Table of Contents
- Clinical trial overview
- Who the trial is for
- Study design and phase
- What the trial measures
- Treatment given in the trial
- Study status and size
Clinical trial overview
The available trial is an interventional study, which means researchers give Human Apotransferrin and then measure what happens in the patients.[1] The study is focused on congenital atransferrinaemia and hypotransferrinaemia, two rare conditions linked to very low transferrin levels.[1]
The trial brief summary says it is designed to investigate the pharmacokinetics, efficacy, and safety of apotransferrin replacement therapy.[1] 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.[1]
Who the trial is for
The study population includes patients with congenital atransferrinaemia or hypotransferrinaemia.[1] These are rare inherited disorders, so the trial is very small and targeted to a specific group of patients.[1]
The source data do not list wider eligibility details such as age limits, sex limits, or other medical requirements.[1] Based on the title and condition field, the main target group is people diagnosed with atransferrinemia-related disease.[1]
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.[1] The study is also listed as Authorised, meaning it has been approved to proceed in the source record.[1]
The intervention is Human Apotransferrin given as a intravenous infusion, which means it is delivered through a vein.[1] The source lists the product as Human Apotransferrin (50g/l) and gives the dose as 360 mg/kg.[1]
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.[1] These are important blood measures that help show whether the treatment may improve anemia-related findings and iron balance.[1]
Hemoglobin is the part of red blood cells that carries oxygen, so a rise toward the normal range may suggest improvement in blood health.[1] Serum ferritin is a blood test that reflects iron stores, so a drop toward normal may show a better iron balance in the body.[1]
Pharmacokinetics means how the body handles the treatment, such as how it moves through the body over time.[1] This helps researchers understand whether Human Apotransferrin behaves as expected after infusion.[1]
Treatment given in the trial
The intervention is Human Apotransferrin, given by intravenous infusion.[1] The study title describes it as a treatment for patients with atransferrinemia, and the summary describes it as replacement therapy.[1]
No other treatments, comparison groups, or placebo details are provided in the source data.[1] Because of that, the article can only describe the trial as a single focused study of Human Apotransferrin in a rare disease setting.[1]
Study status and size
The trial status is listed as Authorised.[1] The planned enrollment is 5 participants, which shows that this is a very small study designed for a rare condition.[1]
With such a small enrollment, the study is likely intended to gather early clinical information rather than provide a final answer for all patients.[1] The source data do not provide final results, so the article can only report the study plan and endpoints.[1]
