Ignite Creation Date:
2025-12-25 @ 2:59 AM
Ignite Modification Date:
2026-04-11 @ 7:47 AM
Study NCT ID:
NCT02588833
Status:
COMPLETED
Last Update Posted:
2021-01-11
First Post:
2015-10-27
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006456', 'term': 'Hemoglobinuria'}, {'id': 'D006461', 'term': 'Hemolysis'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D011507', 'term': 'Proteinuria'}, {'id': 'D014555', 'term': 'Urination Disorders'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D020924', 'term': 'Urological Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D010335', 'term': 'Pathologic Processes'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000716074', 'term': 'pegcetacoplan'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@apellis.com', 'phone': '1-833-284-6361', 'title': 'Apellis Clinical Trial Information Line', 'organization': 'Apellis Pharmaceuticals, Inc'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.', 'description': 'The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.', 'eventGroups': [{'id': 'EG000', 'title': 'Cohort 1', 'description': 'Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 2, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.', 'otherNumAtRisk': 20, 'deathsNumAtRisk': 20, 'otherNumAffected': 17, 'seriousNumAtRisk': 20, 'deathsNumAffected': 1, 'seriousNumAffected': 6}], 'otherEvents': [{'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasal abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Ophthalmic herpes zoster', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Paronychia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pleural effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Injection site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gingival bleeding', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Electrocardiogram QT prolonged', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Blood alkaline phosphatase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Blood lactate dehydrogenase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Liver function test abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Serum ferritin decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rash maculo-papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dry skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Petechiae', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Purpura', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Skin hypopigmentation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 6, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Febrile neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haemolytic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Chromaturia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Chronic kidney disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Post procedural haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Road traffic accident', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypoalbuminaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cataract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Conjunctival haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Dry eye', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cholangitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hepatic steatosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Uterine haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'seriousEvents': [{'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal neoplasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Aplastic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Intravascular haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Joint dislocation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Pneumonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Paroxysmal nocturnal haemoglobinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Cholecystitis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': 'Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'title': 'All TEAEs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}]}, {'title': 'Treatment related TEAEs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Serious AEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs leading to study drug discontinuation', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs leading to death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs with mild intensity', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs with moderate intensity', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs with severe intensity', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs with life threatening intensity', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.', 'description': 'TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2105.2', 'spread': '1078.79', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.', 'unitOfMeasure': 'units per liter (U/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'PRIMARY', 'title': 'Mean Percentage Change From Baseline in LDH at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-84.8', 'spread': '14.04', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'PRIMARY', 'title': 'Mean Change From Baseline in Haptoglobin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.066', 'spread': '0.1245', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'gram per liter (g/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'PRIMARY', 'title': 'Mean Percentage Change From Baseline in Haptoglobin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '166.176', 'spread': '311.3656', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'PRIMARY', 'title': 'Mean Change From Baseline in Hemoglobin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.68', 'spread': '2.690', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'g/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'PRIMARY', 'title': 'Mean Percentage Change From Baseline in Hemoglobin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '49.86', 'spread': '43.254', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.1', 'spread': '11.09', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': "The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-105.9', 'spread': '70.28', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.', 'unitOfMeasure': '10^9 cells/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Mean Percentage Change From Baseline in ARC at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-47.5', 'spread': '26.86', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in Total Bilirubin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-29.9', 'spread': '24.34', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'micromole per liter (umol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Mean Percentage Change From Baseline in Total Bilirubin at Day 365', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '-60.9', 'spread': '19.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported.'}, {'type': 'SECONDARY', 'title': 'Number of Subjects Receiving Red Blood Cell (RBC) Transfusions', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': 'Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From Day 1 up to Day 533.', 'description': 'The number of on-study RBC transfusions were monitored throughout the treatment period.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cohort 1', 'description': 'Subjects received subcutaneous (SC) injections of pegcetacoplan 180 milligrams (mg)/day for up to 28 days.'}, {'id': 'FG001', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}], 'periods': [{'title': 'Part 1', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'One subject was enrolled in both cohorts, this subject was enrolled twice.', 'groupId': 'FG000', 'numSubjects': '3'}, {'comment': 'One subject was enrolled in both cohorts, this subject was enrolled twice.', 'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Part 2A', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Part 2A is not applicable since Cohort 1 subjects proceeded immediately to Part 3.', 'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Part 2B', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '18'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Part 2C', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'comment': '17 subjects completed Part 2B and 3 entered extension study without participating in Part 2C.', 'groupId': 'FG001', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Part 3 (Safety Follow-up)', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Only subjects who did not enter extension study or Part 2C entered Part 3.', 'groupId': 'FG000', 'numSubjects': '3'}, {'comment': 'Only subjects who did not enter extension study or Part 2C entered Part 3.', 'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Other', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'This Phase 1b, pilot study was conducted at 9 sites in 5 countries (Hong Kong, Malaysia, New Zealand, Thailand and United States) in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who had not received treatment with eculizumab (Soliris®) in the past, between 01 December 2015 and 26 August 2019. A total of 22 subjects were enrolled.', 'preAssignmentDetails': 'Subjects could participate in more than 1 cohort. The study consisted of 3 parts: Part 1 for Cohorts 1 and 2, Part 2 (Part 2A, Part 2B and Part 2C) for Cohort 2 only and Part 3 (safety follow-up). Cohort 2 dosing was not initiated until all subjects in Cohort 1 had completed Part 1 and review of emerging safety and efficacy data. Overall, 2 cohorts of 22 unique subjects were evaluated.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Cohort 1', 'description': 'Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days.'}, {'id': 'BG001', 'title': 'Cohort 2', 'description': 'Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day.'}, {'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': '3', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Maori', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}]}, {'title': 'Unknown', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The Intent-To-Treat (ITT) population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. In this study, subjects could participate in more than 1 cohort. Overall, 2 cohorts of 22 unique subjects were evaluated as one subject participated in cohorts 1 and 2.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-10-25', 'size': 1701245, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-07-22T14:05', 'hasProtocol': True}, {'date': '2019-12-19', 'size': 1202571, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-07-22T14:06', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-12-01', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2020-12', 'completionDateStruct': {'date': '2019-08-26', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-12-14', 'studyFirstSubmitDate': '2015-10-27', 'resultsFirstSubmitDate': '2020-07-31', 'studyFirstSubmitQcDate': '2015-10-27', 'lastUpdatePostDateStruct': {'date': '2021-01-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-12-14', 'studyFirstPostDateStruct': {'date': '2015-10-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-01-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-08-26', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity', 'timeFrame': 'From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.', 'description': 'TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.'}, {'measure': 'Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.'}, {'measure': 'Mean Percentage Change From Baseline in LDH at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.'}, {'measure': 'Mean Change From Baseline in Haptoglobin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.'}, {'measure': 'Mean Percentage Change From Baseline in Haptoglobin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.'}, {'measure': 'Mean Change From Baseline in Hemoglobin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.'}, {'measure': 'Mean Percentage Change From Baseline in Hemoglobin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.'}], 'secondaryOutcomes': [{'measure': 'Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': "The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only."}, {'measure': 'Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.'}, {'measure': 'Mean Percentage Change From Baseline in ARC at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.'}, {'measure': 'Mean Change From Baseline in Total Bilirubin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.'}, {'measure': 'Mean Percentage Change From Baseline in Total Bilirubin at Day 365', 'timeFrame': 'Baseline (Day 1) and Day 365.', 'description': 'Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.'}, {'measure': 'Number of Subjects Receiving Red Blood Cell (RBC) Transfusions', 'timeFrame': 'From Day 1 up to Day 533.', 'description': 'The number of on-study RBC transfusions were monitored throughout the treatment period.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PNH', 'Paroxysmal Nocturnal Hemoglobinuria', 'Complement inhibitor', 'Anemia', 'Hemoglobinuria', 'Hemolysis', 'Hematologic diseases', 'Extravascular hemolysis (EVH)', 'Intravascular hemolysis (IVH)', 'C3 inhibitor'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'referencesModule': {'references': [{'pmid': '39079163', 'type': 'DERIVED', 'citation': 'Panse J, Daguindau N, Okuyama S, Peffault de Latour R, Schafhausen P, Straetmans N, Al-Adhami M, Persson E, Wong RSM. Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin >/=10 g/dL) who had received eculizumab or were naive to complement inhibitors. PLoS One. 2024 Jul 29;19(7):e0306407. doi: 10.1371/journal.pone.0306407. eCollection 2024.'}, {'pmid': '35869170', 'type': 'DERIVED', 'citation': 'Wong RSM, Pullon HWH, Amine I, Bogdanovic A, Deschatelets P, Francois CG, Ignatova K, Issaragrisil S, Niparuck P, Numbenjapon T, Roman E, Sathar J, Xu R, Al-Adhami M, Tan L, Tse E, Grossi FV. Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2022 Sep;101(9):1971-1986. doi: 10.1007/s00277-022-04903-x. Epub 2022 Jul 22.'}]}, 'descriptionModule': {'briefSummary': 'The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past.\n\nAn exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.', 'detailedDescription': "This is a Phase Ib, open-label, multiple ascending dose, pilot study in patients with PNH who have not received eculizumab (Soliris)® in the past. Two cohorts of 3 subjects are planned for evaluation.\n\nSafety will be assessed throughout the study; serial blood and urine samples will be collected for these assessments. Blood samples will be collected for the assessment of pegcetacoplan PK. Additional samples for assessment of PD will also be collected.\n\nThe study will consist of four parts;\n\n* Part 1: subjects will receive pegcetacoplan for 28 days.\n* Part 2A: subjects may continue to receive pegcetacoplan for a further 56 days if there is evidence of perceived clinical benefit following review of available safety, PK and PD data by the investigator and sponsor\n* Part 2B/Part 2C: subjects may continue to receive daily pegcetacoplan treatment for up to 364 days if there is ongoing evidence of clinical benefit following review of the available safety, PK and PD data. After completion of Part 2B, subjects could continue treatment with pegcetacoplan by transitioning to an open-label extension study or Part 2C, if enrollment into the extension study was not yet available. Subjects entering Part 2C continued their pegcetacoplan dose and regimen until enrollment in the open-label extension study was available.\n* Part 3: Safety follow up Screening will take place within 30 days prior to the start of dosing on Day 1. If needed (see inclusion criteria), Neisseria menigitides types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively), and Haemophilus influenzae Type B (Hib) vaccinations will be administered at least 14 days prior to dosing on Day 1.\n\nSubjects will be entered into Part 1 of the study on Day 1 at a time designated by the PI. During Part 1, the first 3 daily SC doses of pegcetacoplan (Day 1 to 3) as well as doses on Day 8, 15 and 22 will be administered at the clinical site. From Day 4 to Day 28 daily doses of pegcetacoplan will be administered off-site by a trained study nurse at the subject's home, workplace, or other location convenient to the subject with the exception of those days where dosing is at the clinical site. Following ongoing review of available safety, PK and PD data by the investigator and sponsor, subjects showing evidence of perceived clinical benefit may progress to Part 2A and then to Part 2B of the study and continue to receive daily doses of pegcetacoplan until Day 84 and then until Day 364.\n\nCohort 2 will not be initiated until all subjects in Cohort 1 have reached the Day 29 visit and the SMC has reviewed emerging safety and efficacy data and determined that, at the initial dose, pegcetacoplan has an acceptable safety and tolerability profile.\n\nThe planned length of participation in the study for each subject in Cohort 2 is approximately 444 days (14.5 months) from Day 30 through completion of the Day 414 Exit visit procedures).\n\nThe study is planned to take place over approximately 24 months (from screening of Cohort 1 through completion of Cohort 2).\n\nThis time period may change in the event that the study is terminated early, additional cohorts are enrolled, additional time is required to review safety between cohorts, or extended safety and PK sampling is added for a cohort."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female\n* At least 18 years old (inclusive)\n* Weigh \\>55 kg and have a body mass index (BMI) ≤38.0 kg/m2\n* Diagnosed with PNH (white blood cell (WBC) clone \\>10%)\n* Lactose dehydrogenase (LD) ≥2 times the upper limit of normal\n* Last transfusion within 12 months prior to screening\n* Platelet count of \\>30,000/mm3\n* Absolute neutrophil count \\>cells/500 µL\n* Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study\n* Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study\n* Able to provide documentary evidence of Neisseria meningitidis, Pneumococcal conjugate vaccine (multivalent) or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 dosing, OR willing to receive vaccinations against Neisseria meningitidis at least two weeks prior to dosing on Day 1 with a booster on Day 57, and PCV13 and Hib vaccines at least two weeks prior to dosing on Day 1.\n* Willing and able to give informed consent\n\nExclusion Criteria:\n\n* Prior eculizumab (Soliris)® treatment\n* Active bacterial infection\n* Known infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)\n* Hereditary complement deficiency\n* History of bone marrow transplantation\n* Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.\n* Participation in any other investigational drug trial or exposure to another investigational agent, device or procedure within 30 days\n* Evidence of QTcF prolongation defined as \\>450 ms for males and \\>470 ms for females at screening\n* Creatinine clearance (CrCl) \\<50 mL/min (Cockcroft-Gault formula) at screening\n* Breast-feeding women\n* History of meningococcal disease'}, 'identificationModule': {'nctId': 'NCT02588833', 'acronym': 'PADDOCK', 'briefTitle': 'Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Apellis Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)', 'orgStudyIdInfo': {'id': 'APL2-CP-PNH-204'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': '180 mg pegcetacoplan/day', 'interventionNames': ['Drug: Pegcetacoplan']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': '270 mg pegcetacoplan/day', 'interventionNames': ['Drug: Pegcetacoplan']}], 'interventions': [{'name': 'Pegcetacoplan', 'type': 'DRUG', 'otherNames': ['APL-2'], 'description': 'Complement (C3) Inhibitor', 'armGroupLabels': ['Cohort 1', 'Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Hong Kong', 'country': 'Hong Kong', 'facility': 'Prince of Wales Hospital', 'geoPoint': {'lat': 22.27832, 'lon': 114.17469}}, {'city': 'Hong Kong', 'country': 'Hong Kong', 'facility': 'Queen Mary Hospital', 'geoPoint': {'lat': 22.27832, 'lon': 114.17469}}, {'zip': '68000', 'city': 'Ampang', 'state': 'Selangor', 'country': 'Malaysia', 'facility': 'Hospital Ampang'}, {'zip': '3240', 'city': 'Hamilton', 'state': 'Waikato Region', 'country': 'New Zealand', 'facility': 'Waikato Hospital', 'geoPoint': {'lat': -37.78333, 'lon': 175.28333}}, {'zip': '10400', 'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Ramathibodi Hospital', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}, {'zip': '10700', 'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Siriraj hospital', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}, {'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Phramongkutklao Hospital', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}], 'overallOfficials': [{'name': 'Federico Grossi, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Apellis Pharmaceuticals, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Apellis Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}