Ignite Creation Date:
2025-12-26 @ 11:12 PM
Ignite Modification Date:
2026-03-02 @ 5:54 PM
Study NCT ID:
NCT01825512
Status:
COMPLETED
Last Update Posted:
2021-05-04
First Post:
2013-04-03
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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': 'D000077543', 'term': 'Deferiprone'}, {'id': 'D007531', 'term': 'Isoflurophate'}, {'id': 'D000077588', 'term': 'Deferasirox'}], 'ancestors': [{'id': 'D011728', 'term': 'Pyridones'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D063066', 'term': 'Organofluorophosphonates'}, {'id': 'D063065', 'term': 'Organophosphonates'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ceo@cvbf.net', 'phone': '+393936698076', 'title': 'Donato Bonifazi', 'organization': 'Consorzio per Valutazioni Biologiche e Farmacologiche'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'through study completion, an average of 1 year for subject', 'eventGroups': [{'id': 'EG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution', 'otherNumAtRisk': 193, 'deathsNumAtRisk': 193, 'otherNumAffected': 152, 'seriousNumAtRisk': 193, 'deathsNumAffected': 0, 'seriousNumAffected': 13}, {'id': 'EG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg', 'otherNumAtRisk': 197, 'deathsNumAtRisk': 197, 'otherNumAffected': 89, 'seriousNumAtRisk': 197, 'deathsNumAffected': 0, 'seriousNumAffected': 14}], 'otherEvents': [{'term': 'cough', 'notes': 'cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 21, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 25, 'numAffected': 16}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'cough'}, {'term': 'neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 26, 'numAffected': 18}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 15, 'numAffected': 11}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 40, 'numAffected': 28}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 56, 'numAffected': 32}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 24, 'numAffected': 20}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 42, 'numAffected': 33}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 28, 'numAffected': 23}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 18, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 11, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'testicular injury', 'notes': 'testicular injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'testicular injury'}, {'term': 'splenectomy', 'notes': 'splenectomy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'splenectomy'}, {'term': 'agranulocytosis', 'notes': 'agranulocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'agranulocytosis'}, {'term': 'neutropenia', 'notes': 'neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'neutropenia'}, {'term': 'sickle cell anaemia with crisis subjects affected/exposed', 'notes': 'sickle cell anaemia with crisis subjects affected/exposed', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'sickle cell anaemia'}, {'term': 'seizure', 'notes': 'seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'seizure'}, {'term': 'pyrexia', 'notes': 'pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'pyrexia'}, {'term': 'chest pain', 'notes': 'chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'chest pain'}, {'term': 'gait disturbance', 'notes': 'gait disturbance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'gait disturbance'}, {'term': 'diarrhoea', 'notes': 'diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'diarrhoea'}, {'term': 'hypertransaminasaemia', 'notes': 'hypertransaminasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'hypertransaminasaemi'}, {'term': 'acute kidney injury', 'notes': 'acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'acute kidney injury'}, {'term': 'pain in extremity', 'notes': 'pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'pain in extremity'}, {'term': 'gastroenteritis', 'notes': 'gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'gastroenteritis'}, {'term': 'meningitis meningococcal', 'notes': 'meningitis meningococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'meningitis meningoco'}, {'term': 'herpangina', 'notes': 'herpangina', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'herpangina'}, {'term': 'pneumonia', 'notes': 'pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'pneumonia'}, {'term': 'subcutaneous abscess', 'notes': 'subcutaneous abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'subcutaneous abscess'}, {'term': 'urinary tract infection', 'notes': 'urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'urinary tract infect'}, {'term': 'herpes virus infection', 'notes': 'herpes virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'herpes virus infecti'}, {'term': 'impetigo', 'notes': 'impetigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'impetigo'}, {'term': 'upper respiratory tract infection', 'notes': 'upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 193, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 197, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'upper respiratory tr'}], 'frequencyThreshold': '4'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Successfully Chelated Patients', 'denoms': [{'units': 'Participants', 'counts': [{'value': '125', 'groupId': 'OG000'}, {'value': '146', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'OG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}], 'classes': [{'categories': [{'measurements': [{'value': '69', 'groupId': 'OG000'}, {'value': '80', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Treatment success rate', 'ciNumSides': 'ONE_SIDED', 'ciPctValue': '95', 'paramValue': '-12.5', 'ciLowerLimit': '-12.5', 'nonInferiorityType': 'NON_INFERIORITY', 'nonInferiorityComment': 'Deferiprone was declared non inferior to Deferasirox if the lower limit of the 95% confidence interval for the difference in the proportion of successful chelation in the two groups is above -12.5%.'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'at baseline and after 12 months', 'description': 'Percentage of successfully chelated patients is assessed by serum ferritin levels (in all patients) and cardiac MRI T2\\* (in patients above 10 years of age able to perform an MRI scan without sedation)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Per-protocol population 1 (PP1): number of patients for whom the primary composite efficacy endpoint data were available at baseline and after 1 year of treatment (271 subjects)'}, {'type': 'SECONDARY', 'title': 'Liver MRI', 'denoms': [{'units': 'Participants', 'counts': [{'value': '46', 'groupId': 'OG000'}, {'value': '60', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'OG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.848', 'spread': '0.887', 'groupId': 'OG000'}, {'value': '-2.975', 'spread': '0.776', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.074', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.128', 'ciLowerLimit': '-0.213', 'ciUpperLimit': '4.468', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.18', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'GLM model'}], 'paramType': 'MEAN', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in liver iron concentration (measured using liver MRI), assessed as difference between value at 12 months minus value at baseline.', 'unitOfMeasure': 'mg/g', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Per-protocol population 3 (PP3): number of patients for whom liver iron concentration were available at baseline and after 1 year of treatment (106 subjects)'}, {'type': 'SECONDARY', 'title': 'Cardiac MRI T2*', 'denoms': [{'units': 'Participants', 'counts': [{'value': '49', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'OG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}], 'classes': [{'categories': [{'measurements': [{'value': '0.488', 'spread': '1.284', 'groupId': 'OG000'}, {'value': '1.121', 'spread': '1.169', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.717', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.633', 'ciLowerLimit': '-4.085', 'ciUpperLimit': '2.819', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.741', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'GLM Analysis'}], 'paramType': 'MEAN', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in cardiac iron concentration (measured using cardiac MRI T2\\*), assessed as difference between value at 12 months minus value at baseline. MRI T2\\* is a non-invasive method based on gradient echo (GRE) sequences, where T2\\* represents the spin-spin relaxation times, measured in milliseconds. The faster the curve decreases (ie, the smaller T2\\*), the greater amount of iron is in the tissue. Treatment success was assessed as follows: if baseline cardiac T2\\* was less than 20 ms, an increase of 10% or more after 1 year of treatment was defined as treatment success; if baseline cardiac T2\\* was more than 20 ms, any increase or a decrease of less than 10% after 1 year of treatment was defined as treatment success.', 'unitOfMeasure': 'milliseconds (ms)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Per-protocol population 3 (PP3): number of patients for whom cardiac T2\\* concentration were available at baseline and after 1 year of treatment (108 subjects)'}, {'type': 'SECONDARY', 'title': 'Ferritin Level', 'denoms': [{'units': 'Participants', 'counts': [{'value': '137', 'groupId': 'OG000'}, {'value': '166', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'OG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}], 'classes': [{'categories': [{'measurements': [{'value': '-397.583', 'spread': '121.794', 'groupId': 'OG000'}, {'value': '-398.184', 'spread': '110.619', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.997', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.601', 'ciLowerLimit': '-323.580', 'ciUpperLimit': '324.781', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '164.734', 'statisticalMethod': 'GLM', 'nonInferiorityType': 'NON_INFERIORITY', 'nonInferiorityComment': 'Non inferiority of Deferiprone to Deferasirox is tested considering a non-inferiority margin of 400 ng/mL.'}], 'paramType': 'MEAN', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in serum ferritin level, assessed as difference between value at 12 months minus value at baseline.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Per-protocol population 2 (PP2): number of patients for whom the per-protocol centralised serum ferritin concentration data were available at baseline and after 1 year of treatment (303 subjects, this population was larger than PP1 because PP2 included patients who did not have cardiac T2\\* data)'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'FG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}], 'periods': [{'title': 'Baseline', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '194'}, {'groupId': 'FG001', 'numSubjects': '199'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '193'}, {'groupId': 'FG001', 'numSubjects': '197'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '2'}]}]}, {'title': 'Treatment', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '193'}, {'groupId': 'FG001', 'numSubjects': '197'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '140'}, {'groupId': 'FG001', 'numSubjects': '170'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '53'}, {'groupId': 'FG001', 'numSubjects': '27'}]}]}], 'preAssignmentDetails': 'Informed consent was collected for 435 patients that were enrolled in the study, however 42 of them were excluded from the study for the following reasons: 17 did not meet inclusion criteria, 5 withdrew the consent and 20 were lost to follow-up.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week\n\nDeferiprone: Deferiprone 80 mg/mL oral solution'}, {'id': 'BG001', 'title': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week\n\nDeferasirox: Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': '<6 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '59', 'groupId': 'BG000'}, {'value': '58', 'groupId': 'BG001'}, {'value': '117', 'groupId': 'BG002'}]}]}, {'title': '> or equal to 6 years and <10 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '47', 'groupId': 'BG000'}, {'value': '47', 'groupId': 'BG001'}, {'value': '94', 'groupId': 'BG002'}]}]}, {'title': '> or equal to 10 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '87', 'groupId': 'BG000'}, {'value': '92', 'groupId': 'BG001'}, {'value': '179', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '80', 'groupId': 'BG000'}, {'value': '93', 'groupId': 'BG001'}, {'value': '173', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '113', 'groupId': 'BG000'}, {'value': '104', 'groupId': 'BG001'}, {'value': '217', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Europe', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '56', 'groupId': 'BG000'}, {'value': '56', 'groupId': 'BG001'}, {'value': '112', 'groupId': 'BG002'}]}]}, {'title': 'North Africa', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '121', 'groupId': 'BG000'}, {'value': '122', 'groupId': 'BG001'}, {'value': '243', 'groupId': 'BG002'}]}]}, {'title': 'Rest of Africa', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'Asia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'North America', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Latin America', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'Missing', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '30', 'groupId': 'BG000'}, {'value': '28', 'groupId': 'BG001'}, {'value': '58', 'groupId': 'BG002'}]}]}, {'title': 'Tunisia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '28', 'groupId': 'BG000'}, {'value': '28', 'groupId': 'BG001'}, {'value': '56', 'groupId': 'BG002'}]}]}, {'title': 'Egypt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '96', 'groupId': 'BG000'}, {'value': '99', 'groupId': 'BG001'}, {'value': '197', 'groupId': 'BG002'}]}]}, {'title': 'Albania', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '39', 'groupId': 'BG002'}]}]}, {'title': 'Greece', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'Cyprus', 'denoms': [{'units': 'Participants', 'counts': [{'value': '193', 'groupId': 'BG000'}, {'value': '197', 'groupId': 'BG001'}, {'value': '390', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Ferritin level', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '163', 'groupId': 'BG000'}, {'value': '173', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '2756', 'spread': '2175', 'groupId': 'BG000'}, {'value': '2989', 'spread': '2409', 'groupId': 'BG001'}, {'value': '2876', 'spread': '2298', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'ng/ml', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'The difference is due to missing data at baseline'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-12-10', 'size': 1597605, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-01-29T08:48', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 435}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-03-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-10', 'completionDateStruct': {'date': '2017-09-21', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-08', 'studyFirstSubmitDate': '2013-04-03', 'resultsFirstSubmitDate': '2021-01-27', 'studyFirstSubmitQcDate': '2013-04-03', 'lastUpdatePostDateStruct': {'date': '2021-05-04', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-03-15', 'studyFirstPostDateStruct': {'date': '2013-04-05', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-04-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-09-21', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Successfully Chelated Patients', 'timeFrame': 'at baseline and after 12 months', 'description': 'Percentage of successfully chelated patients is assessed by serum ferritin levels (in all patients) and cardiac MRI T2\\* (in patients above 10 years of age able to perform an MRI scan without sedation)'}], 'secondaryOutcomes': [{'measure': 'Liver MRI', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in liver iron concentration (measured using liver MRI), assessed as difference between value at 12 months minus value at baseline.'}, {'measure': 'Cardiac MRI T2*', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in cardiac iron concentration (measured using cardiac MRI T2\\*), assessed as difference between value at 12 months minus value at baseline. MRI T2\\* is a non-invasive method based on gradient echo (GRE) sequences, where T2\\* represents the spin-spin relaxation times, measured in milliseconds. The faster the curve decreases (ie, the smaller T2\\*), the greater amount of iron is in the tissue. Treatment success was assessed as follows: if baseline cardiac T2\\* was less than 20 ms, an increase of 10% or more after 1 year of treatment was defined as treatment success; if baseline cardiac T2\\* was more than 20 ms, any increase or a decrease of less than 10% after 1 year of treatment was defined as treatment success.'}, {'measure': 'Ferritin Level', 'timeFrame': 'at baseline and after 12 months', 'description': 'Change in serum ferritin level, assessed as difference between value at 12 months minus value at baseline.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['chronic iron overload', 'hereditary haemoglobinopathy', 'beta thalassaemia major', 'chelating agents', 'deferiprone', 'deferasirox', 'children', 'paediatrics'], 'conditions': ['Chronic Iron Overload']}, 'referencesModule': {'references': [{'pmid': '33926431', 'type': 'DERIVED', 'citation': 'Giannuzzi V, Felisi M, Bonifazi D, Devlieger H, Papanikolaou G, Ragab L, Fattoum S, Tempesta B, Reggiardo G, Ceci A. Ethical and procedural issues for applying researcher-driven multi-national paediatric clinical trials in and outside the European Union: the challenging experience of the DEEP project. BMC Med Ethics. 2021 Apr 29;22(1):49. doi: 10.1186/s12910-021-00618-2.'}, {'pmid': '32470438', 'type': 'DERIVED', 'citation': 'Maggio A, Kattamis A, Felisi M, Reggiardo G, El-Beshlawy A, Bejaoui M, Sherief L, Christou S, Cosmi C, Della Pasqua O, Del Vecchio GC, Filosa A, Cuccia L, Hassab H, Kreka M, Origa R, Putti MC, Spino M, Telfer P, Tempesta B, Vitrano A, Tsang YC, Zaka A, Tricta F, Bonifazi D, Ceci A. Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial. Lancet Haematol. 2020 Jun;7(6):e469-e478. doi: 10.1016/S2352-3026(20)30100-9.'}]}, 'descriptionModule': {'briefSummary': 'Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40 mg/kg/day in paediatric patients (1 month \\< 18 years old) affected by hereditary haemoglobinopathies and requiring frequent transfusions and chelation.', 'detailedDescription': 'Haemoglobinopathies are a group of inherited disorders characterized by structural variations of the haemoglobin molecule. Most of the patients affected require for survival chronic red blood cells transfusions to overcome ineffective erythropoiesis. Unfortunately, all chronically transfused patients become clinically iron overloaded as there is no physiological mechanism for the removal of iron from the body. The pathologic changes and clinical manifestations associated to chronic iron overload are common among all transfusional iron-overload patients, albeit best documented in patients with beta-thalassemia major. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove the harmful iron accumulation in the body.\n\nCurrently, in the clinical practice particularly in children and adolescents, the criteria leading to the choice of the chelating agent include also the adherence to therapy, thus favouring the use of oral chelators (Ceci A et al., 2011) DFP (Deferiprone) was the first oral chelator authorised in Europe in 1999 as second line treatment for the treatment of iron overload in patients with thalassaemia major when DFO (Deferoxamine) is contraindicated or inadequate. However, despite a wide experience of DFP with iron overloaded (specifically thalassaemic )patients, limited data are available for younger children. For this reason the need for additional data in younger children is expressively included in the 2009 PDCO (Paediatric Committee) Priority List.\n\nThe purpose of this study is to assess the non-inferiority of DFP compared to DFX (deferasirox)in paediatric patients affected by hereditary haemoglobinopathies requiring chronic transfusions and chelation. Non inferiority will be established in terms of percentage of patients successfully chelated, as assessed by serum ferritin levels (in all patients) and cardiac MRI T2\\* (in patients above 10 years of age able to have an MRI scan without sedation).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '1 Month', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment\n* Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease\n* Patients on current treatment with deferoxamine (DFO) or DFX or DFP in a chronic transfusion program receiving at least 150 mL/kg/year of packed red blood cells (corresponding approximately to 12 transfusions);\n* For patients naïve to chelation treatment: patients that have received at least 150 mL/kg of packed red blood cells (corresponding to approximately 12 transfusions) in a chronic transfusion program and with serum ferritin levels ≥ 800 ng/mL;\n* Until availability of results from the PK Study (Study DEEP-1, EudraCT n. 2012-000658-67) for patients aged from 1 month to less than 6 years: known intolerance or contraindication to DFO;\n* Written informed consent and patient's informed assent, relating to his/her comprehension abilities and level of maturity\n\nExclusion Criteria:\n\n* Patients with intolerance or known contraindication to either DFP or DFX\n* Patients receiving DFX at a dose \\> 40 mg/kg/day or DFP at a dose \\> 100 mg/kg/day at screening\n* Platelet count \\<100.000/mm3 during the run-in phase\n* Absolute neutrophils count \\<1.500/mm3 during the run-in phase\n* Hb levels lower than 8g/dL during the run-in phase\n* Evidence of abnormal liver function\n* Iron overload from causes other than transfusional haemosiderosis\n* Severe heart dysfunction secondary to iron overload\n* Serum creatinine level \\> ULN (Upper Limit of Normal) for age during the run-in phase\n* History of significant medical or psychiatric disorder\n* The patient has received another investigational drug within 30 days prior to this clinical trial\n* Fever and other signs/symptoms of infection in the 10 days before baseline assessment\n* Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids\n* Positive test for β-HCG (Human chorionic gonadotropin) and lactating female patients"}, 'identificationModule': {'nctId': 'NCT01825512', 'briefTitle': 'Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients', 'organization': {'class': 'OTHER', 'fullName': 'Consorzio per Valutazioni Biologiche e Farmacologiche'}, 'officialTitle': 'Multicentre, Randomised, Open Label, Non-inferiority Trial to Evaluate the Efficacy and Safety of Deferiprone Compared to Deferasirox in Patients Aged From 1 Month to Less Than 18 Years Affected by Transfusion Dependent Haemoglobinopathies', 'orgStudyIdInfo': {'id': 'DEEP-2'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Deferiprone', 'description': '75-100 mg/kg/day seven days per week', 'interventionNames': ['Drug: Deferiprone']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Deferasirox', 'description': '20 to 40 mg/kg/day seven days per week', 'interventionNames': ['Drug: Deferasirox']}], 'interventions': [{'name': 'Deferiprone', 'type': 'DRUG', 'otherNames': ['DFP'], 'description': 'Deferiprone 80 mg/mL oral solution', 'armGroupLabels': ['Deferiprone']}, {'name': 'Deferasirox', 'type': 'DRUG', 'otherNames': ['DFX', 'ATC Code:V03AC03'], 'description': 'Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg', 'armGroupLabels': ['Deferasirox']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Lushnjë', 'country': 'Albania', 'facility': "Hospital 'Ihsan Çabej'", 'geoPoint': {'lat': 40.94194, 'lon': 19.705}}, {'city': 'Tirana', 'country': 'Albania', 'facility': 'Qendra Spitalore Universitare "Nene Tereza" Tirane', 'geoPoint': {'lat': 41.32744, 'lon': 19.81866}}, {'city': 'Nicosia', 'country': 'Cyprus', 'facility': 'Department of Medical and Public health Services of the Ministry of Health', 'geoPoint': {'lat': 35.17284, 'lon': 33.35397}}, {'city': 'Alexandria', 'country': 'Egypt', 'facility': 'Alexandria University Hospital - Faculty of Medicine', 'geoPoint': {'lat': 31.20176, 'lon': 29.91582}}, {'city': 'Cairo', 'country': 'Egypt', 'facility': 'Cairo University Faculty of Medicine', 'geoPoint': {'lat': 30.06263, 'lon': 31.24967}}, {'city': 'Zagazig', 'country': 'Egypt', 'facility': 'Zagazig University Hospitals', 'geoPoint': {'lat': 30.58768, 'lon': 31.502}}, {'city': 'Athens', 'country': 'Greece', 'facility': 'National And Kapodistrian University of Athens', 'geoPoint': {'lat': 37.98376, 'lon': 23.72784}}, {'city': 'Lentini', 'state': 'SR', 'country': 'Italy', 'facility': 'Centro di Thalassemia, Ospedale Civile di Lentini', 'geoPoint': {'lat': 37.28556, 'lon': 14.99737}}, {'city': 'Bari', 'country': 'Italy', 'facility': 'Università di Bari - Facoltà di Medicina', 'geoPoint': {'lat': 41.12066, 'lon': 16.86982}}, {'city': 'Cagliari', 'country': 'Italy', 'facility': 'ASL Cagliari Ospedale Regionale per le Microcitemie', 'geoPoint': {'lat': 39.23054, 'lon': 9.11917}}, {'city': 'Catania', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi', 'geoPoint': {'lat': 37.49223, 'lon': 15.07041}}, {'city': 'Cosenza', 'country': 'Italy', 'facility': 'Presidio Ospedaliero "Annunziata", Centro di Studi della Microcitemia', 'geoPoint': {'lat': 39.2989, 'lon': 16.25307}}, {'city': 'Florence', 'country': 'Italy', 'facility': 'A.O.Universitaria Meyer', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'city': 'Modena', 'country': 'Italy', 'facility': 'Clinica Pediatrica Policlinico di Modena', 'geoPoint': {'lat': 44.64783, 'lon': 10.92539}}, {'city': 'Napoli', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Rilievo Nazionale "Antonio Cardarelli"', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'city': 'Padua', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Palermo', 'country': 'Italy', 'facility': 'Ospedali Riuniti Villa Sofia - Cervello', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'city': 'Palermo', 'country': 'Italy', 'facility': 'U.O.C. Ematologia - Emoglobinopatie, Ospedale G. Di Cristina', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'city': 'Sassari', 'country': 'Italy', 'facility': 'Clinica Pediatrica Università - ASL 1 D.H per Talassemia', 'geoPoint': {'lat': 40.72586, 'lon': 8.55552}}, {'city': 'Tunis', 'country': 'Tunisia', 'facility': 'Centre National de Greffe de Moelle Osseuse Tunis', 'geoPoint': {'lat': 36.81897, 'lon': 10.16579}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Barts Health NHS Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Romford', 'country': 'United Kingdom', 'facility': "Queen's Hospital", 'geoPoint': {'lat': 51.57515, 'lon': 0.18582}}], 'overallOfficials': [{'name': 'Donato Bonifazi, Dr', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Consorzio per Valutazioni Biologiche e Farmacologiche'}, {'name': 'Aurelio Maggio, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Ospedali Riuniti Villa Sofia-Cervello'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Consorzio per Valutazioni Biologiche e Farmacologiche', 'class': 'OTHER'}, 'collaborators': [{'name': 'European Commission', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}