Clinical trial to investigate the treatment with apotransferrin of patients with atransferrinemia

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-017409-13-DE

Enrollment

Registered

2010-12-20

Start date

2011-05-25

Completion date

Unknown

Last updated

2024-08-12

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

Conditions

Atransferrinemia MedDRA version: 20.0 Level: LLT Classification code 10055212 Term: Hypochromic microcytic anemia System Organ Class: 100000004851 MedDRA version: 20.0 Level: LLT Classification code 10002272 Term: Anemia System Organ Class: 100000004851 MedDRA version: 21.0 Level: PT Classification code 10065973 Term: Iron overload System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 21.0 Level: PT Classification code 10005620 Term: Blood iron increased System Or

Interventions

Product Name: apotransferrin Product Code: apotransferrin Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human Apotransferrin Other descriptive name: APOTRANSFERRIN Concentration unit

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl 2. Informed consent Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Known with allergic reactions against human plasma or plasma products 2. Having detectable anti-IgA antibodies

Design outcomes

Primary

Measure	Time frame
Main Objective: To investigate the pharmacokinetics, efficacy and safety of apotransferrin replacement therapy;Secondary Objective: To determine the optimal dose regimen;Primary end point(s): The primary efficacy parameters are the concentration of transferrin, ferritin, Hb and iron in derum as well as the level of transferrin saturation and total binding capacity. PK parameters.;Timepoint(s) of evaluation of this end point: 1. after first phase of the study (108 weeks); 2. after 3 years follow-up; 3. after end of extended follow-up	—

Measure

Time frame

Main Objective: To investigate the pharmacokinetics, efficacy and safety of apotransferrin replacement therapy;Secondary Objective: To determine the optimal dose regimen;Primary end point(s): The primary efficacy parameters are the concentration of transferrin, ferritin, Hb and iron in derum as well as the level of transferrin saturation and total binding capacity. PK parameters.;Timepoint(s) of evaluation of this end point: 1. after first phase of the study (108 weeks); 2. after 3 years follow-up; 3. after end of extended follow-up

—

Secondary

Measure	Time frame
Secondary end point(s): Safety and clinical tolerance will be monitored by adverse event surveillance and laboratory measurements.;Timepoint(s) of evaluation of this end point: Monitoring on ongoing basis	—

Countries

Germany, Italy, Spain

Contacts

Public ContactIlona Kleine Budde

Prothya Biosolutions Netherlands B.V.

ilona.kleinebudde@prothya.com+31205123537

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026