Apotransferrin in Atransferrinemia

Summary

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.

Timeline

Start

2010-12

Primary completion

2022-03-31

Completion

2028-01

Drugs

Indications