Ignite Creation Date:
2025-12-25 @ 1:07 AM
Ignite Modification Date:
2025-12-25 @ 11:19 PM
Study NCT ID:
NCT04115293
Status:
COMPLETED
Last Update Posted:
2025-07-16
First Post:
2019-10-02
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}], 'ancestors': [{'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000719268', 'term': 'zilucoplan'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'UCBCares@ucb.com', 'phone': '001 844 599 2273', 'title': 'UCB', 'organization': 'Cares'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up])', 'description': 'A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.', 'otherNumAtRisk': 88, 'deathsNumAtRisk': 88, 'otherNumAffected': 34, 'seriousNumAtRisk': 88, 'deathsNumAffected': 1, 'seriousNumAffected': 13}, {'id': 'EG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.', 'otherNumAtRisk': 86, 'deathsNumAtRisk': 86, 'otherNumAffected': 41, 'seriousNumAtRisk': 86, 'deathsNumAffected': 1, 'seriousNumAffected': 11}], 'otherEvents': [{'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 11, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 18, 'numAffected': 14}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Lipase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Amylase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 19, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 16, 'numAffected': 13}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Myasthenia gravis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 9, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Aphthous ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Oesophageal candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'COVID-19 pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Herpes simplex meningoencephalitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Lipase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Metastases to meninges', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Basal cell carcinoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Cerebral haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Cerebrovascular accident', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Myasthenia gravis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Hyperemesis gravidarum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Chronic obstructive pulmonary disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Pulmonary embolism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}, {'term': 'Angioedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 88, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 86, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.30', 'groupId': 'OG000', 'lowerLimit': '-3.17', 'upperLimit': '-1.43'}, {'value': '-4.39', 'groupId': 'OG001', 'lowerLimit': '-5.28', 'upperLimit': '-3.50'}]}]}], 'analyses': [{'pValue': '<0.001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.09', 'ciLowerLimit': '-3.24', 'ciUpperLimit': '-0.95', 'statisticalMethod': 'MMRM ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.25', 'groupId': 'OG000', 'lowerLimit': '-4.32', 'upperLimit': '-2.17'}, {'value': '-6.19', 'groupId': 'OG001', 'lowerLimit': '-7.29', 'upperLimit': '-5.08'}]}]}], 'analyses': [{'pValue': '<0.001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.94', 'ciLowerLimit': '-4.39', 'ciUpperLimit': '-1.49', 'statisticalMethod': 'MMRM ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-5.42', 'groupId': 'OG000', 'lowerLimit': '-6.98', 'upperLimit': '-3.86'}, {'value': '-8.62', 'groupId': 'OG001', 'lowerLimit': '-10.22', 'upperLimit': '-7.01'}]}]}], 'analyses': [{'pValue': '0.0023', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-3.20', 'ciLowerLimit': '-5.24', 'ciUpperLimit': '-1.16', 'statisticalMethod': 'MMRM ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing \\[0 to 6\\], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.16', 'groupId': 'OG000', 'lowerLimit': '-4.65', 'upperLimit': '-1.67'}, {'value': '-5.65', 'groupId': 'OG001', 'lowerLimit': '-7.17', 'upperLimit': '-4.12'}]}]}], 'analyses': [{'pValue': '0.0128', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.49', 'ciLowerLimit': '-4.45', 'ciUpperLimit': '-0.54', 'statisticalMethod': 'MMRM ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': "The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement.", 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'The median time to first receipt of rescue therapy was not estimated due to the less number of events.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'The median time to first receipt of rescue therapy was not estimated due to the less number of events.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.', 'unitOfMeasure': 'Days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '5.8', 'groupId': 'OG000'}, {'value': '14.0', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0885', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.608', 'ciLowerLimit': '0.866', 'ciUpperLimit': '7.860', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '46.1', 'groupId': 'OG000'}, {'value': '73.1', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.184', 'ciLowerLimit': '1.662', 'ciUpperLimit': '6.101', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '33.0', 'groupId': 'OG000'}, {'value': '58.0', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0012', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.865', 'ciLowerLimit': '1.518', 'ciUpperLimit': '5.409', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}, {'value': '86', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'OG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'classes': [{'categories': [{'measurements': [{'value': '70.5', 'groupId': 'OG000'}, {'value': '76.7', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])', 'description': 'A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'FG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '88'}, {'groupId': 'FG001', 'numSubjects': '86'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '84'}, {'groupId': 'FG001', 'numSubjects': '82'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study started to enroll participants in September 2019 and concluded in December 2021.', 'preAssignmentDetails': 'The Participant flow refers to the Randomized Set.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'BG000'}, {'value': '86', 'groupId': 'BG001'}, {'value': '174', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.'}, {'id': 'BG001', 'title': 'Zilucoplan 0.3 mg/kg', 'description': 'Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '62', 'groupId': 'BG000'}, {'value': '64', 'groupId': 'BG001'}, {'value': '126', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '22', 'groupId': 'BG001'}, {'value': '48', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '53.3', 'spread': '15.7', 'groupId': 'BG000'}, {'value': '52.6', 'spread': '14.6', 'groupId': 'BG001'}, {'value': '53.0', 'spread': '15.1', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '47', 'groupId': 'BG000'}, {'value': '52', 'groupId': 'BG001'}, {'value': '99', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '41', 'groupId': 'BG000'}, {'value': '34', 'groupId': 'BG001'}, {'value': '75', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'American Indian/Alaska native', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '21', 'groupId': 'BG002'}]}]}, {'title': 'Black', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '13', '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': '62', 'groupId': 'BG000'}, {'value': '66', 'groupId': 'BG001'}, {'value': '128', 'groupId': 'BG002'}]}]}, {'title': 'Other/Mixed', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Missing', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Hispanic or Latino', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '79', 'groupId': 'BG000'}, {'value': '72', 'groupId': 'BG001'}, {'value': '151', 'groupId': 'BG002'}]}]}, {'title': 'Missing', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-12-18', 'size': 1957773, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-11-22T06:43', 'hasProtocol': True}, {'date': '2021-12-06', 'size': 1938923, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-11-22T06:43', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 174}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-09-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2021-12-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-07-04', 'studyFirstSubmitDate': '2019-10-02', 'resultsFirstSubmitDate': '2022-12-19', 'studyFirstSubmitQcDate': '2019-10-02', 'lastUpdatePostDateStruct': {'date': '2025-07-16', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-12-19', 'studyFirstPostDateStruct': {'date': '2019-10-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-01-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-12-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.'}, {'measure': 'Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing \\[0 to 6\\], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.'}, {'measure': 'Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': "The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement."}, {'measure': 'Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period', 'timeFrame': 'From Baseline to End of Treatment (Week 12)', 'description': 'Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.'}, {'measure': 'Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.'}, {'measure': 'Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.'}, {'measure': 'Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12', 'timeFrame': 'End of Treatment (Week 12)', 'description': 'Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.'}, {'measure': 'Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)', 'timeFrame': 'From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])', 'description': 'A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Myasthenia Gravis, Generalized']}, 'referencesModule': {'references': [{'pmid': '37059508', 'type': 'RESULT', 'citation': 'Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7.'}, {'pmid': '38400914', 'type': 'RESULT', 'citation': 'Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24.'}, {'pmid': '39314260', 'type': 'RESULT', 'citation': 'de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024.'}, {'pmid': '40504283', 'type': 'RESULT', 'citation': 'Hewamadduma C, Freimer M, Genge A, Leite MI, Utsugisawa K, Vu T, Boroojerdi B, Grimson F, Savic N, Vanderkelen M, Howard JF Jr; RAISE-XT study team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025 Jun 12;272(7):457. doi: 10.1007/s00415-025-13113-0.'}]}, 'descriptionModule': {'briefSummary': 'The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '74 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of gMG \\[Myasthenia Gravis Foundation of America (MGFA) Class II-IV\\] at Screening\n* Positive serology for acetylcholine receptor (AChR) autoantibodies\n* MG-ADL Score of ≥ 6 at Screening and Baseline\n* QMG score ≥ 12 at Screening and Baseline\n* No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period\n* No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period\n\nExclusion Criteria:\n\n* Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period\n* History of meningococcal disease\n* Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline'}, 'identificationModule': {'nctId': 'NCT04115293', 'acronym': 'RAISE', 'briefTitle': 'Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis', 'organization': {'class': 'INDUSTRY', 'fullName': 'UCB Pharma'}, 'officialTitle': 'A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis', 'orgStudyIdInfo': {'id': 'RA101495-02.301'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '0.3 mg/kg zilucoplan (RA101495)', 'interventionNames': ['Drug: zilucoplan (RA101495)']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'zilucoplan (RA101495)', 'type': 'DRUG', 'description': 'Daily subcutaneous (SC) injection', 'armGroupLabels': ['0.3 mg/kg zilucoplan (RA101495)']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Daily subcutaneous (SC) injection', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '36604', 'city': 'Mobile', 'state': 'Alabama', 'country': 'United States', 'facility': 'Site 41: Diagnostic and Medical Clinic', 'geoPoint': {'lat': 30.69436, 'lon': -88.04305}}, {'zip': '85028', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Site 116: Neuromuscular Clinic and Research Center', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90033', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Site 4: University of Southern California', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': 'Site 31: 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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, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}