Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias

• Status: Terminated
• Trial end date: 2019-06-18
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ApoPharma
• Treatments: Deferiprone; Deferoxamine

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