Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
Condition: Iron Overload; Sickle Cell Disease; Other Anemias
MeSH terms: Anemia; Anemia, Sickle Cell; Iron Overload
Intervention: Deferiprone; Deferoxamine
Intervention MeSH terms: Deferiprone; Deferoxamine
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 230.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: sickle cell disease; Iron overload; Deferiprone; Ferriprox; deferoxamine; Chelation

Summary:

      This research is being done so that we can look at the safety and efficacy of deferiprone in
      people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron
      from the body. We will be comparing deferiprone with deferoxamine, another drug that removes
      iron from the body.
    

Eligibility criteria:

        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

        Exclusion Criteria:

          1. Thalassemia syndromes;

          2. Myelodysplastic syndrome (MDS) or myelofibrosis;

          3. Diamond Blackfan anemia;

          4. Primary bone marrow failure;

          5. 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* <10ms;

         15. Myocardial infarction, cardiac arrest or cardiac failure within 1 year before
             screening/baseline visit;

         16. Unable to undergo MRI

         17. 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