Clinical Trials for Blood Iron Increased
There is currently 1 ongoing clinical trial investigating treatments for conditions associated with elevated blood iron levels. This trial focuses on atransferrinemia, a rare genetic disorder where the body lacks sufficient transferrin protein to properly transport iron, leading to iron accumulation in organs. The trial is being conducted across multiple European countries and is testing a replacement therapy called Human Apotransferrin.
Clinical trial locations
- Germany
- Italy
- Spain
Study on the Use of Human Apotransferrin for Treating Patients with Atransferrinemia
This study is investigating a treatment for atransferrinemia, a very rare blood disorder where the body has extremely low levels of transferrin, a crucial protein responsible for transporting iron throughout the bloodstream. When transferrin is missing or deficient, iron cannot be properly distributed to where it is needed, causing it to accumulate dangerously in organs like the liver and heart. At the same time, patients experience anemia because iron is not available for making hemoglobin, the protein in red blood cells that carries oxygen.
Who can participate: The study is looking for patients who have a confirmed diagnosis of atransferrinemia, specifically with transferrin levels below 40 mg/dL. Both men and women of various ages can participate. Participants must be willing and able to sign an informed consent form, which explains all aspects of the study and confirms their agreement to take part.
Who cannot participate: The study excludes patients who do not have congenital atransferrinemia or hypotransferrinemia. Those who fall outside the specific age ranges set by the study, who are unwilling or unable to follow study procedures and attend appointments, or who have other medical conditions that might interfere with the study results or compromise their safety are also excluded.
What the study involves: The investigational treatment is Human Apotransferrin, given as an intravenous infusion directly into the bloodstream. The solution contains 50 grams per liter of human apotransferrin. The study uses a dose escalation approach, meaning participants will receive different doses to help researchers determine the most effective and safe amount of the therapy. The treatment works by replacing the missing or deficient transferrin protein, allowing iron to be properly bound and transported to cells throughout the body.
Study goals: Researchers are evaluating three main aspects of this treatment. First, they want to understand the pharmacokinetics, which means how the body absorbs, distributes, processes, and eliminates the drug. Second, they will measure effectiveness by monitoring increases in hemoglobin and hematocrit levels, which indicate healthier blood function, as well as decreases in serum ferritin and reduction of iron overload in the liver and heart. Third, they will carefully assess safety by monitoring for any adverse effects throughout the study period.
The study involves regular check-ups and monitoring appointments. After initial assessments and confirmation of eligibility, participants will begin receiving the treatment through intravenous infusions. The frequency and duration of these infusions will be adjusted based on individual response and study protocols. Throughout participation, the study team will conduct regular evaluations to track how well the treatment is working and to ensure participant safety. The study is estimated to conclude by January 2028.
Summary
Currently, there is one clinical trial addressing conditions related to elevated blood iron levels, specifically focusing on the rare genetic disorder atransferrinemia. This trial is being conducted across three European countries: Germany, Italy, and Spain, reflecting a coordinated international effort to study this extremely rare condition. The trial is investigating Human Apotransferrin as a protein replacement therapy delivered intravenously, with the goal of restoring the body’s ability to properly transport iron and prevent harmful iron accumulation in vital organs. Given the rarity of atransferrinemia, this study represents an important opportunity for patients with this condition to access an investigational treatment designed specifically for their needs.
