Apotransferrin in Atransferrinemia

Phase 2

Active, not recruiting

• Conditions: Congenital Atransferrinemia
• Interventions: Drug: Human apotransferrin

• Registration Number: NCT01797055
• Lead Sponsor: Prothya Biosolutions

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-12-01
• Study First Submitted: 2013-02-20
• Study First Submitted QC: 2013-02-20
• Study First Posted: 2013-02-22
• Primary Completion: 2022-03-31
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-30
• Last Update Posted: 2025-08-03
• Study Completion: 2028-01-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 5

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
human apotransferrin	Human apotransferrin	intravenous apotransferrin every 4-8 weeks

Primary Outcome Measures

Name	Time	Method
Hemoglobin	15 year	Hemoglobin within normal range
pharmacokinetics of transferrin	first infusion and year 3	serum transferrin levels measured on several time points before and after infusion
iron overload in organs	15 years	Elimination of iron overload (liver, heart)

Secondary Outcome Measures

Name	Time	Method
Erythrocytes	15 years	Erythrocytes within normal values
Iron overload	15 years	Serum Ferritin within normal values
Hematocrit	15 years	Hematocrit within normal values

Trial Locations

Locations (3)

Klinikum Aschaffenburg; 🇩🇪 Aschaffenburg, Germany

AO San Gerardo; 🇮🇹 Monza, Italy

Vall d'Hebron Hospital; 🇪🇸 Barcelona, Spain