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1 clinical trial in this condition
GermanyIn short
There is currently 1 ongoing clinical trial for hypochromic anaemia, focusing on a rare genetic form of the condition called atransferrinemia. This trial is testing a treatment called human apotransferrin in patients across multiple European countries, aiming to restore the body's ability to transport iron properly and reduce organ damage caused by iron overload.
This clinical trial is investigating a treatment for a very rare blood disorder called atransferrinemia, which is a form of hypochromic anaemia. In this condition, the body has extremely low levels of transferrin, a protein that acts like a shuttle to carry iron through the bloodstream. Without enough transferrin, iron cannot reach the cells that need it to make healthy red blood cells, leading to anaemia. At the same time, iron builds up in organs like the liver and heart, potentially causing serious damage.
Who can participate: To join this study, participants must have a confirmed diagnosis of atransferrinemia, meaning their transferrin levels are below 40 mg/dL. Both men and women can participate, and people from vulnerable populations who may need extra care are also eligible. Most importantly, participants must be willing and able to sign an informed consent form, which explains all aspects of the study.
Who cannot participate: The study excludes patients who do not have the specific conditions of congenital atransferrinemia or hypotransferrinaemia. People outside the specified age range for the trial cannot join. Anyone who is unable or unwilling to follow the study procedures, attend appointments regularly, or follow the study team's instructions will be excluded. Additionally, patients with other medical conditions that might interfere with the study or put them at risk cannot participate.
What the study involves: The trial tests a treatment called human apotransferrin, which is given as an intravenous infusion directly into the bloodstream. The solution contains 50 grams per liter of human apotransferrin. The main goal is to understand how the body processes this treatment, determine the best and safest dose, and evaluate how well it works to manage the condition.
Participants will receive different doses of apotransferrin to help researchers find the most effective amount. Throughout the study, which is expected to continue until January 2028, regular check-ups will monitor important blood markers such as hemoglobin and hematocrit levels, which indicate healthy blood function. The study will also track iron levels in organs like the liver and heart to ensure the treatment reduces dangerous iron overload without causing new problems.
Investigational drug: Apotransferrin is a protein-based therapeutic agent designed to replace the missing or deficient transferrin in patients. It works by binding to free iron in the blood and transporting it to cells where it is needed for vital functions, particularly for making hemoglobin in red blood cells. This approach aims to address both the anaemia and the iron overload that characterize atransferrinemia.
Currently, only one clinical trial is actively recruiting patients with hypochromic anaemia, specifically those with the rare genetic form called atransferrinemia. This trial is taking place across three European countries: Germany, Italy, and Spain. The focus is entirely on testing human apotransferrin as a potential treatment to restore normal iron transport in the body, improve blood cell production, and reduce the dangerous buildup of iron in vital organs. For patients with this extremely rare condition, this trial represents an important opportunity to access a novel treatment approach that directly addresses the underlying cause of their condition.
This guide is here to help you understand the condition. It does not replace a conversation with your doctor, who knows your situation best.
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