was measured on a 4-point Likert scale. The total FACIT-Fatigue Scale score for this study could range from 0-52, where 0 means the participants were very much fatigued during their usual daily activities and 52 means the participants were not at all fatigued during their usual daily activities. A negative change from Baseline indicated a worse outcome."}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Immune Mediated Necrotizing Myopathy']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM). Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or matching placebo for 8 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Clinical diagnosis of IMNM (Immune-Mediated Necrotizing Myopathy)\n* Positive serology for anti-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase (HMGCR) or anti-signal recognition particle (SRP) autoantibodies\n* Clinical evidence of weakness (≤ grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group\n* Creatine kinase (CK) of \\>1000 U/L at Screening\n* No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study\n* No changes in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study\n\nExclusion Criteria:\n\n* History of meningococcal disease\n* Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening\n* Recent initiation of intravenous immunoglobulin (IVIG) (i.e., first cycle administered less than 90 days prior to Baseline)\n* Rituximab use within 90 days prior to Baseline or anticipated to occur during study\n* Statin use within 30 days prior to Baseline or anticipated to occur during study\n* Plasma exchange within 4 weeks prior to Baseline or expected to occur during the 8-week Treatment Period'}, 'identificationModule': {'nctId': 'NCT04025632', 'briefTitle': 'Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy', 'organization': {'class': 'INDUSTRY', 'fullName': 'UCB Pharma'}, 'officialTitle': 'A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy', 'orgStudyIdInfo': {'id': 'RA101495-02.202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '0.3 mg/kg zilucoplan', 'description': 'Daily subcutaneous (SC) injection', 'interventionNames': ['Drug: zilucoplan']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Daily subcutaneous (SC) injection', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'zilucoplan', 'type': 'DRUG', 'description': 'Daily subcutaneous (SC) inection', 'armGroupLabels': ['0.3 mg/kg zilucoplan']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Daily subcutaneous (SC) inection', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': 'University of California Irvine', 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '32209', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida Health Jacksonville', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'University of South Florida', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'National Institute of Health', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine in Saint Louis', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '11021', 'city': 'Great Neck', 'state': 'New York', 'country': 'United States', 'facility': 'Northwell Health Neuroscience Institute', 'geoPoint': {'lat': 40.80066, 'lon': -73.72846}}, {'zip': '43221', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Ohio State University', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '38018', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Wesley Neurology Clinic', 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}, {'zip': '78756', 'city': 'Austin', 'state': 'Texas', 'country': 'United States', 'facility': 'Austin Neuromuscular Center', 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Health Science Center at Houston', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Universitaire Pitié Salpêtrière', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Amsterdam UMC', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': 'WC1N3BG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'University College London Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M6 8HD', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Salford Royal NHS Barnes Clinical Research Facility', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'UCB Cares', 'role': 'STUDY_DIRECTOR', 'affiliation': 'UCB Pharma'}]}, 'ipdSharingStatementModule': {'url': 'http://www.Vivli.org', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'timeFrame': 'Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.', 'ipdSharing': 'YES', 'description': 'Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.', 'accessCriteria': 'Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ra Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}