Apotransferrin in Atransferrinemia

Prothya Biosolutions

Status and phase: Active, not recruiting; Phase 2

Conditions: Congenital Atransferrinemia

Treatments: Drug: Human apotransferrin

Study type: Interventional

Funder types: Industry

Identifiers

NCT01797055

MD2009.04

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

human apotransferrin

Experimental group

Description:

intravenous apotransferrin every 4-8 weeks

Treatment:

Drug: Human apotransferrin