A study of a drug (Ferriprox®) that removes extra iron from the body, to see how safe it is and how well it works in patients with sickle cell disease or other anaemias

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 300

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the efficacy of deferiprone vs. deferoxamine in the treatment of iron overload in patients with sickle cell disease or other anemias.;<br> Secondary Objective: To evaluate the effect of deferiprone vs. deferoxamine on the patients’ quality of life;<br> To evaluate the safety and tolerability of deferiprone vs. deferoxamine<br> ;Primary end point(s): The change from baseline to Week 52 in liver iron concentration (measured by MRI).;Timepoint(s) of evaluation of this end point: MRI scans will be performed at the screening/baseline, Week 26, and Week 52 (or early termination) visits

Secondary Outcome Measures

Name	Time	Method

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