In short
Clinical trials are investigating Human Apotransferrin in people with congenital atransferrinaemia and hypotransferrinaemia. These studies aim to check safety, how the treatment behaves in the body, and whether it can improve blood test results such as hemoglobin and ferritin.
Key points
- Clinical trials of Human Apotransferrin are studying rare inherited blood conditions, especially congenital atransferrinaemia and hypotransferrinaemia. The main trial is a Phase 2 interventional study with 5 participants. It is designed to assess safety, pharmacokinetics, and early signs of benefit. Researchers are measuring hemoglobin, transferrin pharmacokinetics, and serum ferritin. The study is authorised and focuses on patients with these rare conditions.
Clinical trial overview
The available trial is an interventional study, which means researchers give Human Apotransferrin and then measure what happens in the patients. The study is focused on congenital atransferrinaemia and hypotransferrinaemia, two rare conditions linked to very low transferrin levels.
The trial brief summary says it is designed to investigate the pharmacokinetics, efficacy, and safety of apotransferrin replacement therapy. In simple terms, the study is asking whether the treatment reaches the body in the expected way, may help the disease, and can be given safely.
Who the trial is for
The study population includes patients with congenital atransferrinaemia or hypotransferrinaemia. These are rare inherited disorders, so the trial is very small and targeted to a specific group of patients.
The source data do not list wider eligibility details such as age limits, sex limits, or other medical requirements. Based on the title and condition field, the main target group is people diagnosed with atransferrinemia-related disease.
Study design and phase
This trial is in Phase 2, which usually means researchers are looking more closely at whether the treatment shows benefit while still watching safety. The study is also listed as Authorised, meaning it has been approved to proceed in the source record.
The intervention is Human Apotransferrin given as a intravenous infusion, which means it is delivered through a vein. The source lists the product as Human Apotransferrin (50g/l) and gives the dose as 360 mg/kg.
What the trial measures
The primary outcomes are increase of hemoglobin into the normal range, pharmacokinetics of transferrin, and decrease of serum ferritin into the normal range. These are important blood measures that help show whether the treatment may improve anemia-related findings and iron balance.
Hemoglobin is the part of red blood cells that carries oxygen, so a rise toward the normal range may suggest improvement in blood health. Serum ferritin is a blood test that reflects iron stores, so a drop toward normal may show a better iron balance in the body.
Pharmacokinetics means how the body handles the treatment, such as how it moves through the body over time. This helps researchers understand whether Human Apotransferrin behaves as expected after infusion.
Treatment given in the trial
The intervention is Human Apotransferrin, given by intravenous infusion. The study title describes it as a treatment for patients with atransferrinemia, and the summary describes it as replacement therapy.
No other treatments, comparison groups, or placebo details are provided in the source data. Because of that, the article can only describe the trial as a single focused study of Human Apotransferrin in a rare disease setting.
Study status and size
The trial status is listed as Authorised. The planned enrollment is 5 participants, which shows that this is a very small study designed for a rare condition.
With such a small enrollment, the study is likely intended to gather early clinical information rather than provide a final answer for all patients. The source data do not provide final results, so the article can only report the study plan and endpoints.