Ignite Creation Date:
2025-12-25 @ 3:09 AM
Ignite Modification Date:
2026-02-22 @ 4:52 PM
Study NCT ID:
NCT01874405
Status:
COMPLETED
Last Update Posted:
2013-06-11
First Post:
2013-05-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Retrospective Evaluation of Adult and Pediatric Transfusion-dependent Patients Treated With Deferasirox Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019190', 'term': 'Iron Overload'}, {'id': 'D013789', 'term': 'Thalassemia'}], 'ancestors': [{'id': 'D019189', 'term': 'Iron Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 80}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2003-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-06', 'completionDateStruct': {'date': '2012-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-06-06', 'studyFirstSubmitDate': '2013-05-26', 'studyFirstSubmitQcDate': '2013-06-06', 'lastUpdatePostDateStruct': {'date': '2013-06-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-06-11', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'cardiac T2* in patients treated with deferasirox', 'timeFrame': 'at least 1 year', 'description': 'change from baseline to end of study in cardiac T2\\*, as measured by Magnetic Resonance, in patients with iron overload (cardiac T2\\* \\<20 ms at baseline)'}, {'measure': 'cardiac T2* in patients treated with deferasirox', 'timeFrame': 'at least 1 year', 'description': 'maintenance from baseline to end of study of cardiac T2\\* in not iron overloaded patients (cardiac T2\\* \\>20 ms at baseline)'}], 'secondaryOutcomes': [{'measure': 'cardiac function in patient undergoing deferasirox treatment', 'timeFrame': 'at least 1 year', 'description': 'change in left and right ejection fraction, telediastolic and telesystolic volumes, stroke volumes, cardiac output, myocardial mass, measured by Cardiac Magnetic Resonance, from baseline to end of study'}, {'measure': 'change in liver iron concentration', 'timeFrame': 'at least 1 year'}, {'measure': 'maintenance of normal endocrine function in patients without endocrine dysfunction and improvement in disease severity in patients affected by endocrine dysfunction from baseline to end of study', 'timeFrame': 'at least 3 years', 'description': 'Thyroid function (TSH, free triiodothyronine and free thyroxine serum free T4 levels), pancreatic cell function (basal glycemia, glycated hemoglobin level), bone mineral density (z-score) will be evaluated by the closest assessment to baseline (first deferasirox exposure) and to the end of study'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Iron overload', 'thalassaemia', 'deferasirox', 'magnetic resonance', 'endocrine function'], 'conditions': ['Iron Overload']}, 'referencesModule': {'references': [{'pmid': '25197009', 'type': 'DERIVED', 'citation': 'Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26.'}]}, 'descriptionModule': {'briefSummary': 'Iron overload is a leading cause of morbidity and mortality in transfusion-dependent patients. Deferasirox is the most promising iron chelator agent in several clinical scenarios. The investigators propose a retrospective study (chart review) to evaluate comprehensive iron overload management in transfusion-dependent patients treated with deferasirox for up to 5-10 years in a real clinical practice setting.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Adult and pediatric transfusion- dependent patients (male and female) with different underlying cronic anemias who received iron chelation therapy with deferasirox during the observational study period and underwent at least 2 cardiac MRI scans at the Pozzuoli site.\n\nAll consecutive patients visited at the participating sites starting from March 2003 to October 2012 will be entered in this observational study (chart review) provided all the inclusion an no exclusion criteria are met.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Transfusion- dependent patients (\\> 2 years);\n* Ongoing deferasirox therapy during the study period;\n* ≥ 2 Magnetic Resonance scans (one at baseline and at least one post baseline - as per clinical need) during study period (this criteria is not mandatory for patients undergoing only the endocrine subanalysis and participating only to the cardiac analysis);\n* Available medical history including relevant clinical and laboratory data (e.g serum ferritin, liver function tests, renal function tests, endocrine parameters ) at baseline before starting deferasirox treatment\n\nExclusion Criteria:\n\n* Non transfusion- dependent patients;\n* Other chelation therapy than deferasirox;\n* Absence of complete medical history as above specified'}, 'identificationModule': {'nctId': 'NCT01874405', 'briefTitle': 'Retrospective Evaluation of Adult and Pediatric Transfusion-dependent Patients Treated With Deferasirox Therapy', 'organization': {'class': 'OTHER', 'fullName': 'University of Campania Luigi Vanvitelli'}, 'officialTitle': 'Effectiveness of Deferasirox Therapy on Comprehensive Management of Iron Overload in Adult and Pediatric Transfusion-dependent Patients: a Long Term Retrospective Study', 'orgStudyIdInfo': {'id': 'DIPEDNA-2013-1'}}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Silverio Perrotta, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Campania Luigi Vanvitelli'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Campania Luigi Vanvitelli', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD', 'investigatorFullName': 'Silverio Perrotta', 'investigatorAffiliation': 'University of Campania Luigi Vanvitelli'}}}}