Dose escalating study to evaluate pharmacokinetics, efficacy and safety of apotransferrin in atransferrinemia patients

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-515986-33-00

Acronym

MD2009.04

Enrollment

Registered

2024-08-02

Start date

2010-11-12

Completion date

Unknown

Last updated

2026-01-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital atransferrinaemia/ hypotransferrinaemia

Brief summary

Increase of hemoglobin (to normal range), Pharmacokinetics of transferrin, Decrease of serum ferritin (to normal range)

Detailed description

Increase of hematocrit (to normal range), Increase of erythrocytes (to normal range), Decrease of iron overload (liver, heart), Transferrin levels, transferrin saturation, and serum iron in relation to efficacy, Undetectable NTBI/LPI levels

Interventions

DRUGHuman Apotransferrin (50g/l)

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Increase of hemoglobin (to normal range), Pharmacokinetics of transferrin, Decrease of serum ferritin (to normal range)	—

Secondary

Measure	Time frame
Increase of hematocrit (to normal range), Increase of erythrocytes (to normal range), Decrease of iron overload (liver, heart), Transferrin levels, transferrin saturation, and serum iron in relation to efficacy, Undetectable NTBI/LPI levels	—

Countries

Germany, Italy, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026