This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
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 long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias. Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
134
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Number of Patients With Adverse Events
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
Time frame: From the first day of the study until the last study visit (Week 104 or early termination)
Change From Baseline in Liver Iron Concentration (LIC)
LIC was measured by MRI, in units of mg of iron per gram of liver (dry weight). The change from baseline in LIC was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Time frame: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Cardiac MRI T2*
The change from baseline in cardiac MRI T2\* was determined for three different periods of exposure to deferiprone: one year, two years, and three years
Time frame: One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Serum Ferritin
The change from baseline in serum ferritin (SF) was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Time frame: One year, two years, and three years after the start of deferiprone therapy
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The Children's Hospital of Philadephia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Hospital for Sick Kids
Toronto, Ontario, Canada
Zagazig University
Alexandria, Egypt
Ain Shams University
Cairo, Egypt
Cairo University
Cairo, Egypt
Pediatric Hospital of Cairo University
Cairo, Egypt
...and 3 more locations