Apotransferrin in Patients With β-thalassemia

Study Overview
=================
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.

Official Title
-----------------
Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia

Conditions
-----------------
β-thalassemia Intermedia

Intervention / Treatment
-----------------
* Biological: human apotransferrin


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: human apotransferrin<br>Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks. | Biological: human apotransferrin<br>* Intravenous infusions<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Erythropoiesis | Change of haemoglobin level and/or or change of number of RBC units transfused/week | 17 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Ctrough calculated from serum transferrin levels | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | 
| Cmin | Cmin calculated from serum transferrin levels | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | 
| tmax | tmax calculated from serum transferrin levels | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | 
| Cmax | Cmax calculated from serum transferrin levels | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | 
| AUCτ | AUCτ calculated from serum transferrin levels | predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days | 
| Ctrough | Ctrough calculated from serum transferrin levels | predose | 
| Adverse events | Number of adverse events | 17 weeks |