Apotransferrin in Patients With β-thalassemia

Prothya Biosolutions

Status and phase: Terminated; Phase 2

Conditions: β-thalassemia Intermedia

Treatments: Biological: human apotransferrin

Study type: Interventional

Funder types: Industry

Identifiers

NCT03993613

MD2014.01

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

human apotransferrin

Experimental group

Description:

Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.

Treatment:

Biological: human apotransferrin