Ignite Creation Date:
2025-12-24 @ 5:10 PM
Ignite Modification Date:
2026-02-28 @ 11:20 AM
Study NCT ID:
NCT02049450
Status:
COMPLETED
Last Update Posted:
2017-07-17
First Post:
2014-01-28
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D013163', 'term': 'Splenomegaly'}], 'ancestors': [{'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'}, {'id': 'D006984', 'term': 'Hypertrophy'}, {'id': 'D020763', 'term': 'Pathological Conditions, Anatomical'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C540383', 'term': 'ruxolitinib'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'trialandresults.registries@novartis.com', 'phone': '862-778-8300', 'title': 'Study Director', 'organization': 'Novartis Pharmaceuticals'}, 'certainAgreement': {'otherDetails': "The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse Events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit.', 'eventGroups': [{'id': 'EG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement', 'otherNumAtRisk': 30, 'otherNumAffected': 23, 'seriousNumAtRisk': 30, 'seriousNumAffected': 6}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Increased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pleuritic pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}], 'seriousEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Drug-induced liver injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}, {'term': 'Pneumonia viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 30, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (18.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'classes': [{'categories': [{'measurements': [{'value': '-5.934', 'spread': '22.1681', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'week 6 to week 30 interval', 'description': "Change of RBC transfusion requirement measured as percent change of the hematocrit-adjusted volume of transfused RBC and observed during within on-treatment interval (any time-points of RBC transfusion between week 6 and week 30 driven by the individual patient's need) compared to baseline (defined by pre-treatment interval between Week - 24 to start of treatment).", 'unitOfMeasure': '% change of hematocrit-adjusted volume', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Per-Protocol Set (PPS) consisted of a subset of patients in the Safety Set who were compliant with requirements of the Study Protocol. Patients were excluded from the PPS if: they had no or incomplete history of RBC transfusions within 24 weeks prior to the first dose of ruxolitinib or discontinued treatment with ruxolitinib prior to Week 18.'}, {'type': 'SECONDARY', 'title': 'Percentage Change in Spleen Volume (cm3)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'classes': [{'title': '% change from baseline at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-19.733', 'spread': '16.0539', 'groupId': 'OG000'}]}]}, {'title': '% change from baseline at Week 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-26.829', 'spread': '16.6936', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, week 12, week 30', 'description': 'Change of spleen volume from baseline at week 12 and week 30 as measured by magnetic imaging resonance (MRI) or computed tomography (CT).', 'unitOfMeasure': 'percentage change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Set consisted of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study.'}, {'type': 'SECONDARY', 'title': 'Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'classes': [{'title': 'Weeks 0 - 6 )', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.43', 'spread': '10.135', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 6 - 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.87', 'spread': '10.555', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 12 - 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.78', 'spread': '11.081', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 18 - 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.56', 'spread': '9.760', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 24 - 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.06', 'spread': '14.321', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, weeks 0 - 30', 'description': 'Change from baseline in pre-transfusion hemoglobin levels', 'unitOfMeasure': 'percentage change of hemoglobin levels', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Set consists of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study.'}, {'type': 'SECONDARY', 'title': 'Percentage Change in Spleen Length (cm) Below the Left Coastal Margin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'classes': [{'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-11.19', 'spread': '15.376', 'groupId': 'OG000'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-22.11', 'spread': '23.604', 'groupId': 'OG000'}]}]}, {'title': 'Week 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-25.01', 'spread': '24.178', 'groupId': 'OG000'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-26.94', 'spread': '25.343', 'groupId': 'OG000'}]}]}, {'title': 'Week 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-33.85', 'spread': '25.251', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-49.29', 'spread': '26.792', 'groupId': 'OG000'}]}]}, {'title': 'Week 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-56.32', 'spread': '29.994', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-56.93', 'spread': '29.552', 'groupId': 'OG000'}]}]}, {'title': 'Week 30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-57.40', 'spread': '36.970', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, weeks 1,2,3,4,6,12,18,24,30', 'description': 'Change of spleen length from baseline over time measured by palpitation by time', 'unitOfMeasure': 'percentage change in spleen length', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Set consists of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK) Parameter of Cmin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '10 mg Bid', 'description': 'Patients who received INC422 10mg bid'}, {'id': 'OG001', 'title': '15mg Bid', 'description': 'Patients who received INC422 15mg bid'}, {'id': 'OG002', 'title': '20mg Bid', 'description': 'Patients who received INC422 20mg bid'}], 'classes': [{'title': 'Week 2 (Day 15)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '7.5800', 'spread': '7.57959', 'groupId': 'OG000'}, {'value': 'NA', 'spread': 'NA', 'comment': 'N/A = per protocol, dose increase was not allowed prior to Week 6 so at week 2 no dose \\>10mg BID was expected', 'groupId': 'OG001'}, {'value': 'NA', 'spread': 'NA', 'comment': 'N/A = per protocol, dose increase was not allowed prior to Week 6 so at week 2 no dose \\>10mg BID was expected', 'groupId': 'OG002'}]}]}, {'title': 'Week 12 (Day 85)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '9.1300', 'spread': '7.61039', 'groupId': 'OG000'}, {'value': '18.5400', 'spread': '23.99940', 'groupId': 'OG001'}, {'value': '20.2300', 'spread': '25.98617', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'week 2, week 12', 'description': 'C min of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 15 (Week 2), and Day 85 (Week 12). Cmin was collected immediately prior to dosing. n= number of patients with valid PK samples as per definition of the PK analysis set.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK analysis set includes all patients with at least one evaluable PK sample at any visit.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK) Parameter of Cmax', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '30', 'groupId': 'OG001'}, {'value': '16', 'groupId': 'OG002'}, {'value': '2', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': '5mg Bid', 'description': 'Patients who received INC422 5mg bid'}, {'id': 'OG001', 'title': '10mg Bid', 'description': 'Patients who received INC422 10mg bid'}, {'id': 'OG002', 'title': '15mg Bid', 'description': 'Patients who received INC422 15mg bid'}, {'id': 'OG003', 'title': '20mg Bid', 'description': 'Patients who received INC422 20mg bid'}], 'classes': [{'title': 'Day 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '29', 'groupId': 'OG001'}, {'value': '16', 'groupId': 'OG002'}, {'value': '2', 'groupId': 'OG003'}]}], 'categories': [{'measurements': [{'value': '58.2000', 'spread': '0.00', 'groupId': 'OG000'}, {'value': '126.8000', 'spread': '58.70337', 'groupId': 'OG001'}, {'value': '0.00', 'spread': '0.00', 'groupId': 'OG002'}, {'value': '0.00', 'spread': '0.00', 'groupId': 'OG003'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '16', 'groupId': 'OG002'}, {'value': '2', 'groupId': 'OG003'}]}], 'categories': [{'measurements': [{'value': '56.7000', 'spread': '0.00', 'groupId': 'OG000'}, {'value': '125.400', 'spread': '40.61805', 'groupId': 'OG001'}, {'value': '0.00', 'spread': '0.00', 'groupId': 'OG002'}, {'value': '0.00', 'spread': '0.00', 'groupId': 'OG003'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}, {'value': '16', 'groupId': 'OG002'}, {'value': '2', 'groupId': 'OG003'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}, {'value': '107.2100', 'spread': '50.07525', 'groupId': 'OG001'}, {'value': '245.6900', 'spread': '50.00362', 'groupId': 'OG002'}, {'value': '185.0000', 'spread': '97.58074', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1, Week 2 (Day 15), Week 12 (Day 85)', 'description': 'Cmax (1h) of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 1, Week 2, and Week 12. Cmax was collected within a +/- 1 hour post dose.\n\nn= number of patients with valid PK samples as per definition of the PK analysis set.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PK analysis set includes all patients with at least one evaluable PK sample at any visit.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '30'}]}, {'type': 'Discontinued Treatment Prior to Week 30', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'Entered Extension Phase', 'achievements': [{'comment': 'Of 26 who completed Core phase 18 moved into Ext. phase; All 18 eventually discontinued Ext. phase', 'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Completed = Completed Core phase of study', 'groupId': 'FG000', 'numSubjects': '26'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Patients/guardian decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Approximately 30 patients were planned to be enrolled in the study. 30 patients were analyzed in the full analysis, PK, and safety sets; 27 patients were analyzed in the per-protocol set.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'INC424 (Ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '25.9', 'spread': '6.83', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '18', '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': 30}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-05-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-06', 'completionDateStruct': {'date': '2016-04-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-06-19', 'studyFirstSubmitDate': '2014-01-28', 'resultsFirstSubmitDate': '2017-03-28', 'studyFirstSubmitQcDate': '2014-01-28', 'lastUpdatePostDateStruct': {'date': '2017-07-17', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-03-28', 'studyFirstPostDateStruct': {'date': '2014-01-30', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-06-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-04-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC)', 'timeFrame': 'week 6 to week 30 interval', 'description': "Change of RBC transfusion requirement measured as percent change of the hematocrit-adjusted volume of transfused RBC and observed during within on-treatment interval (any time-points of RBC transfusion between week 6 and week 30 driven by the individual patient's need) compared to baseline (defined by pre-treatment interval between Week - 24 to start of treatment)."}], 'secondaryOutcomes': [{'measure': 'Percentage Change in Spleen Volume (cm3)', 'timeFrame': 'baseline, week 12, week 30', 'description': 'Change of spleen volume from baseline at week 12 and week 30 as measured by magnetic imaging resonance (MRI) or computed tomography (CT).'}, {'measure': 'Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals', 'timeFrame': 'baseline, weeks 0 - 30', 'description': 'Change from baseline in pre-transfusion hemoglobin levels'}, {'measure': 'Percentage Change in Spleen Length (cm) Below the Left Coastal Margin', 'timeFrame': 'baseline, weeks 1,2,3,4,6,12,18,24,30', 'description': 'Change of spleen length from baseline over time measured by palpitation by time'}, {'measure': 'Pharmacokinetics (PK) Parameter of Cmin', 'timeFrame': 'week 2, week 12', 'description': 'C min of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 15 (Week 2), and Day 85 (Week 12). Cmin was collected immediately prior to dosing. n= number of patients with valid PK samples as per definition of the PK analysis set.'}, {'measure': 'Pharmacokinetics (PK) Parameter of Cmax', 'timeFrame': 'Day 1, Week 2 (Day 15), Week 12 (Day 85)', 'description': 'Cmax (1h) of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 1, Week 2, and Week 12. Cmax was collected within a +/- 1 hour post dose.\n\nn= number of patients with valid PK samples as per definition of the PK analysis set.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['thalassemia', 'thalassemia major', 'spleen enlargement', 'INC424', 'ruxolitinib'], 'conditions': ['Thalassemia Major']}, 'descriptionModule': {'briefSummary': 'Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as early death, growth retardation, bone deformations and iron overload due to blood transfusions. Splenectomy can significantly reduce transfusion requirement in thalassemia patients, but it is associated with an increased risk of serious complications such as sepsis and thrombosis. Preliminary preclinical and clinical data suggested that JAK2 inhibition, by reducing spleen size, could improve hemoglobin levels, thereby eliminating the need for splenectomy and reducing transfusion requirement and related iron overload.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.\n* Patients with spleen enlargement at Screening, defined as spleen palpable below the costal margin and spleen volume of ≥ 450 cm3 as confirmed by MRI (or CT scan in applicable patients).\n* Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening\n\nExclusion Criteria:\n\n* Splenectomy prior to or planned during the study\n* Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)\n* Hemoglobin \\<65 g/L (\\<4.0 mmol/L) at Screening\n* Platelet count \\<75×109/L, absolute neutrophils count \\< 1.5×109/L at Screening.\n* Estimated MDRD \\< 30 mL/min/1.73 m2 at Screening.\n* ALT (SGPT) levels \\>5 times ULN at Screening.\n* Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis.\n* HIV positivity'}, 'identificationModule': {'nctId': 'NCT02049450', 'briefTitle': 'Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'A Single Arm, Multicenter, Phase IIa Study to Explore the Efficacy and Safety of Ruxolitinib (INC424) in Regularly Transfused Patients With Thalassemia', 'orgStudyIdInfo': {'id': 'CINC424X2201'}, 'secondaryIdInfos': [{'id': '2013-002812-28', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'INC424 (ruxolitinib) - Study Treatment', 'description': 'Regularly transfused adult patients with thalassemia and spleen enlargement.', 'interventionNames': ['Drug: ruxolitinib']}], 'interventions': [{'name': 'ruxolitinib', 'type': 'DRUG', 'otherNames': ['INC424'], 'description': 'Ruxolitinib was taken at a starting dose of 10 mg twice daily with dose adjustments within the range of 5 to 25 mg twice daily.', 'armGroupLabels': ['INC424 (ruxolitinib) - Study Treatment']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'GR-115 27', 'city': 'Athens', 'state': 'GR', 'country': 'Greece', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 37.98376, 'lon': 23.72784}}, {'zip': '20122', 'city': 'Milan', 'state': 'MI', 'country': 'Italy', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '90146', 'city': 'Palermo', 'state': 'PA', 'country': 'Italy', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'zip': '1107 2020', 'city': 'Beirut', 'country': 'Lebanon', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 33.89332, 'lon': 35.50157}}, {'zip': '10700', 'city': 'Bangkok', 'country': 'Thailand', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}, {'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}}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', '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 review 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 availability is according to the criteria and process described on www.clinicalstudydatarequest.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}