Ignite Creation Date:
2025-12-24 @ 11:44 PM
Ignite Modification Date:
2025-12-25 @ 9:37 PM
Study NCT ID:
NCT01250951
Status:
COMPLETED
Last Update Posted:
2016-12-12
First Post:
2010-11-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D019190', 'term': 'Iron Overload'}], 'ancestors': [{'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D019189', 'term': 'Iron Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077588', 'term': 'Deferasirox'}], '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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 111}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-12', 'completionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-12-08', 'studyFirstSubmitDate': '2010-11-11', 'studyFirstSubmitQcDate': '2010-11-29', 'lastUpdatePostDateStruct': {'date': '2016-12-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-12-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade', 'timeFrame': 'Baseline assessment is followed by monthly assessments for up to 1 year'}], 'secondaryOutcomes': [{'measure': 'changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination', 'timeFrame': 'Baseline assessment is followed by monthly assessments for up to 1 year.'}, {'measure': 'changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade', 'timeFrame': 'at baseline and 1 at year (at the end of study).'}, {'measure': 'Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.', 'timeFrame': 'From the start of study up to 1 year'}]}, 'conditionsModule': {'keywords': ['Iron Overload', 'hemotranfusion', 'deferasirox', 'ferritin', 'Rare anemia', 'Transfusional Iron Overload'], 'conditions': ['Myelodysplastic Syndrome', 'Thalassemia']}, 'referencesModule': {'references': [{'type': 'RESULT', 'citation': 'Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. Epub 2009 Dec 8. Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. Epub 2009 Nov 30.'}], 'seeAlsoLinks': [{'url': 'http://www.nlm.nih.gov/medlineplus/druginfo/meds/a606002.html', 'label': 'Deferasirox'}, {'url': 'http://www.nlm.nih.gov/medlineplus/thalassemia.html', 'label': 'Thalassemia'}, {'url': 'http://vsearch.nlm.nih.gov/vivisimo/cgi-bin/query-meta?v%3aproject=medlineplus&v%3asources=medlineplus-bundle&query=myelodysplastic%20syndrome&', 'label': 'Myelodisplastic Syndrome'}, {'url': 'http://www.medlit.ru/journal/annotation/1375', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Age ≥ 2 years\n2. Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)\n3. ECOG Performance Status ≤ 2\n4. Transfusion overload confirmed with ferritin level \\>1000 µg/l.\n5. No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).\n6. Serum creatine level \\> ULN\n7. No proteinuria\n8. Liver enzymes level \\< 5 ULN.\n9. No pregnancy or lactation\n10. Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.\n\nExclusion Criteria:\n\n1. Age \\< 2 years\n2. No iron overload (Ferritin level \\<1000 µg/l).\n3. Primary iron overload (hereditary hemochromatosis)\n4. Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)\n5. Elevated serum creatinine \\> ULN or/and proteinuria\n6. Liver enzymes level \\>5 ULN.\n7. Pregnancy or lactation.\n\nOther protocol-defined inclusion/exclusion criteria may apply"}, 'identificationModule': {'nctId': 'NCT01250951', 'briefTitle': 'This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': '1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.', 'orgStudyIdInfo': {'id': 'CICL670ARU01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Deferasirox', 'interventionNames': ['Drug: Deferasirox']}], 'interventions': [{'name': 'Deferasirox', 'type': 'DRUG', 'otherNames': ['ICL670'], 'armGroupLabels': ['Deferasirox']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Moscow', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'Novartis Investigative Site', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}