Last updated on July 2017

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


Brief description of 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.

Detailed Study 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.

Clinical Study Identifier: NCT02041299

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Caroline Fradette, PhD

Children's Hospital Oakland
Oakland, CA United States
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Caroline Fradette, PhD

University of Illinois at Chicago
Chicago, IL United States
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Renee Gardner

Children's Hospital
New Orleans, LA United States
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Caroline Fradette, PhD

University of Michigan Comprehensive Cancer Center
Ann Arbor, MI United States
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Sharada Sarnaik

Children's Hospital of Michigan
Detroit, MI United States
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Caroline Fradette, PhD

Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, NY United States
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Janet Kwiatkowski

The Children's Hospital of Philadephia
Philadelphia, PA United States
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Caroline Fradette, PhD

Thomas Jefferson University
Philadelphia, PA United States
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Julie Kantor

Medical University of South Carolina
Charleston, SC United States
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Caroline Fradette, PhD

Centro Infantil Boldrini
Campinas, Brazil
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Caroline Fradette, PhD

Hospital de Cl nicas de Porto Alegre
Porto Alegre, Brazil
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Caroline Fradette, PhD

Instituto Estadual de Hematologia
Rio de Janeiro, Brazil
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Caroline Fradette, PhD

Casa de Sa de Santa Marcelina
São Paulo, Brazil
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Caroline Fradette, PhD

Universidade Federal de S o Paulo
São Paulo, Brazil
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Suzan Williams

Hospital for Sick Kids
Toronto, ON Canada
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Mohammed Elsafy

Zagazig University
Alexandria, Egypt
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Mohsen Saleh Elalfy

Ains Shams University
Cairo, Egypt
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Mona Hamdy

Cairo University
Cairo, Egypt
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Amal El Beshlawy

Pediatric Hospital of Cairo University
Cairo, Egypt
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Ahmed Mansour

Mansoura University Children's Hospital
Mansoura, Egypt
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Abdulrahman Alshehri

Asser Central Hospital
Abha, Saudi Arabia
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Mohammed Hassan Qari

King Abdulaziz University Hospital
Jeddah, Saudi Arabia
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Caroline Fradette, PhD

King Khalid University Hospital
Riyadh, Saudi Arabia
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Banu Kaya

Barts and The London
London, United Kingdom
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Baba Inusa

Evelina Children's Hospital
London, United Kingdom
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Caroline Fradette, PhD

Imperial College Healthcare NHS Trust
London, United Kingdom
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Caroline Fradette, PhD

St. Mary's Hospital and Hammersmith Hospital
London, United Kingdom
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