{ "FullStudy":{ "Rank":218168, "Study":{ "ProtocolSection":{ "IdentificationModule":{ "NCTId":"NCT01511848", "OrgStudyIdInfo":{ "OrgStudyId":"AinShamsU" }, "Organization":{ "OrgFullName":"Ain Shams University", "OrgClass":"OTHER" }, "BriefTitle":"Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload", "OfficialTitle":"A Prospective Randomized Comparative Study of Efficacy and Safety of Combined Deferiprone (DFP) and Deferasirox Versus DFP and Desferrioxamine (DFO) Therapy in Diseases With Severe Iron Overload" }, "StatusModule":{ "StatusVerifiedDate":"February 2012", "OverallStatus":"Unknown status", "LastKnownStatus":"Not yet recruiting", "ExpandedAccessInfo":{ "HasExpandedAccess":"No" }, "StartDateStruct":{ "StartDate":"February 2012" }, "PrimaryCompletionDateStruct":{ "PrimaryCompletionDate":"February 2013", "PrimaryCompletionDateType":"Anticipated" }, "CompletionDateStruct":{ "CompletionDate":"February 2013", "CompletionDateType":"Anticipated" }, "StudyFirstSubmitDate":"January 6, 2012", "StudyFirstSubmitQCDate":"January 18, 2012", "StudyFirstPostDateStruct":{ "StudyFirstPostDate":"January 19, 2012", "StudyFirstPostDateType":"Estimate" }, "LastUpdateSubmitDate":"February 3, 2012", "LastUpdatePostDateStruct":{ "LastUpdatePostDate":"February 6, 2012", "LastUpdatePostDateType":"Estimate" } }, "SponsorCollaboratorsModule":{ "ResponsibleParty":{ "ResponsiblePartyType":"Principal Investigator", "ResponsiblePartyInvestigatorFullName":"Mohsen Saleh Elalfy", "ResponsiblePartyInvestigatorTitle":"professour of pediatrics", "ResponsiblePartyInvestigatorAffiliation":"Ain Shams University" }, "LeadSponsor":{ "LeadSponsorName":"Ain Shams University", "LeadSponsorClass":"OTHER" } }, "OversightModule":{ "OversightHasDMC":"Yes" }, "DescriptionModule":{ "BriefSummary":"Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload\n\nPrimary Outcome Measures:\n\n• The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator.\n\nSecondary Outcome Measures:\n\n• The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.", "DetailedDescription":"Study population:\n\nBeta-thalassemia major patients; Patients with high iron stores Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months Liver iron >14 mg/g dry weight- by R2 MRI\nOther causes of transfusional hemosiderosis\n\nEstimated Enrollment: 30 patients in each arm Study Start Date: January 1st ; 2011 enrollment period 8 weeks Estimated Study Completion Date: End of February 1212\n\nArms Assigned Interventions\n\nArm 1: Patients will be treated with combined DFP and deferasirox. Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (better tolerated if started and then built up over 4 weeks).\n\nDose may be increased up to 100mg/kg/day guided by serum ferritin. Agranulocytosis risk 1-2%. Monitor TLC and granulocytic count / 2weeks. Patients need to be continually educated about this risk, know that they must stop DFP if they have a fever or infection.\n\nDrug: Deferasirox will be orally administered at a dose of 20 mg/kg once daily at evening for 5 days.\n\nOther Name: ICL670 Arm 2: Patients will be treated for 6 days with a combination of deferoxamine and DFP.\n\nInterventions:\n\nDrug: Deferoxamine\nDrug: DFP Drug: Deferoxamine Deferoxamine will be administered subcutaneously over 8 hours for 5 days at a dose of 40 mg/kg.\n\nOther Name: Desferal, DFO Drug: DFP DFP: will be orally administered at a dose of 75mg/kg orally in 3 divided doses for 7 days\n\nEligibility Ages Eligible for Study : 6 to 18Years Genders Eligible for Study: Both\n\nInclusion Criteria:\n\nSubjects with transfusional iron overload after Approval of Ethical committee giving written informed consent.\nSubjects must have a serum ferritin greater than >2500 ng/mL, a platelet count greater than 100,000/mm3, and a serum creatinine within the normal range.\nA woman of childbearing potential must have a negative serum pregnancy test at screening. She must use a medically acceptable form of birth control during the study and for 1 month afterward.\nThe subjects must also have a level of understanding and willingness to cooperate with the confinement and procedures described in the consent form and scheduled by the study site. In addition, he/she must be able to provide voluntary written informed consent.\n\nExclusion Criteria:\n\nSubjects with a past history of agranulocytosis, history of clinically significant gastrointestinal, renal, hepatic ALT > 10 times high normal, OR > 50% increase of serum creatinine from basal value, pulmonary or cardiovascular disease. Patients with a history of tuberculosis, epilepsy, psychosis, glaucoma or any other condition, which in the opinion of the investigators, would jeopardize the safety of the subject or impact the validity of the study results.\nSubjects with HIV positive or have active HCV.\nA history of serious immunologic hypersensitivity to any medication, such as prophylaxis or angioedema.\nParticipation in a previous investigational drug study within the 30 days preceding screening..\nWomen who are pregnant, or breast-feeding.\nCurrent alcohol or drug abuse.\nAn inability to adhere to the designated procedures and restrictions of this protocol.\nSubjects receiving warfarin, digoxin, or anti-arrhythmic or anti-seizure medications.\nSubjects with a known allergy to Exjade or DFP that prevents chronic administration." }, "ConditionsModule":{ "ConditionList":{ "Condition":[ "Beta-thalassemia Major", "Sickle Cell Disease", "Iron Hemosiderosis" ] }, "KeywordList":{ "Keyword":[ "Iron chelation", "Iron balance", "Secondary iron overload", "deferoxamine", "deferasirox", "Deferiprone", "1- Beta-thalassemia major patients;", "Patients with high iron stores", "Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months", "Liver iron >14 mg/g dry weight- by R2 MRI", "2- Sickle cell disease", "3- Other causes of transfusional iron hemosiderosis" ] } }, "DesignModule":{ "StudyType":"Interventional", "PhaseList":{ "Phase":[ "Phase 2", "Phase 3" ] }, "DesignInfo":{ "DesignAllocation":"Randomized", "DesignInterventionModel":"Crossover Assignment", "DesignPrimaryPurpose":"Treatment", "DesignMaskingInfo":{ "DesignMasking":"None (Open Label)" } }, "EnrollmentInfo":{ "EnrollmentCount":"60", "EnrollmentType":"Anticipated" } }, "ArmsInterventionsModule":{ "ArmGroupList":{ "ArmGroup":[ { "ArmGroupLabel":"arm 1", "ArmGroupType":"Active Comparator", "ArmGroupDescription":"30 Patients will be treated with combined DFP and deferasirox.", "ArmGroupInterventionList":{ "ArmGroupInterventionName":[ "Drug: DFP (ferriprox) and deferasirox (ICL 670)" ] } },{ "ArmGroupLabel":"arm 2", "ArmGroupType":"Active Comparator", "ArmGroupDescription":"Patients will be treated for 6 days with a combination of deferoxamine and DFP", "ArmGroupInterventionList":{ "ArmGroupInterventionName":[ "Drug: DFP, DFO" ] } } ] }, "InterventionList":{ "Intervention":[ { "InterventionType":"Drug", "InterventionName":"DFP (ferriprox) and deferasirox (ICL 670)", "InterventionDescription":"Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg 3 times/d (better tolerated if started and then built up over 4 weeks).\n\nDose may be increased up to 100mg/kg/day guided by serum ferritin. Agranulocytosis risk 1-2%. Monitor TLC and granulocytic count / 2weeks. Patients need to be continually educated about this risk, know that they must stop DFP if they have a fever or infection.\n\nDrug: Deferasirox will be orally administered at a dose of 20 mg/kg once daily at evening for 5 days.\n\nOther Name: ICL670", "InterventionArmGroupLabelList":{ "InterventionArmGroupLabel":[ "arm 1" ] } },{ "InterventionType":"Drug", "InterventionName":"DFP, DFO", "InterventionDescription":"Drug: Deferoxamine It will be administered subcutaneously over 8 hours for 5 days at a dose of 40 mg/kg.\n\nOther Name: Desferal, DFO Drug: DFP DFP: will be orally administered at a dose of 75mg/kg orally in 3 divided doses for 7 days", "InterventionArmGroupLabelList":{ "InterventionArmGroupLabel":[ "arm 2" ] } } ] } }, "OutcomesModule":{ "PrimaryOutcomeList":{ "PrimaryOutcome":[ { "PrimaryOutcomeMeasure":"to assess efficacy of combining DFP and deferasirox compared to combined DFP and DFO in decreasing the serum ferritin level in conditions with severe chronic iron overload.", "PrimaryOutcomeDescription":"• The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.", "PrimaryOutcomeTimeFrame":"12 months" } ] }, "SecondaryOutcomeList":{ "SecondaryOutcome":[ { "SecondaryOutcomeMeasure":"to determine the safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.", "SecondaryOutcomeDescription":"• The secondary outcome measure is to determine the number of patients who will develop adverse reactions upon administering the drugs in combination.", "SecondaryOutcomeTimeFrame":"12 months" } ] } }, "EligibilityModule":{ "EligibilityCriteria":"Inclusion Criteria:\n\nSubjects with transfusional iron overload secondary to thalassemia major , sickle cell disease showing up-trend in SF aged 6 Years or older, may participate after Approval of Ethical committee giving written informed consent.\nSubjects must have a serum ferritin greater than >2500 ng/mL, a platelet count greater than 100,000/mm3, and a serum creatinine within the normal range.\nA woman of childbearing potential must have a negative serum pregnancy test at screening. She must use a medically acceptable form of birth control during the study and for 1 month afterward.\nThe subjects must also have a level of understanding and willingness to cooperate with the confinement and procedures described in the consent form and scheduled by the study site. In addition, he/she must be able to provide voluntary written informed consent.\n\nExclusion Criteria:\n\nSubjects with a past history of agranulocytosis, history of clinically significant gastrointestinal, renal, hepatic ALT > 10 times high normal, OR > 50% increase of serum creatinine from basal value, pulmonary or cardiovascular disease. Patients with a history of tuberculosis, epilepsy, psychosis, glaucoma or any other condition, which in the opinion of the investigators, would jeopardize the safety of the subject or impact the validity of the study results.\nSubjects with HIV positive or have active HCV.\nA history of serious immunologic hypersensitivity to any medication, such as anaphylaxis or angioedema.\nParticipation in a previous investigational drug study within the 30 days preceding screening..\nWomen who are pregnant, or breast-feeding.\nCurrent alcohol or drug abuse.\nAn inability to adhere to the designated procedures and restrictions of this protocol.\nSubjects receiving warfarin, digoxin, or anti-arrhythmic or anti-seizure medications.\nSubjects with a known allergy to Exjade or DFP that prevents chronic administration.", "HealthyVolunteers":"No", "Gender":"All", "MinimumAge":"6 Years", "MaximumAge":"18 Years", "StdAgeList":{ "StdAge":[ "Child", "Adult" ] } }, "ContactsLocationsModule":{ "CentralContactList":{ "CentralContact":[ { "CentralContactName":"Amira A M Adly, Asst. prof.", "CentralContactRole":"Contact", "CentralContactPhone":"0105245837", "CentralContactEMail":"amiradiabetes@yahoo.com" } ] }, "OverallOfficialList":{ "OverallOfficial":[ { "OverallOfficialName":"Mohsen S. Elalfy, professour", "OverallOfficialAffiliation":"Ain Shams University", "OverallOfficialRole":"Principal Investigator" } ] }, "LocationList":{ "Location":[ { "LocationFacility":"Pediatric Hematology clinic, Ain Shams University", "LocationCity":"Cairo", "LocationCountry":"Egypt" } ] } }, "ReferencesModule":{ "ReferenceList":{ "Reference":[ { "ReferencePMID":"25600572", "ReferenceType":"derived", "ReferenceCitation":"Elalfy MS, Adly AM, Wali Y, Tony S, Samir A, Elhenawy YI. Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients. Eur J Haematol. 2015 Nov;95(5):411-20. doi: 10.1111/ejh.12507. Epub 2015 Mar 27." } ] } } }, "DerivedSection":{ "MiscInfoModule":{ "VersionHolder":"April 22, 2020" }, "InterventionBrowseModule":{ "InterventionMeshList":{ "InterventionMesh":[ { "InterventionMeshId":"D000077588", "InterventionMeshTerm":"Deferasirox" },{ "InterventionMeshId":"D000077543", "InterventionMeshTerm":"Deferiprone" } ] }, "InterventionAncestorList":{ "InterventionAncestor":[ { "InterventionAncestorId":"D000007502", "InterventionAncestorTerm":"Iron Chelating Agents" },{ "InterventionAncestorId":"D000002614", "InterventionAncestorTerm":"Chelating Agents" },{ "InterventionAncestorId":"D000064449", "InterventionAncestorTerm":"Sequestering Agents" },{ "InterventionAncestorId":"D000045504", "InterventionAncestorTerm":"Molecular Mechanisms of Pharmacological Action" } ] }, "InterventionBrowseLeafList":{ "InterventionBrowseLeaf":[ { "InterventionBrowseLeafId":"M1821", "InterventionBrowseLeafName":"Deferiprone", "InterventionBrowseLeafAsFound":"Ferriprox", "InterventionBrowseLeafRelevance":"high" },{ "InterventionBrowseLeafId":"M1848", "InterventionBrowseLeafName":"Deferasirox", "InterventionBrowseLeafAsFound":"Deferasirox", "InterventionBrowseLeafRelevance":"high" },{ "InterventionBrowseLeafId":"M9116", "InterventionBrowseLeafName":"Iron", "InterventionBrowseLeafRelevance":"low" },{ "InterventionBrowseLeafId":"M9117", "InterventionBrowseLeafName":"Iron Chelating Agents", "InterventionBrowseLeafRelevance":"low" },{ "InterventionBrowseLeafId":"M4443", "InterventionBrowseLeafName":"Chelating Agents", "InterventionBrowseLeafRelevance":"low" } ] }, "InterventionBrowseBranchList":{ "InterventionBrowseBranch":[ { "InterventionBrowseBranchAbbrev":"All", "InterventionBrowseBranchName":"All Drugs and Chemicals" },{ "InterventionBrowseBranchAbbrev":"Micro", "InterventionBrowseBranchName":"Micronutrients" } ] } }, "ConditionBrowseModule":{ "ConditionMeshList":{ "ConditionMesh":[ { "ConditionMeshId":"D000000755", "ConditionMeshTerm":"Anemia, Sickle Cell" },{ "ConditionMeshId":"D000013789", "ConditionMeshTerm":"Thalassemia" },{ "ConditionMeshId":"D000017086", "ConditionMeshTerm":"beta-Thalassemia" },{ "ConditionMeshId":"D000019190", "ConditionMeshTerm":"Iron Overload" },{ "ConditionMeshId":"D000006486", "ConditionMeshTerm":"Hemosiderosis" } ] }, "ConditionAncestorList":{ "ConditionAncestor":[ { "ConditionAncestorId":"D000000745", "ConditionAncestorTerm":"Anemia, Hemolytic, Congenital" },{ "ConditionAncestorId":"D000000743", "ConditionAncestorTerm":"Anemia, Hemolytic" },{ "ConditionAncestorId":"D000000740", "ConditionAncestorTerm":"Anemia" },{ "ConditionAncestorId":"D000006402", "ConditionAncestorTerm":"Hematologic Diseases" },{ "ConditionAncestorId":"D000006453", "ConditionAncestorTerm":"Hemoglobinopathies" },{ "ConditionAncestorId":"D000030342", "ConditionAncestorTerm":"Genetic Diseases, Inborn" },{ "ConditionAncestorId":"D000019189", "ConditionAncestorTerm":"Iron Metabolism Disorders" },{ "ConditionAncestorId":"D000008659", "ConditionAncestorTerm":"Metabolic Diseases" } ] }, "ConditionBrowseLeafList":{ "ConditionBrowseLeaf":[ { "ConditionBrowseLeafId":"M10890", "ConditionBrowseLeafName":"Neoplasm Metastasis", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M15140", "ConditionBrowseLeafName":"Thalassemia", "ConditionBrowseLeafAsFound":"Thalassemia", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M17991", "ConditionBrowseLeafName":"beta-Thalassemia", "ConditionBrowseLeafAsFound":"Beta Thalassemia", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M3696", "ConditionBrowseLeafName":"Body Weight", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M2666", "ConditionBrowseLeafName":"Anemia, Sickle Cell", "ConditionBrowseLeafAsFound":"Sickle Cell Disease", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M19762", "ConditionBrowseLeafName":"Iron Overload", "ConditionBrowseLeafAsFound":"Iron Overload", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M8155", "ConditionBrowseLeafName":"Hemosiderosis", "ConditionBrowseLeafAsFound":"Hemosiderosis", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M2651", "ConditionBrowseLeafName":"Anemia", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M2654", "ConditionBrowseLeafName":"Anemia, Hemolytic", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M8130", "ConditionBrowseLeafName":"Hemolysis", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M2656", "ConditionBrowseLeafName":"Anemia, Hemolytic, Congenital", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M8073", "ConditionBrowseLeafName":"Hematologic Diseases", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M8122", "ConditionBrowseLeafName":"Hemoglobinopathies", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M22270", "ConditionBrowseLeafName":"Genetic Diseases, Inborn", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M10222", "ConditionBrowseLeafName":"Metabolic Diseases", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M19761", "ConditionBrowseLeafName":"Iron Metabolism Disorders", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"T5646", "ConditionBrowseLeafName":"Thalassemia", "ConditionBrowseLeafAsFound":"Thalassemia", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"T739", "ConditionBrowseLeafName":"Beta-thalassemia", "ConditionBrowseLeafAsFound":"Beta Thalassemia", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"T5256", "ConditionBrowseLeafName":"Sickle Cell Anemia", "ConditionBrowseLeafAsFound":"Sickle Cell Disease", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"T2728", "ConditionBrowseLeafName":"Hemosiderosis", "ConditionBrowseLeafAsFound":"Hemosiderosis", "ConditionBrowseLeafRelevance":"high" } ] }, "ConditionBrowseBranchList":{ "ConditionBrowseBranch":[ { "ConditionBrowseBranchAbbrev":"BC04", "ConditionBrowseBranchName":"Cancers and Other Neoplasms" },{ "ConditionBrowseBranchAbbrev":"BC23", "ConditionBrowseBranchName":"Symptoms and General Pathology" },{ "ConditionBrowseBranchAbbrev":"All", "ConditionBrowseBranchName":"All Conditions" },{ "ConditionBrowseBranchAbbrev":"BC15", "ConditionBrowseBranchName":"Blood and Lymph Conditions" },{ "ConditionBrowseBranchAbbrev":"BC16", "ConditionBrowseBranchName":"Diseases and Abnormalities at or Before Birth" },{ "ConditionBrowseBranchAbbrev":"BC18", "ConditionBrowseBranchName":"Nutritional and Metabolic Diseases" },{ "ConditionBrowseBranchAbbrev":"Rare", "ConditionBrowseBranchName":"Rare Diseases" } ] } } } } } }