Safety and Efficacy of Early-Start Deferiprone Treatment in Infants and Young Children Newly Diagnosed with Transfusion-Dependent Beta Thalassemia

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This study assesses whether early chelation with deferiprone is effective at delaying the progression of iron overload in infants and young children. Patients with beta thalassemia who are over 6 months old and less than 10 years old, receive regular transfusions, but who have not yet started chelation therapy, are eligible for this study. Subjects will be randomly assigned to take either deferiprone or a placebo three times daily for up to a year. Regular blood tests will be performed to track iron levels and monitor safety. Physical exams, blood and urine tests, and interviews will be done throughout the study to monitor safety and compliance with the medicine.

Eligibility & Criteria

IRB #:
17-014674
Official Title:
Safety and Efficacy of Early-Start Deferiprone Treatment in Infants and Young Children Newly Diagnosed with Transfusion-Dependent Beta Thalassemia
Study Phase:
Phase IV
Eligible Age Range:
6 Months - 10 Years
Gender:
All
Study Categories: