The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Intravenous infusions
Academic Medical Centre
Amsterdam-Zuidoost, North Holland, Netherlands
Erythropoiesis
Change of haemoglobin level and/or or change of number of RBC units transfused/week
Time frame: 17 weeks
Change from baseline in serum iron
Time frame: 17 weeks
Change from baseline in change plasma levels of advanced glycation end products
Time frame: 17 weeks
Change in spleen size
Time frame: at baseline and at 16 weeks
Change from baseline in reticulocyte count
Time frame: 17 weeks
Change from baseline in erythropoietin levels
Time frame: 17 weeks
Ctrough
Ctrough calculated from serum transferrin levels
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmin
Cmin calculated from serum transferrin levels
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
tmax
tmax calculated from serum transferrin levels
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmax
Cmax calculated from serum transferrin levels
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
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AUCτ
AUCτ calculated from serum transferrin levels
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Ctrough
Ctrough calculated from serum transferrin levels
Time frame: predose
Adverse events
Number of adverse events
Time frame: 17 weeks