Ignite Creation Date:
2026-03-26 @ 3:21 PM
Ignite Modification Date:
2026-04-11 @ 1:19 AM
Study NCT ID:
NCT01254227
Status:
COMPLETED
Last Update Posted:
2021-07-26
First Post:
2010-09-21
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077588', 'term': 'Deferasirox'}, {'id': 'D003676', 'term': 'Deferoxamine'}], 'ancestors': [{'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D014230', 'term': 'Triazoles'}, {'id': 'D001393', 'term': 'Azoles'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D006877', 'term': 'Hydroxamic Acids'}, {'id': 'D006898', 'term': 'Hydroxylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D006880', 'term': 'Hydroxy Acids'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Novartis.email@novartis.com', 'phone': '862-778-8300', 'title': 'Study Director', 'organization': 'Novartis Pharmaceuticals'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit, up to 24 months with 28 days of Follow up', 'description': 'The analysis was performed on safety set population defined as all participants who received study drug and had at least one post-baseline safety assessment.', 'eventGroups': [{'id': 'EG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.', 'otherNumAtRisk': 60, 'deathsNumAtRisk': 60, 'otherNumAffected': 52, 'seriousNumAtRisk': 60, 'deathsNumAffected': 1, 'seriousNumAffected': 17}], 'otherEvents': [{'term': 'Thrombocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Atrioventricular block first degree', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Sinus tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Conductive deafness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Deafness bilateral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Deafness neurosensory', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Eustachian tube dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Enteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 14}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Acute tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Tooth abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 10}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Allergic transfusion reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Traumatic arthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Blood creatinine increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cardiac murmur', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Electrocardiogram QT prolonged', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Electrocardiogram T wave amplitude decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Electrocardiogram T wave inversion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Urine protein/creatinine ratio increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 6}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Type 1 diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 8}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 12}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Bone pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 9}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 6}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dysphonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dermoid cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Irritable bowel syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pancreatitis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Reflux gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Biliary colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abscess neck', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Febrile infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Chest injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Spinal column injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Calcium deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hypophosphataemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Type 1 diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Costochondritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Ovarian germ cell teratoma benign', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Altered state of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Ischaemic stroke', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'VIIth nerve paralysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Fallopian tube cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Ovarian cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Drug reaction with eosinophilia and systemic symptoms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 60, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Cardiac Iron Content From Baseline to Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.09', 'groupId': 'OG000', 'lowerLimit': '1.04', 'upperLimit': '1.15'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'From Baseline to Month 12', 'description': 'Cardiac T2\\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\\* value of \\<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\\* or do not have any post-baseline T2\\* are excluded from the analysis.', 'unitOfMeasure': 'ratio', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '44', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.50', 'groupId': 'OG000', 'lowerLimit': '5.86', 'upperLimit': '24.70'}]}]}, {'title': 'Month12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19.23', 'groupId': 'OG000', 'lowerLimit': '10.80', 'upperLimit': '31.90'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '33.33', 'groupId': 'OG000', 'lowerLimit': '19.75', 'upperLimit': '50.39'}]}]}, {'title': 'Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '47.22', 'groupId': 'OG000', 'lowerLimit': '31.99', 'upperLimit': '62.99'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. Here, "Number of participants analyzed" included all participants who were evaluable for the specified timepoints.'}, {'type': 'SECONDARY', 'title': 'Change in Cardiac Iron Content From Baseline to Month 6,18 and 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.02', 'groupId': 'OG000', 'lowerLimit': '0.98', 'upperLimit': '1.07'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.17', 'groupId': 'OG000', 'lowerLimit': '1.08', 'upperLimit': '1.28'}]}]}, {'title': 'Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.30', 'groupId': 'OG000', 'lowerLimit': '1.17', 'upperLimit': '1.44'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'From Baseline to Months 6, 18 and 24', 'description': 'The change in cardiac iron content was calculated as ratio of Cardiac T2\\* at different time points; the efficacy endpoint analyses were performed on the Full Analysis Set (FAS).', 'unitOfMeasure': 'ratio', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint. Here, "Number of participants analyzed" included all participants who were evaluable for the specified timepoints.'}, {'type': 'SECONDARY', 'title': 'Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '66.5', 'spread': '5.32', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1', 'spread': '4.62', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.2', 'spread': '4.84', 'groupId': 'OG000'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '7.04', 'groupId': 'OG000'}]}]}, {'title': 'Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.9', 'spread': '5.98', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'Magnetic resonance imaging (MRI)-measured cardiac T2\\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.', 'unitOfMeasure': 'Percent Ejection Fraction', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint.'}, {'type': 'SECONDARY', 'title': 'Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '67.1', 'spread': '5.55', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '47', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.2', 'spread': '5.35', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.6', 'spread': '4.40', 'groupId': 'OG000'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.1', 'spread': '6.10', 'groupId': 'OG000'}]}]}, {'title': 'Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.4', 'spread': '4.25', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'Magnetic resonance imaging (MRI)-measured cardiac T2\\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.', 'unitOfMeasure': 'Percent Ejection Fraction', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint.'}, {'type': 'SECONDARY', 'title': 'Time to Achieve From Baseline (FAS) of at Least 10% at Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'categories': [{'measurements': [{'value': '722.0', 'comment': 'NA: It was not estimable.', 'groupId': 'OG000', 'lowerLimit': '520.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'At 24 months', 'description': 'Time from date of start of study treatment to date when first achieving T2\\* ≥ 10 ms (but at least 10% relative increase from baseline) was summarized using the reverse Kaplan-Meier estimates (1 - Kaplan-Meier estimates) for the FAS.', 'unitOfMeasure': 'milliseconds/ms', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint.'}, {'type': 'SECONDARY', 'title': 'Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.18', 'spread': '1.045', 'groupId': 'OG000'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '48', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.31', 'spread': '1.442', 'groupId': 'OG000'}]}]}, {'title': 'Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '46', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.93', 'spread': '1.429', 'groupId': 'OG000'}]}]}, {'title': 'Month 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.51', 'spread': '1.348', 'groupId': 'OG000'}]}]}, {'title': 'Month 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.14', 'spread': '1.381', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From the Baseline, Month 6, 12, 18 and Month 24', 'description': 'Cardiac iron concentration (mg Fe/g dw) was quantified using the formula (cardiac iron concentration (mg Fe/g dw) = 45 \\* T2\\* (ms) \\^ (-1.22) and analyzed over time.', 'unitOfMeasure': 'mg Fe/g dw', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set includes all participants to whom study treatment had assigned. Participants were considered evaluable for the efficacy endpoint if they had received at least one dose of study treatment and had baseline and a post baseline assessment prior to or on the time of the assessment of the corresponding efficacy endpoint.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 Milligrams per Kilogram per day (mg/kg/day) every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '60'}]}, {'type': 'Completion of 12 Months', 'achievements': [{'groupId': 'FG000', 'numSubjects': '39'}]}, {'type': 'Completion of 24 Months', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '26'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Abnormal test procedure result', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Administrative problems', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Protocol deviation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study was conducted at 18 centers in 8 countries.', 'preAssignmentDetails': 'A total 60 participants were enrolled in the study of which 34 completed the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants', 'description': 'Participants received an initial combined dose of Deferasirox (DFX) 20 mg/kg/day every day and Deferoxamine (DFO) 40 mg/kg/day (DFO: 5 days/week for at least 8 hours/day). Deferasirox was increased to 30 mg/kg/day every day at Month 1 Further dose increases upto 40 mg/kg/day every day at Month 6 visit. Participants could be switched from the Deferasirox - Deferoxamine (DFX - DFO) combination to Deferasirox monotherapy at the highest tolerable dose (30-40 mg/kg/day) at any time point after 6 months.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '22.8', 'spread': '7.33', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '32', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '28', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 60}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-07', 'completionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-07-23', 'studyFirstSubmitDate': '2010-09-21', 'resultsFirstSubmitDate': '2021-04-29', 'studyFirstSubmitQcDate': '2010-12-03', 'lastUpdatePostDateStruct': {'date': '2021-07-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-06-01', 'studyFirstPostDateStruct': {'date': '2010-12-06', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-06-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Cardiac Iron Content From Baseline to Month 12', 'timeFrame': 'From Baseline to Month 12', 'description': 'Cardiac T2\\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\\* value of \\<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\\* or do not have any post-baseline T2\\* are excluded from the analysis.'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit.'}, {'measure': 'Change in Cardiac Iron Content From Baseline to Month 6,18 and 24', 'timeFrame': 'From Baseline to Months 6, 18 and 24', 'description': 'The change in cardiac iron content was calculated as ratio of Cardiac T2\\* at different time points; the efficacy endpoint analyses were performed on the Full Analysis Set (FAS).'}, {'measure': 'Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'Magnetic resonance imaging (MRI)-measured cardiac T2\\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.'}, {'measure': 'Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24', 'timeFrame': 'From the Months 6, 12, 18 and 24', 'description': 'Magnetic resonance imaging (MRI)-measured cardiac T2\\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.'}, {'measure': 'Time to Achieve From Baseline (FAS) of at Least 10% at Month 24', 'timeFrame': 'At 24 months', 'description': 'Time from date of start of study treatment to date when first achieving T2\\* ≥ 10 ms (but at least 10% relative increase from baseline) was summarized using the reverse Kaplan-Meier estimates (1 - Kaplan-Meier estimates) for the FAS.'}, {'measure': 'Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24', 'timeFrame': 'From the Baseline, Month 6, 12, 18 and Month 24', 'description': 'Cardiac iron concentration (mg Fe/g dw) was quantified using the formula (cardiac iron concentration (mg Fe/g dw) = 45 \\* T2\\* (ms) \\^ (-1.22) and analyzed over time.'}]}, 'conditionsModule': {'keywords': ['Cardiac iron overload,', 'deferasirox,', 'deferoxamine'], 'conditions': ['Cardiac Iron Overload']}, 'referencesModule': {'references': [{'pmid': '25934475', 'type': 'DERIVED', 'citation': 'Aydinok Y, Kattamis A, Cappellini MD, El-Beshlawy A, Origa R, Elalfy M, Kilinc Y, Perrotta S, Karakas Z, Viprakasit V, Habr D, Constantinovici N, Shen J, Porter JB; HYPERION Investigators. Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload. Blood. 2015 Jun 18;125(25):3868-77. doi: 10.1182/blood-2014-07-586677. Epub 2015 May 1.'}], 'seeAlsoLinks': [{'url': 'https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=12963', 'label': 'link to the results'}]}, 'descriptionModule': {'briefSummary': 'This study will evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed be deferasirox monotherapy in patients with severe iron overload due to chronic blood transfusions.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '10 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy\n* Myocardial T2\\* value that is ≥ 5 and \\< 10 ms\n* Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (MRI)\n* Liver Iron Concentration (LIC) ≥ 7 mg Fe /g dw as determined by R2 MRI.\n* Lifetime history of at least 50 units of red blood cell transfusions, and must be receiving at least ≥ 8 units/yr of red blood cell transfusions\n* Serum ferritin ≥ 1000 ng/mL\n\nExclusion Criteria:\n\n* Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)\n* Patients unable to undergo study assessments including MRI\n* Patients with serum creatinine greater than Upper limit of normal ULN)range or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥1.0 mg/mg in a non-first void urine sample at baseline.\n\nOther protocol-defined inclusion/exclusion criteria may apply'}, 'identificationModule': {'nctId': 'NCT01254227', 'acronym': 'HYPERION', 'briefTitle': 'Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion (HYPERION)', 'orgStudyIdInfo': {'id': 'CICL670A2214'}, 'secondaryIdInfos': [{'id': '2010-021062-29', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Deferasirox', 'description': 'Deferoxamine combination followed by Deferasirox monotherapy', 'interventionNames': ['Drug: Deferasirox and Deferoxamine']}], 'interventions': [{'name': 'Deferasirox and Deferoxamine', 'type': 'DRUG', 'armGroupLabels': ['Deferasirox']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'M5G 2C4', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Cairo', 'country': 'Egypt', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 30.06263, 'lon': 31.24967}}, {'zip': 'GR-115 27', 'city': 'Athens', 'state': 'GR', 'country': 'Greece', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 37.98376, 'lon': 23.72784}}, {'zip': '265 04', 'city': 'Patra - RIO', 'state': 'GR', 'country': 'Greece', 'facility': 'Novartis Investigative Site'}, {'zip': '09121', 'city': 'Cagliari', 'state': 'CA', 'country': 'Italy', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 39.23054, 'lon': 9.11917}}, {'zip': '16128', 'city': 'Genova', 'state': 'GE', 'country': 'Italy', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 45.21604, 'lon': 11.87211}}, {'zip': '80138', 'city': 'Naples', 'country': 'Italy', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}, {'zip': '10002', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': '10330', 'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}, {'zip': '10700', 'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}, {'zip': '01330', 'city': 'Adana', 'country': 'Turkey (Türkiye)', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 36.98615, 'lon': 35.32531}}, {'zip': '07070', 'city': 'Antalya', 'country': 'Turkey (Türkiye)', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 36.90812, 'lon': 30.69556}}, {'zip': '34093', 'city': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}, {'zip': '35040', 'city': 'Izmir', 'country': 'Turkey (Türkiye)', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 38.41273, 'lon': 27.13838}}, {'zip': 'NW1 2BU', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'url': 'https://www.clinicalstudydatarequest.com', 'ipdSharing': 'YES', 'description': 'Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data is currently available according to the process described on www.clinicalstudydatarequest.com.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}