The Efficacy and Safety of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias

Study point of contact

Fernando Tricta, MD
[email protected]
Caroline Fradette, PhD
[email protected]


9 United States sites

8 Egypt sites

5 Brazil sites

4 United Kingdom sites

3 Saudi Arabia sites

3 Tunisia sites

3 Turkey sites

1 Canada site

1 Qatar site


> 2 Years


Phase 4

Study type








About the study

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.

participation requirements

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

participation restrictions

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

8. History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or

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


  • Oakland, California, United States, Children's Hospital Oakland, 94609 [Completed]
  • Chicago, Illinois, United States, University of Illinois at Chicago, 60612 [Withdrawn]
  • New Orleans, Louisiana, United States, Children's Hospital, 70118 [Completed]
  • Ann Arbor, Michigan, United States, University of Michigan Comprehensive Cancer Center, 48109 [Completed]
  • Detroit, Michigan, United States, Children's Hospital of Michigan, 48201 [Active, not recruiting]
  • New Hyde Park, New York, United States, Steven and Alexandra Cohen Children's Medical Center of New York, 11040 [Withdrawn]
  • Philadelphia, Pennsylvania, United States, The Children's Hospital of Philadephia, 19104-4399 [Recruiting]
  • Philadelphia, Pennsylvania, United States, Thomas Jefferson University, 19107 [Completed]
  • Charleston, South Carolina, United States, Medical University of South Carolina, 29425 [Completed]
  • Campinas, Brazil, Centro Infantil Boldrini [Completed]
  • Porto Alegre, Brazil, Hospital de Clínicas de Porto Alegre [Withdrawn]
  • Rio de Janeiro, Brazil, Instituto Estadual de Hematologia [Withdrawn]
  • São Paulo, Brazil, Casa de Saúde Santa Marcelina [Completed]
  • São Paulo, Brazil, Universidade Federal de São Paulo [Completed]
  • Toronto, Ontario, Canada, Hospital for Sick Kids [Completed]
  • Alexandria, Egypt, Alexandria University [Not yet recruiting]
  • Alexandria, Egypt, Pediatric Department and Clinical Research Center, Faculty of Medicine [Not yet recruiting]
  • Alexandria, Egypt, Zagazig University [Completed]
  • Cairo, Egypt, Ains Shams University [Recruiting]
  • Cairo, Egypt, Cairo University [Recruiting]
  • Cairo, Egypt, Pediatric Hospital of Cairo University [Recruiting]
  • Mansoura, Egypt, Mansoura University Children's Hospital [Completed]
  • Tanta, Egypt, Tanta University Hospital [Not yet recruiting]
  • Doha, Qatar, Hamad Medical Corporation National Center for Cancer Care and Research [Not yet recruiting]
  • Abha, Saudi Arabia, Asser Central Hospital [Recruiting]
  • Jeddah, Saudi Arabia, King Abdulaziz University Hospital [Withdrawn]
  • Riyadh, Saudi Arabia, King Khalid University Hospital [Terminated]
  • Tunis, Bad Saadoun, Tunisia, National Center for Bone Marrow Transplantation [Recruiting]
  • Sousse, Tunisia, Farhat Hached Hospital, Hematology Department [Recruiting]
  • Tunis, Tunisia, Principal Military Hospital of Instruction of Tunis [Recruiting]
  • Adana, Turkey, Cukurova University [Completed]
  • Ankara, Turkey, Hacettepe University [Completed]
  • Istanbul, Turkey, Istanbul University [Recruiting]
  • London, United Kingdom, Barts and The London [Completed]
  • London, United Kingdom, Evelina Children's Hospital [Recruiting]
  • London, United Kingdom, Imperial College Healthcare NHS Trust [Completed]
  • London, United Kingdom, St. Mary's Hospital and Hammersmith Hospital [Withdrawn]