The efficacy and safety of Ferriprox® for the treatment of transfusional iron overload in patients with sickle cell disease or other anemias - FIRST (Ferriprox in patients with iron overload in sickle cell disease trial)

ApoPharma Inc.0 个研究点目标入组 300 人开始时间: 2013年10月10日最近更新: 2019年12月16日

试验速览

暂无简介。

•1.Male or female = 2 years of age;
•2.Have sickle cell disease (confirmed by Hb electrophoresis or more specific tests) or other conditions with iron overload from repeated blood transfusions (see exclusion criteria for exceptions);
•3.Baseline LIC >7 mg/g dw (measured by MRI);
•4.Patients who have received no less than 20 transfusions of RBCs;
•5.Patients who have received at least 1 transfusion per year in the last 2 years and who are expected to have a continuing requirement (based on Investigator’s judgement) during the duration of the trial
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 120
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 170
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 10

•1.Thalassemia syndromes;
•2.Myelodysplastic syndrome (MDS) or myelofibrosis;
•3.Diamond Blackfan anemia;
•4.Primary bone marrow failure;
•5.Patients with a baseline LIC >30 mg/g dw (measured by MRI);
•6.Unable or unwilling to undergo a 7-day washout period if currently being treated with deferiprone or deferoxamine or deferasirox;
•7.Previous discontinuation of treatment with deferiprone or deferoxamine due to adverse events;
•8.History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or deferoxamine;
•9.Treated with hydroxyurea within 30 days;
•10.History of malignancy;
•11.Evidence of abnormal liver function (serum ALT level(s) > 5 times upper limit of normal at screening or creatinine levels >2 times upper limit of normal at screening);
•12.A serious, unstable illness, as judged by the Investigator, during the past 3 months before screening/baseline visit including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease;
•13.Clinically significant abnormal 12-lead ECG findings;
•14.Cardiac MRI T2* <10 ms
•15.Unable to undergo MRI
•16.Presence of metallic objects such as artificial joints, inner ear (cochlear) implants, brain aneurysm clips, pacemakers, and metallic foreign bodies in the eye or other body areas that would prevent use of MRI imaging

发起方