Apotransferrin in Atransferrinemia

• Status: Recruiting
• Trial end date: 2022-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sanquin; Sanquin Plasma Products BV

Criteria

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