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Study on the Use of Human Apotransferrin for Treating Patients with Atransferrinemia

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare blood disorder called atransferrinemia. Atransferrinemia is a condition where the body has very low levels of a protein called transferrin, which is important for transporting iron in the blood. The treatment being tested in this study is called Human Apotransferrin, which is given as a solution through an intravenous infusion. This means the treatment is delivered directly into the bloodstream through a vein.

The purpose of the study is to evaluate how the body processes Human Apotransferrin, as well as its effectiveness and safety in treating atransferrinemia. Participants in the study will receive different doses of Human Apotransferrin to find the most effective and safe dose. The study will monitor changes in important blood components, such as hemoglobin and hematocrit, which are indicators of healthy blood function. It will also look at how the treatment affects iron levels in the body, including the liver and heart, to ensure there is no iron overload.

Throughout the study, participants will have regular check-ups to assess their response to the treatment and to monitor any side effects. The study aims to improve the understanding of how Human Apotransferrin can help manage atransferrinemia and improve the quality of life for those affected by this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after confirming an established diagnosis of atransferrinemia, which is a condition where transferrin levels in the blood are below 40 mg/dL.

    Informed consent must be signed to proceed with the study.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to evaluate baseline health status and confirm eligibility for the study.

  3. Step 3

    Treatment administration

    The treatment involves the administration of human apotransferrin through an intravenous infusion.

    The solution for infusion contains 50g/l of human apotransferrin.

  4. Step 4

    Dose escalation

    The study is designed to evaluate different doses of apotransferrin to determine the most effective and safe dosage.

    The frequency and duration of administration are adjusted based on individual response and study protocols.

  5. Step 5

    Monitoring and evaluation

    Regular monitoring is conducted to assess the pharmacokinetics, which is how the body absorbs, distributes, metabolizes, and excretes the drug.

    Efficacy is evaluated by measuring increases in hemoglobin and hematocrit levels, as well as decreases in serum ferritin and iron overload in the liver and heart.

    Safety assessments are performed to monitor any adverse effects.

  6. Step 6

    Completion of study

    The study is estimated to conclude by January 1, 2028.

    Final evaluations are conducted to assess the overall outcomes and effectiveness of the treatment.

Who can join the trial?

4 criteria

  • Must have a confirmed diagnosis of atransferrinemia, which means having very low levels of a protein called transferrin in the blood, specifically below 40 mg/dL.
  • Must have signed an informed consent, which is a document that explains the study and confirms that you agree to participate.
  • Both male and female participants are eligible.
  • Participants can be from a vulnerable population, which means they might need extra protection or care.

Who cannot join the trial?

5 criteria

  • Patients who do not have the specific condition called congenital atransferrinaemia or hypotransferrinaemia cannot participate. These are rare conditions where the body has very low levels of a protein called transferrin, which is important for carrying iron in the blood.
  • Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups, so if you are outside these age ranges, you would be excluded.
  • Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for people with specific characteristics or conditions.
  • Patients who are not willing or able to follow the study procedures cannot participate. This means you need to be able to attend appointments and follow instructions given by the study team.
  • Patients who have other medical conditions that might interfere with the study cannot participate. This is to ensure the safety of the participants and the accuracy of the study results.
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Investigated drugs

Apotransferrin is a therapy being studied for its effects on patients with atransferrinemia. This therapy aims to replace the missing or deficient protein in the blood, which is crucial for transporting iron throughout the body. The study is focused on understanding how the body processes this therapy, its effectiveness in treating the condition, and its safety for patients.

What is already known about the treatment

Apotransferrin – Apotransferrin is administered intravenously, allowing it to directly enter the bloodstream. It is currently being studied in clinical trials to assess its pharmacokinetics, efficacy, and safety for treating congenital atransferrinemia, a rare blood disorder. The main therapeutic indication for apotransferrin is to replace the missing or deficient transferrin in patients, helping to transport iron throughout the body. At the molecular level, apotransferrin binds to free iron ions, facilitating their transport to cells where they are needed. It is classified as a protein-based therapeutic agent.

Investigated diseases

Congenital Atransferrinemia – This is a rare genetic disorder characterized by the absence or severe deficiency of transferrin, a protein that binds and transports iron in the blood. Without sufficient transferrin, iron cannot be properly distributed throughout the body, leading to iron overload in various organs such as the liver and heart. This condition often results in anemia, as iron is not available for the production of hemoglobin, the protein in red blood cells that carries oxygen. Symptoms may include fatigue, weakness, and developmental delays. Over time, the iron overload can cause damage to organs, but the progression varies among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515986-33-00Protocol codeMD2009.04Estimated enrolment5 patientsSponsorProthya Biosolutions Netherlands B.V.

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