Apotransferrin in Patients With β-thalassemia
Phase 2
Terminated
- Conditions
- β-thalassemia Intermedia
- Interventions
- Biological: human apotransferrin
- Registration Number
- NCT03993613
- Lead Sponsor
- Prothya Biosolutions
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 10
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description human apotransferrin human apotransferrin Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.
- Primary Outcome Measures
Name Time Method Erythropoiesis 17 weeks Change of haemoglobin level and/or or change of number of RBC units transfused/week
- Secondary Outcome Measures
Name Time Method Change from baseline in serum iron 17 weeks 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 Ctrough calculated from serum transferrin levels
Cmin predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days Cmin calculated from serum transferrin levels
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
Trial Locations
- Locations (1)
Academic Medical Centre
🇳🇱Amsterdam-Zuidoost, Noord-Holland, Netherlands