Ignite Creation Date:
2025-12-25 @ 2:45 AM
Ignite Modification Date:
2026-02-23 @ 8:56 PM
Study NCT ID:
NCT02604433
Status:
COMPLETED
Last Update Posted:
2023-04-18
First Post:
2015-10-21
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Algeria', 'Germany', 'Morocco', 'Saudi Arabia']}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000621232', 'term': 'luspatercept'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Clinical.Trials@bms.com', 'phone': 'Please email', 'title': 'Bristol-Myers Squibb Study Director', 'organization': 'Bristol-Myers Squibb'}, 'certainAgreement': {'otherDetails': "Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'All-cause mortality was assessed from first dose to study completion (approximately 56 months) SAEs and NSAEs were assessed from first dose to 90 days following last dose (up to approximately 52 months)', 'description': 'Adverse events were collected in all participants who received at least 1 dose of study drug.', 'eventGroups': [{'id': 'EG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care', 'otherNumAtRisk': 223, 'deathsNumAtRisk': 223, 'otherNumAffected': 211, 'seriousNumAtRisk': 223, 'deathsNumAffected': 4, 'seriousNumAffected': 53}, {'id': 'EG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care', 'otherNumAtRisk': 109, 'deathsNumAtRisk': 109, 'otherNumAffected': 95, 'seriousNumAtRisk': 109, 'deathsNumAffected': 1, 'seriousNumAffected': 8}], 'otherEvents': [{'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 30}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 41}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 14}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 19}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 29}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 30}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 26}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 11}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 38}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 16}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 21}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 47}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 24}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 18}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 36}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 15}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 16}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 93}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 43}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Transfusion reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 5}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Liver iron concentration increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hyperuricaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 16}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 51}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 15}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 72}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 33}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Bone pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 50}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 9}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 8}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 21}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 11}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 28}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 11}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 9}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Osteoporosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 33}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 12}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Spinal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 29}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 78}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 27}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Menstruation irregular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 6}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 48}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 13}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 21}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 41}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 13}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 4}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 23}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Extramedullary haemopoiesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Febrile neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pancytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cardiac arrest', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cardiac failure congestive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cardiac iron overload', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Left ventricular dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Myocardial ischaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Food poisoning', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pancreatitis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hyperpyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cholangitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cholecystitis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 2}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Drug-induced liver injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gallbladder obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hepatitis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Portal vein thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Acute sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dengue fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Epstein-Barr virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gallbladder empyema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Large intestine infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Neutropenic sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Parotid abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pharyngitis bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pulmonary sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyelonephritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Septic shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Splenic abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Urosepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Vestibular neuronitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Viral sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Wound infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Femur fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Radius fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Thermal burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Thoracic vertebral fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Traumatic fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Blood uric acid increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Coombs direct test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Urine albumin/creatinine ratio increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Acute erythroid leukaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hepatocellular carcinoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Meningioma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Cerebral venous sinus thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Depressed level of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Haemorrhage intracranial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Intracranial aneurysm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Neuritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Spinal cord compression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Thrombotic stroke', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Transient ischaemic attack', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Renal colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Renal injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pulmonary embolism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pulmonary hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Deep vein thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Thrombophlebitis superficial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 109, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '21.0', 'groupId': 'OG000'}, {'value': '4.5', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '5.62', 'ciLowerLimit': '2.17', 'ciUpperLimit': '14.53', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Odds ratio 95% confidence intervals (CIs), and p-value were estimated from the Cochran Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization.', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}, {'groupIds': ['OG000', 'OG001'], 'paramType': 'Difference in Percentages', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '16.5', 'ciLowerLimit': '10.0', 'ciUpperLimit': '23.1', 'estimateComment': 'Luspatercept - Placebo', 'nonInferiorityType': 'SUPERIORITY'}, {'groupIds': ['OG000', 'OG001'], 'paramType': 'Common Risk Difference', 'ciPctValue': '95', 'paramValue': '16.5', 'ciLowerLimit': '9.9', 'ciUpperLimit': '23.1', 'estimateComment': 'Luspatercept - Placebo', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Erythroid Response was defined as red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Week 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '19.6', 'groupId': 'OG000'}, {'value': '3.6', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '6.44', 'ciLowerLimit': '2.27', 'ciUpperLimit': '18.26', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Odds ratio 95% CIs, and p-value were estimated from the CMH test stratified by the geographical regions defined at randomization. To control the overall Type 1 error rate for outcomes 2-4, the testing procedure was implemented strictly in order: the test for this outcome was only conducted when there was evidence showing that erythroid response was achieved in the luspatercept group from Week 13 to Week 24 (primary endpoint).', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Weeks 37 - 48 compared to the 12-week interval on or prior to Dose 1 Day 1.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '7.1', 'groupId': 'OG000'}, {'value': '1.8', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0402', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '4.24', 'ciLowerLimit': '0.96', 'ciUpperLimit': '18.79', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Odds ratio, 95% CIs, and p-value were estimated from the Cochran-Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization. To control the overall Type 1 error rate, the testing procedure was done strictly in order: the test for this outcome was only conducted when there was evidence showing erythroid response was achieved in the luspatercept group for the primary endpoint, and 33% hematological improvement was achieved in the luspatercept group in outcome 2.', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Weeks 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '10.3', 'groupId': 'OG000'}, {'value': '0.9', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0017', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '11.92', 'ciLowerLimit': '1.65', 'ciUpperLimit': '86.29', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Odds ratio, 95% CIs, and p-value were estimated from the Cochran-Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization. To control the overall Type 1 error rate, the testing procedure was done strictly in order: the test for this outcome was only conducted when there was evidence showing erythroid response was achieved in the luspatercept group for the primary endpoint, and achievement of objective in the luspatercept group in outcomes 2+3.', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.67', 'spread': '1.792', 'groupId': 'OG000'}, {'value': '0.66', 'spread': '1.774', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LSM Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.34', 'ciLowerLimit': '-1.76', 'ciUpperLimit': '-0.93', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Change from baseline at Week 48 LSM = least squares mean', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline transfusion burden as covariates.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24.', 'unitOfMeasure': 'RBC units', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline In Liver Iron Concentration (LIC) At Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '185', 'groupId': 'OG000'}, {'value': '99', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '0.05', 'spread': '5.770', 'groupId': 'OG000'}, {'value': '-0.00', 'spread': '5.329', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.7598', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.20', 'ciLowerLimit': '-1.10', 'ciUpperLimit': '1.51', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Change from baseline at Week 48 P-value ANCOVA model with geographical regions defined at randomization and baseline LIC as covariates. LS = least square', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Week -12 to Day -1; Treatment: Week 48', 'description': 'Baseline was defined as the last value on or before the first dose of study drug was administered; if multiple values were present for the same date, the average of these values was used. If a participant had 1 postbaseline assessment, it was used as the Week 48 value. If a participant had multiple postbaseline assessments, the last one was used as the Week 48 value. The value of LIC was collected by magnetic resonance imaging. Participants with a LIC value \\> 43 mg/g were not included in the analysis.', 'unitOfMeasure': 'mg/g dry weight', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '129', 'groupId': 'OG000'}, {'value': '66', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Deferasirox', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}, {'value': '49', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-105.0', 'spread': '378.67', 'groupId': 'OG000'}, {'value': '-60.4', 'spread': '297.11', 'groupId': 'OG001'}]}]}, {'title': 'Deferiprone', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-229.1', 'spread': '893.62', 'groupId': 'OG000'}, {'value': '-73.4', 'spread': '614.80', 'groupId': 'OG001'}]}]}, {'title': 'Deferoxamine Mesilate/Deferoxamine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '84.9', 'spread': '523.45', 'groupId': 'OG000'}, {'value': '274.2', 'spread': '613.05', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.2552', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-68.0', 'ciLowerLimit': '-185.8', 'ciUpperLimit': '49.7', 'pValueComment': 'Significance level of 0.050 for 2-sided tests', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Deferasirox: Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.'}, {'pValue': '0.7746', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-76.4', 'ciLowerLimit': '-612.9', 'ciUpperLimit': '460.1', 'pValueComment': 'Significance level of 0.050 for 2-sided tests', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Deferiprone: Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.'}, {'pValue': '0.5186', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-147.3', 'ciLowerLimit': '-673.1', 'ciUpperLimit': '378.5', 'pValueComment': 'Significance level of 0.050 for 2-sided tests', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Deferoxamine Mesilate / Deferoxamine: Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants.', 'unitOfMeasure': 'mg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline In Mean Serum Ferritin At Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '207', 'groupId': 'OG000'}, {'value': '101', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '-247.19', 'spread': '713.767', 'groupId': 'OG000'}, {'value': '100.38', 'spread': '522.047', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-342.59', 'ciLowerLimit': '-498.30', 'ciUpperLimit': '-186.87', 'pValueComment': 'Significance level of 0.050 for 2-sided tests', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates based on an ANCOVA model with geographical regions defined at randomization and baseline serum ferritin as covariates.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'For each participant, the baseline mean serum ferritin level was calculated during the 12 weeks prior to first study drug administration. The postbaseline mean serum ferritin level was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or last 12 weeks of study treatment, if discontinued early. The change was calculated as the difference of post baseline mean serum ferritin level and baseline mean serum ferritin level.', 'unitOfMeasure': 'μg/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '190', 'groupId': 'OG000'}, {'value': '97', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Total Hip', 'denoms': [{'units': 'Participants', 'counts': [{'value': '190', 'groupId': 'OG000'}, {'value': '97', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.01', 'spread': '0.050', 'groupId': 'OG000'}, {'value': '0.01', 'spread': '0.057', 'groupId': 'OG001'}]}]}, {'title': 'Lumbar Spine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '189', 'groupId': 'OG000'}, {'value': '97', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.00', 'spread': '0.063', 'groupId': 'OG000'}, {'value': '0.00', 'spread': '0.078', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.9201', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.00', 'ciLowerLimit': '-0.01', 'ciUpperLimit': '0.01', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Total Hip Bone Mineral Density: Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline BMD measurement as covariates.'}, {'pValue': '0.4620', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.01', 'ciLowerLimit': '-0.02', 'ciUpperLimit': '0.01', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Lumbar Spine Bone Mineral Density: Change from baseline at Week 48 LS = least squares', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline BMD measurement as covariates.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Day 1; Treatment: Week 48', 'description': "For BMD, the lumbar spine and total hip were measured at baseline and 48 weeks by dual energy x-ray absorptiometry (DXA). Baseline was defined as the last value on or before the first dose of study drug is administered; if multiple values are present for the same date, the average of these values was used. If during the 48 week double-blinded treatment period, a participant has only one assessment, it is counted as 'Week 48' visit; if a participant has multiple assessments, the last one is used as 'Week 48' visit. The analysis was done on the population that had at least 2 measurements.", 'unitOfMeasure': 'gm/cm^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline In Myocardial Iron At Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '201', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.83', 'spread': '15.084', 'groupId': 'OG000'}, {'value': '-0.01', 'spread': '6.780', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0543', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean of Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.22', 'ciLowerLimit': '-4.48', 'ciUpperLimit': '0.04', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': 'Change from baseline at Week 48 LS = least square', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline myocardial T2\\* as covariates.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: Day 1; Treatment: Week 48', 'description': 'Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\\* (Unit: ms). T2\\* values correlates with heart failure (HF) risk (e.g. T2\\*\\<6ms: high HF risk).', 'unitOfMeasure': 'ms', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available measurements at the specified timepoints'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in the Transfusion-dependent Quality of Life (TranQol) Questionnaire At Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '200', 'groupId': 'OG000'}, {'value': '94', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Physical Health Domain Score - Change from Baseline', 'categories': [{'measurements': [{'value': '-1.5', 'spread': '14.26', 'groupId': 'OG000'}, {'value': '-0.7', 'spread': '14.24', 'groupId': 'OG001'}]}]}, {'title': 'Total Score - Change from Baseline', 'categories': [{'measurements': [{'value': '0.8', 'spread': '11.56', 'groupId': 'OG000'}, {'value': '-0.4', 'spread': '11.62', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.666', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Physical Health Domain Score - Change from Baseline at Week 24', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.384', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Total Score - Change from Baseline at Week 24', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: 4 weeks prior to Day 1; Treatment: Week 24', 'description': 'The TranQol is a self-administered quality of life tool developed for beta-thalassemia patients. The adult self-report version used in this study, includes 36 questions assessed on a 5-point response, that are grouped into 5 domains (Physical Health, Emotional Health, Sexual Health, Family Functioning, School/Career Functioning). Scores are calculated according to specific scoring algorithms developed by the authors. Both individual domains score and the total score range from 0 (worst) to 100 (best). Total Score and Physical Health domain score are reported. Positive change from baseline values indicate improvement.', 'unitOfMeasure': 'Score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with an evaluable TranQoL questionnaire at screening visit and at least one post-screening visit'}, {'type': 'SECONDARY', 'title': 'Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire At Weeks 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'OG000'}, {'value': '103', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Physical Functioning Domain Score - Change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '197', 'groupId': 'OG000'}, {'value': '91', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.3', 'spread': '6.93', 'groupId': 'OG000'}, {'value': '-0.2', 'spread': '7.86', 'groupId': 'OG001'}]}]}, {'title': 'General Health Domain Score - Change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '197', 'groupId': 'OG000'}, {'value': '91', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '7.18', 'groupId': 'OG000'}, {'value': '0.3', 'spread': '7.03', 'groupId': 'OG001'}]}]}, {'title': 'PCS - Change from Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '197', 'groupId': 'OG000'}, {'value': '91', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.4', 'spread': '7.01', 'groupId': 'OG000'}, {'value': '-0.3', 'spread': '7.97', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.918', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Physical Functioning Domain - Change from Baseline at Week 24', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.857', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'General Health Domain - Change from Baseline at Week 24', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.839', 'groupIds': ['OG000', 'OG001'], 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'PCS - Change from Baseline at Week 24', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24', 'description': 'The SF-36 (version 2) is a generic, self-administered instrument consisting of 8 multi-item scales that assess 8 health domains. The raw score for each health domain is transformed into a 0 (worst) to 100 (best) domain score. The 0-100 scale score for each health domain is further converted to normbased scores using a T-score transformation, with a mean of 50 and a standard deviation (SD) of 10. Higher norm-based T-scores indicate better heath/QoL. The domains/summaries reported are: 1. Physical Functioning (Range of possible T-scores is 19.26 - 57.54) 2. General Health (Range of possible T-scores is 18.95 - 66.50) 3. Physical Component summary (PCS) (Range of possible T-scores is 5.02 - 79.78). Positive change from baseline values indicate improvement.', 'unitOfMeasure': 'T-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with an evaluable SF-36 questionnaire at screening visit and at least one post-screening visit'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Utilized Healthcare Resources During Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Doctor Office Visit', 'categories': [{'measurements': [{'value': '186', 'groupId': 'OG000'}, {'value': '69', 'groupId': 'OG001'}]}]}, {'title': 'Emergency Department Visit', 'categories': [{'measurements': [{'value': '71', 'groupId': 'OG000'}, {'value': '22', 'groupId': 'OG001'}]}]}, {'title': 'Hospital Admission', 'categories': [{'measurements': [{'value': '61', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks)', 'description': 'Number of participants who had any of the following types of Healthcare Resource Utilization (HRU): - a doctor office visit (non-study scheduled) - an emergency department visit - a hospitalization', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Number of Days Spent in Higher Care Hospital Units', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '8.0', 'spread': '23.70', 'groupId': 'OG000'}, {'value': '0.6', 'spread': '0.55', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks)', 'description': "Types of hospitals units considered to be 'higher care' are: - Intensive Care Unit - Coronary Care Unit", 'unitOfMeasure': 'Days', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants who spent time in higher care hospital units'}, {'type': 'SECONDARY', 'title': 'Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '12.1', 'groupId': 'OG000'}, {'value': '1.8', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0015', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '7.6', 'ciLowerLimit': '1.8', 'ciUpperLimit': '32.9', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'groupDescription': 'Odds ratio 95% confidence intervals (CIs), and p-value were estimated from the Cochran Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization.', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, i.e, Days 1 to 56, Days 2 to 57 and so on.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants'}, {'type': 'SECONDARY', 'title': 'Duration of Reduction in Transfusion Burden', 'denoms': [{'units': 'Participants', 'counts': [{'value': '173', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Number of participants analyzed for 33%', 'denoms': [{'units': 'Participants', 'counts': [{'value': '173', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '627.3', 'spread': '390.54', 'groupId': 'OG000'}, {'value': '224.0', 'spread': '155.15', 'groupId': 'OG001'}]}]}, {'title': 'Number of participants analyzed for 50%', 'denoms': [{'units': 'Participants', 'counts': [{'value': '112', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '491.1', 'spread': '386.37', 'groupId': 'OG000'}, {'value': '193.0', 'spread': '142.51', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From first dose to end of study treatment (up to approximately 215 weeks)', 'description': 'Responders were defined as subjects who achieved ≥ 33% reduction or ≥ 50% reduction in Red Blood Cells Transfusion (RBC-T) burden from baseline with a reduction of at least 2 RBC units during any rolling 12-week interval. The duration of reduction is calculated as Last Day of Response - First day of response +1', 'unitOfMeasure': 'Days', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with ≥ 33% reduction or ≥ 50% reduction in RBC-T burden'}, {'type': 'SECONDARY', 'title': 'Longest Duration of Transfusion Independence', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '72.0', 'groupId': 'OG000', 'lowerLimit': '62.0', 'upperLimit': '103.0'}, {'value': '71.5', 'comment': 'Insufficient number of events to determine the upper limit', 'groupId': 'OG001', 'lowerLimit': '62.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, ie, Days 1 to 56, Days 2 to 57 and so on. Longest duration of transfusion independence was estimated based on Kaplan-Meier model.', 'unitOfMeasure': 'Days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants who were transfusion independent for ≥ 8 weeks'}, {'type': 'SECONDARY', 'title': 'Time to Erythroid Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '173', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': '≥ 33% Transfusion Burden Reduction', 'denoms': [{'units': 'Participants', 'counts': [{'value': '173', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '96.3', 'spread': '163.92', 'groupId': 'OG000'}, {'value': '163.5', 'spread': '148.78', 'groupId': 'OG001'}]}]}, {'title': '≥ 50% Transfusion Burden Reduction', 'denoms': [{'units': 'Participants', 'counts': [{'value': '112', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '189.1', 'spread': '257.69', 'groupId': 'OG000'}, {'value': '160.9', 'spread': '160.17', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0195', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-67.27', 'ciLowerLimit': '-123.63', 'ciUpperLimit': '-10.91', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': '≥ 33% Transfusion Burden Reduction', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.7473', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '28.24', 'ciLowerLimit': '-144.87', 'ciUpperLimit': '201.34', 'pValueComment': 'Significance level of 0.050 for 2-sided tests.', 'estimateComment': 'luspatercept - placebo', 'groupDescription': '≥ 50% Transfusion Burden Reduction', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From first dose to 48 weeks following first dose', 'description': 'Time to erythroid response was defined as the time from first dose of the study drug to first erythroid response. This is reported for participants with a ≥ 33% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval., as well as participants with a ≥ 50% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval.', 'unitOfMeasure': 'Days', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants who achieved erythroid response'}, {'type': 'SECONDARY', 'title': 'Post-Baseline Transfusion Event Frequency', 'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Week 1 - 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.1', 'spread': '2.03', 'groupId': 'OG000'}, {'value': '7.9', 'spread': '1.65', 'groupId': 'OG001'}]}]}, {'title': 'Week 25 - 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '201', 'groupId': 'OG000'}, {'value': '96', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.0', 'spread': '2.02', 'groupId': 'OG000'}, {'value': '7.6', 'spread': '1.61', 'groupId': 'OG001'}]}]}, {'title': 'Week 49 - 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '177', 'groupId': 'OG000'}, {'value': '62', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.0', 'spread': '2.04', 'groupId': 'OG000'}, {'value': '7.5', 'spread': '1.28', 'groupId': 'OG001'}]}]}, {'title': 'Week 73 - 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '155', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.0', 'spread': '2.13', 'groupId': 'OG000'}, {'value': '7.0', 'spread': '1.00', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'The number of transfusion events after start of study treatment were evaluated. For the definition of transfusion events, if multiple transfusions happen on the same date, they are counted as one event; if multiple transfusions happen on two consecutive dates, they are counted as one event; if multiple transfusions happen on three consecutive dates, they are counted as two events. Results are presented in 24-week intervals, up to 96 weeks after start of study treatment', 'unitOfMeasure': 'Number of transfusions', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants who received transfusions'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Clearance (CL/F)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '0.437', 'spread': '38.5', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'L/day', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Volume of Distribution of the Central Compartment (V1/F)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '7.08', 'spread': '26.7', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'Liters', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Elimination Half-life (t1/2)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '11.2', 'spread': '25.7', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'days', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Time to Reach Maximum Concentration (Tmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '5.48', 'groupId': 'OG000', 'lowerLimit': '3.35', 'upperLimit': '7.74'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'Days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration for the Starting Dose (Cmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '5.64', 'spread': '25.1', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration at Steady State for the Starting Dose (Cmax,ss)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '8.31', 'spread': '30.1', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Area Under the Concentration-Time Curve at Steady State for the Starting Dose (AUCss)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'categories': [{'measurements': [{'value': '129', 'spread': '36.0', 'groupId': 'OG000'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337', 'unitOfMeasure': 'day*μg/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized participants with available PK measurements for Luspatercept'}, {'type': 'SECONDARY', 'title': 'Participants With Treatment-Emergent Adverse Events (TEAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '223', 'groupId': 'OG000'}, {'value': '109', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': '≥ 1 Treatment-emergent adverse event (TEAE)', 'categories': [{'measurements': [{'value': '219', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}]}, {'title': 'Serious TEAE', 'categories': [{'measurements': [{'value': '53', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}, {'title': 'Grade ≥ 3 TEAE', 'categories': [{'measurements': [{'value': '84', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related TEAE', 'categories': [{'measurements': [{'value': '135', 'groupId': 'OG000'}, {'value': '31', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related Serious TEAE', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-related TEAE ≥ Grade 3', 'categories': [{'measurements': [{'value': '27', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'TEAE leading to death', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Trt-related TEAE leading to death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'TEAE leading to dose reduction', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'TEAE leading to dose delay', 'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}]}, {'title': 'TEAE leading to drug discontinuation', 'categories': [{'measurements': [{'value': '25', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Trt-related TEAE leading to dose reduction', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Trt-related TEAE leading to dose delay', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Trt-related TEAE leading to drug discontinuation', 'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose to 90 days following last dose (up to approximately 52 months)', 'description': 'An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A serious AE is any AE occurring at any dose that - Results in death - Is life-threatening - Requires or prolongs existing inpatient hospitalization - Results in persistent or significant disability/incapacity - Is a congenital anomaly/birth defect - Constitutes an important medical event. The Investigator assessed the relationship of each AE to study drug and graded the severity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 4.03): - Grade 1 = Mild - Grade 2 = Moderate (some limitation in activity; no/minimal medical intervention) - Grade 3 = Severe (limitation in activity; medical intervention required) - Grade 4 = Life-threatening - Grade 5 = Death', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All treated participants'}, {'type': 'SECONDARY', 'title': 'Participants With Pre-Existing and/or Treatment-Emergent Antidrug Antibodies (ADA)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '221', 'groupId': 'OG000'}, {'value': '109', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'OG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'classes': [{'title': 'Pre-existing', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Treatment-emergent', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Timeframe: pre-dose, Day 1, Days 22, 64, 106, 148, 232, 316', 'description': 'Number of participants with positive ADA prior to taking study drug and/or during study. A participant was counted as "treatment-emergent" if there was a positive post-baseline sample while the baseline sample was ADA negative, or there was a positive post-baseline sample with a titer ≥ 4-fold of the baseline titer while the baseline sample was ADA positive. A participant was counted as "preexisting" if the baseline sample was ADA positive and the participant was not qualified for "treatment-emergent."', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All treated participants with available measurements'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'FG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'comment': 'Participants randomized', 'achievements': [{'groupId': 'FG000', 'numSubjects': '224'}, {'groupId': 'FG001', 'numSubjects': '112'}]}, {'type': 'Participants Treated', 'comment': 'All participants who received at least 1 dose of study drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '223'}, {'groupId': 'FG001', 'numSubjects': '109'}]}, {'type': 'Completed 24 Weeks of Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '211'}, {'groupId': 'FG001', 'numSubjects': '102'}]}, {'type': 'Completed 48 Weeks of Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '202'}, {'groupId': 'FG001', 'numSubjects': '96'}]}, {'type': 'Completed 96 Weeks of Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '155'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'Completed 144 Weeks of Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '125'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Completed 192 Weeks of Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'comment': 'Completed study per original protocol', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '218'}, {'groupId': 'FG001', 'numSubjects': '106'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '37'}, {'groupId': 'FG001', 'numSubjects': '16'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Other reasons', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '5'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Transition to Rollover Protocol', 'reasons': [{'groupId': 'FG000', 'numSubjects': '169'}, {'groupId': 'FG001', 'numSubjects': '82'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': '336 participants were randomized, 332 participants were treated.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Luspatercept + BSC', 'description': 'Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care'}, {'id': 'BG001', 'title': 'Placebo + BSC', 'description': 'Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '32.2', 'spread': '10.67', 'groupId': 'BG000'}, {'value': '31.9', 'spread': '9.89', 'groupId': 'BG001'}, {'value': '32.1', 'spread': '10.40', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '132', 'groupId': 'BG000'}, {'value': '63', 'groupId': 'BG001'}, {'value': '195', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '92', 'groupId': 'BG000'}, {'value': '49', 'groupId': 'BG001'}, {'value': '141', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '218', 'groupId': 'BG000'}, {'value': '107', 'groupId': 'BG001'}, {'value': '325', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Asian', 'measurements': [{'value': '81', 'groupId': 'BG000'}, {'value': '36', 'groupId': 'BG001'}, {'value': '117', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '122', 'groupId': 'BG000'}, {'value': '60', 'groupId': 'BG001'}, {'value': '182', 'groupId': 'BG002'}]}, {'title': 'Not collected or reported', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'Other', 'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '26', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All randomized participants'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-12-11', 'size': 6186029, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2019-12-04T12:58', 'hasProtocol': True}, {'date': '2021-03-11', 'size': 812244, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_002.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-12-21T08:35', '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': 336}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-05-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-03', 'dispFirstSubmitDate': '2018-05-23', 'completionDateStruct': {'date': '2021-01-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-03-23', 'studyFirstSubmitDate': '2015-10-21', 'dispFirstSubmitQcDate': '2018-05-23', 'resultsFirstSubmitDate': '2019-12-06', 'studyFirstSubmitQcDate': '2015-11-10', 'dispFirstPostDateStruct': {'date': '2018-05-25', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2023-04-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-01-23', 'studyFirstPostDateStruct': {'date': '2015-11-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-01-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-01-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Erythroid Response was defined as red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Week 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1.'}], 'secondaryOutcomes': [{'measure': 'Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Weeks 37 - 48 compared to the 12-week interval on or prior to Dose 1 Day 1.'}, {'measure': 'Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Weeks 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1.'}, {'measure': 'Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1.'}, {'measure': 'Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24', 'description': 'Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24.'}, {'measure': 'Mean Change From Baseline In Liver Iron Concentration (LIC) At Week 48', 'timeFrame': 'Baseline: Week -12 to Day -1; Treatment: Week 48', 'description': 'Baseline was defined as the last value on or before the first dose of study drug was administered; if multiple values were present for the same date, the average of these values was used. If a participant had 1 postbaseline assessment, it was used as the Week 48 value. If a participant had multiple postbaseline assessments, the last one was used as the Week 48 value. The value of LIC was collected by magnetic resonance imaging. Participants with a LIC value \\> 43 mg/g were not included in the analysis.'}, {'measure': 'Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants.'}, {'measure': 'Mean Change From Baseline In Mean Serum Ferritin At Week 48', 'timeFrame': 'Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48', 'description': 'For each participant, the baseline mean serum ferritin level was calculated during the 12 weeks prior to first study drug administration. The postbaseline mean serum ferritin level was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or last 12 weeks of study treatment, if discontinued early. The change was calculated as the difference of post baseline mean serum ferritin level and baseline mean serum ferritin level.'}, {'measure': 'Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48', 'timeFrame': 'Baseline: Day 1; Treatment: Week 48', 'description': "For BMD, the lumbar spine and total hip were measured at baseline and 48 weeks by dual energy x-ray absorptiometry (DXA). Baseline was defined as the last value on or before the first dose of study drug is administered; if multiple values are present for the same date, the average of these values was used. If during the 48 week double-blinded treatment period, a participant has only one assessment, it is counted as 'Week 48' visit; if a participant has multiple assessments, the last one is used as 'Week 48' visit. The analysis was done on the population that had at least 2 measurements."}, {'measure': 'Mean Change From Baseline In Myocardial Iron At Week 48', 'timeFrame': 'Baseline: Day 1; Treatment: Week 48', 'description': 'Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\\* (Unit: ms). T2\\* values correlates with heart failure (HF) risk (e.g. T2\\*\\<6ms: high HF risk).'}, {'measure': 'Mean Change From Baseline in the Transfusion-dependent Quality of Life (TranQol) Questionnaire At Week 24', 'timeFrame': 'Baseline: 4 weeks prior to Day 1; Treatment: Week 24', 'description': 'The TranQol is a self-administered quality of life tool developed for beta-thalassemia patients. The adult self-report version used in this study, includes 36 questions assessed on a 5-point response, that are grouped into 5 domains (Physical Health, Emotional Health, Sexual Health, Family Functioning, School/Career Functioning). Scores are calculated according to specific scoring algorithms developed by the authors. Both individual domains score and the total score range from 0 (worst) to 100 (best). Total Score and Physical Health domain score are reported. Positive change from baseline values indicate improvement.'}, {'measure': 'Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire At Weeks 24', 'timeFrame': 'Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24', 'description': 'The SF-36 (version 2) is a generic, self-administered instrument consisting of 8 multi-item scales that assess 8 health domains. The raw score for each health domain is transformed into a 0 (worst) to 100 (best) domain score. The 0-100 scale score for each health domain is further converted to normbased scores using a T-score transformation, with a mean of 50 and a standard deviation (SD) of 10. Higher norm-based T-scores indicate better heath/QoL. The domains/summaries reported are: 1. Physical Functioning (Range of possible T-scores is 19.26 - 57.54) 2. General Health (Range of possible T-scores is 18.95 - 66.50) 3. Physical Component summary (PCS) (Range of possible T-scores is 5.02 - 79.78). Positive change from baseline values indicate improvement.'}, {'measure': 'Number of Participants Who Utilized Healthcare Resources During Study', 'timeFrame': 'From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks)', 'description': 'Number of participants who had any of the following types of Healthcare Resource Utilization (HRU): - a doctor office visit (non-study scheduled) - an emergency department visit - a hospitalization'}, {'measure': 'Number of Days Spent in Higher Care Hospital Units', 'timeFrame': 'From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks)', 'description': "Types of hospitals units considered to be 'higher care' are: - Intensive Care Unit - Coronary Care Unit"}, {'measure': 'Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, i.e, Days 1 to 56, Days 2 to 57 and so on.'}, {'measure': 'Duration of Reduction in Transfusion Burden', 'timeFrame': 'From first dose to end of study treatment (up to approximately 215 weeks)', 'description': 'Responders were defined as subjects who achieved ≥ 33% reduction or ≥ 50% reduction in Red Blood Cells Transfusion (RBC-T) burden from baseline with a reduction of at least 2 RBC units during any rolling 12-week interval. The duration of reduction is calculated as Last Day of Response - First day of response +1'}, {'measure': 'Longest Duration of Transfusion Independence', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, ie, Days 1 to 56, Days 2 to 57 and so on. Longest duration of transfusion independence was estimated based on Kaplan-Meier model.'}, {'measure': 'Time to Erythroid Response', 'timeFrame': 'From first dose to 48 weeks following first dose', 'description': 'Time to erythroid response was defined as the time from first dose of the study drug to first erythroid response. This is reported for participants with a ≥ 33% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval., as well as participants with a ≥ 50% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval.'}, {'measure': 'Post-Baseline Transfusion Event Frequency', 'timeFrame': 'From first dose through 3 weeks post last dose (up to approximately 218 weeks)', 'description': 'The number of transfusion events after start of study treatment were evaluated. For the definition of transfusion events, if multiple transfusions happen on the same date, they are counted as one event; if multiple transfusions happen on two consecutive dates, they are counted as one event; if multiple transfusions happen on three consecutive dates, they are counted as two events. Results are presented in 24-week intervals, up to 96 weeks after start of study treatment'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Clearance (CL/F)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Volume of Distribution of the Central Compartment (V1/F)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Elimination Half-life (t1/2)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Time to Reach Maximum Concentration (Tmax)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration for the Starting Dose (Cmax)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration at Steady State for the Starting Dose (Cmax,ss)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Pharmacokinetic (PK) Parameters: Bayesian Estimate of Area Under the Concentration-Time Curve at Steady State for the Starting Dose (AUCss)', 'timeFrame': 'Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337'}, {'measure': 'Participants With Treatment-Emergent Adverse Events (TEAE)', 'timeFrame': 'From first dose to 90 days following last dose (up to approximately 52 months)', 'description': 'An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A serious AE is any AE occurring at any dose that - Results in death - Is life-threatening - Requires or prolongs existing inpatient hospitalization - Results in persistent or significant disability/incapacity - Is a congenital anomaly/birth defect - Constitutes an important medical event. The Investigator assessed the relationship of each AE to study drug and graded the severity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 4.03): - Grade 1 = Mild - Grade 2 = Moderate (some limitation in activity; no/minimal medical intervention) - Grade 3 = Severe (limitation in activity; medical intervention required) - Grade 4 = Life-threatening - Grade 5 = Death'}, {'measure': 'Participants With Pre-Existing and/or Treatment-Emergent Antidrug Antibodies (ADA)', 'timeFrame': 'Timeframe: pre-dose, Day 1, Days 22, 64, 106, 148, 232, 316', 'description': 'Number of participants with positive ADA prior to taking study drug and/or during study. A participant was counted as "treatment-emergent" if there was a positive post-baseline sample while the baseline sample was ADA negative, or there was a positive post-baseline sample with a titer ≥ 4-fold of the baseline titer while the baseline sample was ADA positive. A participant was counted as "preexisting" if the baseline sample was ADA positive and the participant was not qualified for "treatment-emergent."'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['ACE-536', 'Safety', 'Efficacy', 'Placebo', 'Red Blood Cell Transfusions', 'Beta -Thalassemia'], 'conditions': ['Erythrocyte Transfusion', 'Beta-Thalassemia']}, 'referencesModule': {'references': [{'pmid': '30504333', 'type': 'RESULT', 'citation': 'Porter J. Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):361-370. doi: 10.1182/asheducation-2018.1.361.'}, {'pmid': '30617198', 'type': 'RESULT', 'citation': 'Piga A, Perrotta S, Gamberini MR, Voskaridou E, Melpignano A, Filosa A, Caruso V, Pietrangelo A, Longo F, Tartaglione I, Borgna-Pignatti C, Zhang X, Laadem A, Sherman ML, Attie KM. Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia. Blood. 2019 Mar 21;133(12):1279-1289. doi: 10.1182/blood-2018-10-879247. Epub 2019 Jan 7.'}, {'pmid': '39947215', 'type': 'DERIVED', 'citation': 'Cappellini MD, Viprakasit V, Georgiev P, Coates TD, Origa R, Khelif A, Liew HK, Tantiworawit A, Chew LP, Khalil A, Ho PJ, Kuo KHM, Holot N, Perin M, Giuseppi AC, Kuo WL, Lai Y, Medlin LF, Bueno LM, Kattamis A, Taher AT. Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent beta-thalassaemia (BELIEVE): final results from a phase 3 randomised trial. Lancet Haematol. 2025 Mar;12(3):e180-e189. doi: 10.1016/S2352-3026(24)00376-4. Epub 2025 Feb 10.'}, {'pmid': '37782758', 'type': 'DERIVED', 'citation': 'Garbowski MW, Ugidos M, Risueno A, Shetty JK, Schwickart M, Hermine O, Porter JB, Thakurta A, Vodala S. Luspatercept stimulates erythropoiesis, increases iron utilization, and redistributes body iron in transfusion-dependent thalassemia. Am J Hematol. 2024 Feb;99(2):182-192. doi: 10.1002/ajh.27102. Epub 2023 Oct 2.'}, {'pmid': '37704579', 'type': 'DERIVED', 'citation': 'Denton CC, Vodala S, Veluswamy S, Hofstra TC, Coates TD, Wood JC. Splenic iron decreases without change in volume or liver parameters during luspatercept therapy. Blood. 2023 Nov 30;142(22):1932-1934. doi: 10.1182/blood.2023021839.'}, {'pmid': '33570654', 'type': 'DERIVED', 'citation': 'Cappellini MD, Taher AT. The use of luspatercept for thalassemia in adults. Blood Adv. 2021 Jan 12;5(1):326-333. doi: 10.1182/bloodadvances.2020002725.'}, {'pmid': '32212518', 'type': 'DERIVED', 'citation': 'Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Thompson A, Laadem A, Shetty JK, Zou J, Zhang J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A; BELIEVE Investigators. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2020 Mar 26;382(13):1219-1231. doi: 10.1056/NEJMoa1910182.'}], 'seeAlsoLinks': [{'url': 'https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html', 'label': 'BMS Clinical Trial Information'}, {'url': 'https://www.bmsstudyconnect.com/s/US/English/USenHome', 'label': 'BMS Clinical Trial Patient Recruiting'}, {'url': 'https://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm', 'label': 'FDA Safety Alerts and Recalls'}]}, 'descriptionModule': {'briefSummary': "This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia.\n\nThe study is divided into the following periods:\n\n* Historical Period,\n* Screening/Run-in Period,\n* Double-blind Treatment Period (48 weeks),\n* Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks),\n* Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation\n* Post-treatment Follow-up Period"}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSubjects must satisfy the following criteria to be enrolled in the study:\n\n1. Male or female, ≥18 years of age at the time of signing the informed consent document (ICF).\n2. Subject must understand and voluntarily sign an Inform Consent Form prior to any study-related assessments/procedures being conducted.\n3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.\n4. Documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia. (β-thalassemia with mutation and/or multiplication of alpha globin is allowed).\n5. Regularly transfused, defined as: 6-20 Red Blood Cell (RBC) units\\* in the 24 weeks prior to randomization and no transfusion-free period for ≥ 35 days during that period.\n\n \\* Sites who prescribe transfusions and have the transfusion records only in volumes should use for conversion of volume to units the below criteria, in order to obtain number of units within the last 24 weeks to assess the eligibility: 1 unit in this protocol refers to a quantity of packed RBCs approximately 200-350 mL. (i) sites who use transfusion bags within this range, or ≥ 350 mL, the conversion in units should be done by dividing the volume transfused to the patient by 350 mL, (ii) sites who use transfusion bags \\< 200 mL, the conversion in units should be done by dividing the volume transfused to the patient by 200 mL.\n6. Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 or 1.\n7. A female of childbearing potential (FCBP) for this study is defined as a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). FCBP participating in the study must:\n\n 1. Have two negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence \\*\\* from heterosexual contact.\n 2. Either commit to true abstinence\\*\\* from heterosexual contact (which must be reviewed on a monthly basis and source documented) If a FCBP engages in sexual activity that may result in a pregnancy, she must agree to use, and be able to comply with, effective\\*\\*\\* contraception without interruption, 28 days prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks (approximately five times the mean terminal half-life of luspatercept based on multiple-dose Pharmacokinetic PK) data) after discontinuation of study therapy.\n\n * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. \\[Periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.\\] \\*\\*\\* Agreement to use highly effective methods of contraception that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly throughout the course of the study.\n\n Such methods include: Combined (estrogen and progesterone/progestin containing) hormonal contraception: Oral; Intravaginal; Transdermal; Progestogen/progestin only hormonal contraception associated with inhibition of ovulation: Oral; Injectable hormonal contraception; Implantable hormonal contraception; Placement of an intrauterine device (IUD); Placement of an intrauterine hormone-releasing system (IUS); Bilateral tubal occlusion; Vasectomized partner; Sexual Abstinence.\n8. Male subjects must:\n\n * Practice true abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 12 weeks (approximately five times the mean terminal half-life of luspatercept based on multiple-dose PK data) following investigational product discontinuation, even if he has undergone a successful vasectomy.\n\nExclusion Criteria:\n\nThe presence of any of the following will exclude a subject from enrollment:\n\n1. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.\n2. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.\n3. Any condition that confounds the ability to interpret data from the study.\n4. A diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H);\n5. Evidence of active hepatitis C (HCV) infection as demonstrated by a positive HCV-RNA test of sufficient sensitivity, or active infectious hepatitis B as demonstrated by the presence of HBsAg and/or HBVDNA-positive,, or known positive human immunodeficiency virus (HIV).\n\n Note: Subjects receiving antiviral therapies should have 2 negative HCVRNA tests 3 months apart.(ie, one test at the end of the antiviral therapy and a second test 3 months following the first test).\n6. Deep Vein Thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to randomization.\n7. Use of chronic anticoagulant therapy is excluded, unless the treatment stopped at least 28 days prior to randomization. Anticoagulant therapies used for prophylaxis for surgery or high risk procedures as well as low Molecular Weight (LMW) heparin for superficial venous thrombosis and chronic aspirin are allowed.\n8. Platelet count \\> 1000 x 109/L\n9. Poorly controlled diabetes mellitus within 24 weeks prior to randomization as defined by short term (eg, hyperosmolar or ketoacidotic crisis) and/or history of diabetic cardiovascular complications (eg, stroke or myocardial infarction).\n10. Treatment with another investigational drug or device ≤ 28 days prior to randomization.\n11. Prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).\n12. Use of an erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to randomization.\n13. Iron chelation therapy, if initiated ≤ 24 weeks prior to randomization (allowed if initiated \\> 24 weeks before or during treatment).\n14. Hydroxyurea treatment ≤ 24 weeks prior to randomization.\n15. Pregnant or lactating females.\n16. Uncontrolled hypertension. Controlled hypertension for this protocol is considered ≤ Grade 1 according to NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (current active minor version).\n17. Major organ damage, including:\n\n 1. Liver disease with alanine aminotransferase (ALT) \\> 3 x the upper limit of normal (ULN) or history of evidence of cirrhosis;\n 2. Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.\n 3. Lung disease, including pulmonary fibrosis or pulmonary hypertension which are clinically significant ie, ≥ Grade 3 NCI CTCAE version 4.0 (current active minor version).\n 4. Creatinine clearance \\< 60 mL/min (per Cockroft-Gault formula).\n18. Proteinuria ≥ Grade 3 according to NCI CTCAE version 4.0 (current active minor version).\n19. Chronic systemic glucocorticoids ≤ 12 weeks prior to randomization (physiologic replacement therapy for adrenal insufficiency is allowed). Single day glucocorticoid treatment (eg, for prevention or treatment of transfusion reactions, is allowed).\n20. Major surgery ≤ 12 weeks prior to randomization (subjects must have completely recovered from any previous surgery prior to randomization).\n21. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product (see Investigator Brochure).\n22. Cytotoxic agents, immunosuppressants ≤ 28 days prior to randomization (ie, antithymocite globulin (ATG) or cyclosporine)\n23. History of malignancy with the exception of:\n\n 1. Curatively resected nonmelanoma skin cancer.\n 2. Curatively treated cervical carcinoma in situ.\n 3. Other solid tumor with no known active disease in the opinion of the investigator.'}, 'identificationModule': {'nctId': 'NCT02604433', 'acronym': 'BELIEVE', 'briefTitle': 'An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'Celgene'}, 'officialTitle': 'A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia', 'orgStudyIdInfo': {'id': 'ACE-536-B-THAL-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Luspatercept (ACE-536) plus Best Supportive Care (BSC)', 'description': 'Luspatercept, subcutaneous(ly) (SC) once every 21 days', 'interventionNames': ['Drug: Luspatercept']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo plus Best Supportive Care (BSC)', 'description': 'normal saline solution subcutaneous(ly) (SC) once every 21 days', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'Luspatercept', 'type': 'DRUG', 'otherNames': ['ACE-536'], 'description': 'Subjects will start with luspatercept at 1 mg/kg dose level.', 'armGroupLabels': ['Luspatercept (ACE-536) plus Best Supportive Care (BSC)']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Placebo, Subcutaneous, every 21 days.', 'armGroupLabels': ['Placebo plus Best Supportive Care (BSC)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital and Research Center at Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Ann and Robert H Lurie Childrens Hospital of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10065', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Weill Cornell Medical College', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Hospital of the University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '2031', 'city': 'Randwick', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Prince of Wales Hospital', 'geoPoint': {'lat': -33.91439, 'lon': 151.24895}}, {'zip': '4101', 'city': 'South Brisbane', 'state': 'Queensland', 'country': 'Australia', 'facility': 'Mater Hospital Brisbane', 'geoPoint': {'lat': -27.48034, 'lon': 153.02049}}, {'zip': '5000', 'city': 'Adelaide', 'state': 'South Australia', 'country': 'Australia', 'facility': 'Royal Adelaide Hospital Institute of Medical and Veterinary Science', 'geoPoint': {'lat': -34.92866, 'lon': 138.59863}}, {'zip': '3168', 'city': 'Clayton', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Monash Medical Centre', 'geoPoint': {'lat': -37.91667, 'lon': 145.11667}}, {'zip': '6009', 'city': 'Nedlands', 'state': 'Western Australia', 'country': 'Australia', 'facility': 'Sir Charles Gairdner Hospital', 'geoPoint': {'lat': -31.98184, 'lon': 115.8073}}, {'zip': '2050', 'city': 'Camperdown', 'country': 'Australia', 'facility': 'Royal Prince Alfred Hospital', 'geoPoint': {'lat': -33.88965, 'lon': 151.17642}}, {'zip': '4002', 'city': 'Plovdiv', 'country': 'Bulgaria', 'facility': 'University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'zip': '1756', 'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Specialized Hospital for Active Treatment of Haematological Diseases - 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