β-thalassemia Intermedia
Conditions
Brief summary
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.
Interventions
BIOLOGICALhuman apotransferrin
Intravenous infusions
Sponsors
Prothya Biosolutions
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anaemia in combination with an elevated HbA2 (\>2.5%) and a haemoglobin of \<6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule. * Age above≥ 17 years. * Adequate renal and hepatic function tests * WHO performance 0, 1 or 2. * Signed informed consent.
Exclusion criteria
* Known with allergic reactions against human plasma or plasma products. * Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease). * Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias. * Pregnant or lactating females. * Known with IgA deficiency with anti-IgA antibodies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Erythropoiesis | 17 weeks | Change of haemoglobin level and/or or change of number of RBC units transfused/week |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in change plasma levels of advanced glycation end products | 17 weeks | — |
| Change in spleen size | at baseline and at 16 weeks | — |
| Change from baseline in reticulocyte count | 17 weeks | — |
| Change from baseline in erythropoietin levels | 17 weeks | — |
| Ctrough | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | Ctrough calculated from serum transferrin levels |
| Change from baseline in serum iron | 17 weeks | — |
| tmax | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | tmax calculated from serum transferrin levels |
| Cmax | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | Cmax calculated from serum transferrin levels |
| AUCτ | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | AUCτ calculated from serum transferrin levels |
| Adverse events | 17 weeks | Number of adverse events |
| Cmin | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | Cmin calculated from serum transferrin levels |
Countries
Netherlands
Outcome results
None listed