Apotransferrin in Atransferrinemia

Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients

Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to investigate the pharmacokinetics, efficacy and safety of Apotransferrin replacement therapy in atransferrinemia patients.

Inclusion Criteria:

• Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl
• Informed consent

Exclusion Criteria:

• Known with allergic reactions against human plasma or plasma products
• Having detectable anti-immunoglobulin A antibodies