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

Information source: ApoPharma
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Iron Overload; Sickle Cell Disease; Other Anemias

Intervention: Deferiprone (Drug); Deferoxamine (Drug)

Phase: Phase 4

Status: Recruiting

Sponsored by: ApoPharma

Official(s) and/or principal investigator(s):
Janet Kwiatkowski, MD, Principal Investigator, Affiliation: Children's Hospital of Philadelphia

Overall contact:
Caroline Fradette, PhD, Phone: 416-401-7543, Email: cfradett@apopharma.com

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.

Clinical Details

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

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Change in liver iron concentration, as measured in mg/g dry weight (dw) using MRI

Secondary outcome:

Change in patient-reported quality of life, as measured by SF-36 or Child Health Questionnaire

Change in cardiac MRI T2*, measured in milliseconds (ms)

Change in serum ferritin, measured in mcg/L

Occurrence of adverse events

Frequency of serious adverse events (SAEs)

Hematology assessments

Blood clinical biochemistry assessments

Abnormal and clinically significant findings in 12-lead ECG

Discontinuations due to AEs

Detailed description: Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the efficacy, safety, and tolerability of deferiprone vs. deferoxamine in patients who have SCD or other anemias, and who require chelation because of the extra iron they are taking in through blood transfusions. About 300 people from North America, South America, Europe, and the Middle East will take part in this study. Participants will be randomized in a 2: 1 ratio to receive therapy for 52 weeks with either deferiprone or deferoxamine, another type of iron chelator. Patients who are randomized to the deferiprone group can choose to get the drug as either tablets or liquid, and must take it three times daily. Patients who are randomized to the deferoxamine group will receive it as a subcutaneous infusion that lasts from 8 to 12 hours and is given 5 to 7 days per week. For both drugs, the starting dosage is based on how much extra iron they have taken in through transfusions in the last 3 months and on the severity of iron load, as measured by serum ferritin levels in the blood and by the amount of iron in the liver and the heart. For deferiprone, the starting dosage will be increased each week over the first 3 weeks; and for both drugs, the dosage may be adjusted up or down during the study based on the level of iron overload and on safety considerations. Patients will need to have their blood count checked every week; to give a blood sample for more detailed safety testing every month; and to give a blood sample for the measurement of serum ferritin every 3 months. Every six months, they will undergo an ECG and an MRI scan, and will be asked to complete a quality of life survey. At the end of the 52 weeks, participants will be invited to enter a 2-year study in which all patients will receive deferiprone, including those who were randomized to receive deferoxamine in the first year.

Eligibility

Minimum age: 2 Years. Maximum age: N/A. Gender(s): Both.

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

Locations and Contacts

Caroline Fradette, PhD, Phone: 416-401-7543, Email: cfradett@apopharma.com

Centro Infantil Boldrini, Campinas, Brazil; Recruiting
Monica Verissimo, Principal Investigator

Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Recruiting
Christina Bittar, Principal Investigator

Instituto Estadual de Hematologia, Rio de Janeiro, Brazil; Recruiting
Clarisse Lobo, Principal Investigator

Casa de Saúde Santa Marcelina, São Paulo, Brazil; Recruiting
Sandra Calegare, Principal Investigator

Universidade Federal de São Paulo, São Paulo, Brazil; Recruiting
Maria Stella Figueiredo, Principal Investigator

Zagazig University, Alexandria, Egypt; Recruiting
Mohammed Badr, Principal Investigator

Zagazig University, Alexandria, Egypt; Recruiting
Mohammed Elsafy, Principal Investigator

Ains Shams University, Cairo, Egypt; Recruiting
Mohsen Saleh Elalfy, Principal Investigator

Cairo University, Cairo, Egypt; Recruiting
Mona Hamdy, Principal Investigator

Pediatric Hospital of Cairo University, Cairo, Egypt; Recruiting
Amal El Beshlawy, Principal Investigator

Mansoura University Children's Hospital, Mansoura, Egypt; Recruiting
Ahmed Mansour, Principal Investigator

Asser Central Hospital, Abha, Saudi Arabia; Not yet recruiting
Abdulrahman Alshehri, Principal Investigator

King Abdulaziz University Hospital, Jeddah, Saudi Arabia; Recruiting
Mohammed Hassan Qari, Principal Investigator

King Khalid University Hospital, Riyadh, Saudi Arabia; Recruiting
Abdulkareem Almomen, Principal Investigator

Barts and The London, London, United Kingdom; Recruiting
Banu Kaya, Principal Investigator

Evelina Children's Hospital, London, United Kingdom; Recruiting
Baba Inusa, Principal Investigator

Imperial College Healthcare NHS Trust, London, United Kingdom; Recruiting
Subarna Chakravorty, Principal Investigator
Mark Layton, Sub-Investigator

St. Mary's Hospital and Hammersmith Hospital, London, United Kingdom; Recruiting
Sadaqat Luqmani, Principal Investigator

Children's Hospital Oakland, Oakland, California, United States; Recruiting
Elliott Vichinsky, Principal Investigator

University of Illinois at Chicago, Chicago, Illinois 60612, United States; Recruiting
Victor Gordeuk, Principal Investigator

Children's Hospital, New Orleans, Louisiana 70118, United States; Recruiting
Renee Gardner, Principal Investigator

University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, United States; Recruiting
Andrew Campbell, Principal Investigator

Children's Hospital of Michigan, Detroit, Michigan 48201, United States; Recruiting
Sharada Sarnaik, Principal Investigator

Steven and Alexandra Cohen Children's Medical Center of New York, New Hyde Park, New York, United States; Withdrawn

Hospital for Sick Kids, Toronto, Ontario, Canada; Recruiting
Suzan Williams, Principal Investigator

The Children's Hospital of Philadephia, Philadelphia, Pennsylvania, United States; Recruiting
Janet Kwiatkowski, Principal Investigator

Thomas Jefferson University, Philadelphia, Pennsylvania, United States; Terminated

Medical University of South Carolina, Charleston, South Carolina, United States; Recruiting
Julie Kantor, Principal Investigator

Additional Information

Starting date: March 2014
Last updated: August 17, 2015

Page last updated: August 20, 2015

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