Haemophilia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 16 Jun 2026 04:02:00 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Haemophilia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study testing fitusiran to prevent bleeding in boys aged 1 to 11 years with hemophilia A or B with or without inhibitors https://clinicaltrials.eu/trial/a-study-testing-fitusiran-to-prevent-bleeding-in-boys-aged-1-to-11-years-with-hemophilia-a-or-b-with-or-without-inhibitors/ Wed, 29 Apr 2026 15:07:59 +0000 https://clinicaltrials.eu/trial/a-study-testing-fitusiran-to-prevent-bleeding-in-boys-aged-1-to-11-years-with-hemophilia-a-or-b-with-or-without-inhibitors/ This study is looking at hemophilia, which is a condition where blood does not clot properly because the body does not make enough of certain clotting proteins. The study focuses on two types of this condition: hemophilia A, where the body lacks enough of a clotting protein called Factor VIII, and hemophilia B, where there is not enough of a clotting protein called Factor IX. Some people with hemophilia develop substances in their blood called inhibitory antibodies that work against these clotting factors, making treatment more difficult. The study will test a medicine called SAR439774, which contains the active substance fitusiran. This medicine is given as an injection under the skin.

The purpose of this study is to see how well fitusiran works to prevent bleeding episodes in boys between 1 and less than 12 years old who have hemophilia A or B, whether or not they have inhibitory antibodies. The study will compare how often bleeding happens when participants use fitusiran versus when they use their usual treatment, which is called standard of care and may include clotting factor concentrates or bypassing agents. Bypassing agents are special medicines used when inhibitory antibodies are present because they help blood clot in a different way.

During the study, participants will receive fitusiran treatment for a period of time, and there will also be a period where they use their standard of care treatment so that doctors can compare the results. The study will look at how many bleeding episodes happen during these different treatment periods, including bleeding that happens on its own without injury and bleeding that happens in the joints. Doctors will also check for any side effects and will measure changes in physical activity, pain levels, and overall quality of life. Some participants may be joining this study after being in a previous study with fitusiran, and they will have a different treatment schedule. The study will last several years to gather enough information about how safe and effective fitusiran is for preventing bleeding in young boys with hemophilia.

]]> Study of Long-term Safety and Efficacy of Fitusiran in Patients with Hemophilia A or B with or without Inhibitory Antibodies to Factor VIII or IX https://clinicaltrials.eu/trial/57036/ Wed, 29 Apr 2026 14:29:19 +0000 https://clinicaltrials.eu/trial/57036/ This clinical trial is focused on studying hemophilia A and B, which are bleeding disorders where the blood doesn’t clot properly. The therapy being investigated is called fitusiran, a medication designed to help control bleeding in people with these conditions. The aim of the study is to observe the long-term safety and how well fitusiran is tolerated by patients.

Participants in the study will receive fitusiran as a solution for injection. It is important to note that some patients involved might also have inhibitors to clotting factors, which means their bodies might reject certain treatments. In these cases, alternative treatments such as Factor VIII Inhibitor Bypassing Activity, Antithrombin III Human, Eptacog Alfa (Activated), Simoctocog Alfa, and Nonacog Alfa may be used. These are all different therapies aimed at helping the blood to clot more effectively.

The trial is designed to track the number of patients who experience any new health problems during the treatment, known as treatment emergent adverse events. It will also look at how often bleeding occurs, how this affects the joints, and the overall quality of life for those receiving fitusiran over a long period. Some participants might be given a placebo for comparison. The information gathered from this study will help better understand the long-term effects and safety of fitusiran in managing hemophilia.

]]> Study of Fitusiran for Preventing Bleeding in Boys Aged 1 to 12 with Hemophilia A or B https://clinicaltrials.eu/trial/study-of-fitusiran-for-preventing-bleeding-in-boys-aged-1-to-12-with-hemophilia-a-or-b/ Wed, 29 Apr 2026 14:28:48 +0000 https://clinicaltrials.eu/trial/study-of-fitusiran-for-preventing-bleeding-in-boys-aged-1-to-12-with-hemophilia-a-or-b/ This clinical trial is focused on studying hemophilia, a condition where blood does not clot properly, leading to excessive bleeding. The study involves children aged 1 to less than 12 years who have severe forms of hemophilia A or hemophilia B. The treatment being tested is called fitusiran, which is given as a solution for injection under the skin. Fitusiran is also known by its code name, SAR439774. The purpose of the study is to confirm the appropriate dose levels of fitusiran for these young patients.

During the study, participants will receive regular doses of fitusiran. The study will monitor how the treatment affects the levels of a protein called antithrombin in the blood, which plays a role in blood clotting. Researchers will also keep track of any side effects that participants might experience and measure the amount of fitusiran in the blood over time. The study aims to ensure that the treatment is safe and effective for children with severe hemophilia.

This trial is open-label, meaning that both the researchers and participants know which treatment is being administered. The study is multinational, involving participants from different countries. The goal is to gather enough information to determine the best dosing strategy for fitusiran in young children with hemophilia, ultimately aiming to improve their quality of life by reducing bleeding episodes.

]]> Study on Fitusiran for Preventing Bleeding in Males Aged 12 and Older with Severe Hemophilia A or B, with or without Inhibitors https://clinicaltrials.eu/trial/study-on-fitusiran-for-preventing-bleeding-in-males-aged-12-and-older-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ Wed, 29 Apr 2026 14:24:06 +0000 https://clinicaltrials.eu/trial/study-on-fitusiran-for-preventing-bleeding-in-males-aged-12-and-older-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ This clinical trial is focused on studying the effects of a medication called fitusiran in individuals with severe hemophilia A or B. Hemophilia is a genetic disorder that affects the blood’s ability to clot, leading to excessive bleeding. The study aims to understand how well fitusiran can prevent bleeding episodes in male participants aged 12 years and older, whether or not they have developed inhibitory antibodies to factor VIII or IX, which are proteins involved in blood clotting.

Participants in the study will receive fitusiran, which is administered as an injection under the skin. The study will observe the frequency of bleeding episodes while participants are on fitusiran treatment. The trial will also compare the bleeding rates during the fitusiran treatment period with those during the standard care period, which may involve other treatments. The study will last for a period of up to 36 months, during which participants will be monitored for any changes in their bleeding patterns and overall health.

In addition to fitusiran, the study will involve other treatments such as coagulation factor VIII, antithrombin III, coagulation factor IX, eptacog alfa (activated), and factor VIII inhibitor bypassing fraction. These treatments are typically used to help manage bleeding in people with hemophilia. The study will help determine the effectiveness and safety of fitusiran in reducing bleeding episodes compared to these other treatments. Participants will be closely monitored for any side effects or adverse events throughout the study.

]]> Hematoonkologia https://clinicaltrials.eu/organisation/hematoonkologia/ Wed, 29 Apr 2026 13:34:18 +0000 https://clinicaltrials.eu/organisation/hematoonkologia/ Fundacja Hematoonkologia is a Polish non-profit organization that serves as a key source of support and information for patients with hematological cancers. The foundation offers accessible materials on diagnosis, treatment methods, symptoms, and living with hematological diseases (e.g., leukemias, lymphomas, multiple myeloma). It also helps families cope with emotional challenges. The foundation works to raise public awareness about hematological cancers.

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