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Active, Not Recruiting

NCT Number: NCT01797055

Apotransferrin in Atransferrinemia

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Congenital Atransferrinemia Anemia Hematologic Diseases Hemic and Lymphatic Diseases Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Klinikum Aschaffenburg, Aschaffenburg, Germany

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Human apotransferrin

Drug

intravenous infusion

Primary outcomes

  1. Hemoglobin

    Time frame: 15 year

    Hemoglobin within normal range

  2. pharmacokinetics of transferrin

    Time frame: first infusion and year 3

    serum transferrin levels measured on several time points before and after infusion

  3. iron overload in organs

    Time frame: 15 years

    Elimination of iron overload (liver, heart)

Secondary outcomes

  1. Iron overload

    Time frame: 15 years

    Serum Ferritin within normal values

  2. Hematocrit

    Time frame: 15 years

    Hematocrit within normal values

  3. Erythrocytes

    Time frame: 15 years

    Erythrocytes within normal values

Other outcomes

  1. transferrin levels

    Time frame: 15 years

    Effective transferrin levels

  2. adverse drug reactions

    Time frame: 15 years

    number of adverse drug reactions

Sponsors and collaborators

Lead sponsor

Prothya Biosolutions

Industry

Registry information

Official study title

Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients

Important dates

Study start
2010
Primary completion
2022
Study completion
2028
First posted
Feb 22, 2013
Registry last updated
Aug 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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