Factor VIII deficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 23 Jun 2026 04:07:50 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Factor VIII deficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Efficacy of efanesoctocog alfa in reducing synovitis in patients with congenital hemophilia A – a multicenter, randomized, open‑label phase 3 trial https://clinicaltrials.eu/trial/efficacy-of-efanesoctocog-alfa-for-reducing-synovitis-in-patients-with-congenital-hemophilia-a/ Wed, 17 Jun 2026 04:03:06 +0000 https://clinicaltrials.eu/trial/efficacy-of-efanesoctocog-alfa-for-reducing-synovitis-in-patients-with-congenital-hemophilia-a/ The study focuses on people born with a blood‑clotting disorder called Congenital hemophilia A, which can cause bleeding into joints and lead to inflammation of the joint lining known as synovitis. The medication being tested is a replacement factor called efanesoctocog alfa, given by intravenous injection to raise the blood’s clotting factor level.

The purpose of the study is to see whether giving a higher amount of this replacement therapy can reduce or eliminate the signs of synovitis over a year. Participants will receive regular infusions of the medicine, will have periodic checks of joint health using ultrasound imaging, and will be followed for about 12 months to record any bleeding events and any side effects.

Throughout the trial, safety will be monitored by recording any adverse events, and the amount of clotting factor in the blood will be measured to understand how long therapeutic levels are maintained. The study will collect information on how often bleeding occurs in joints and the overall number of bleeds, as well as whether the joint inflammation improves.

]]> A Phase III study of NXT007 compared with simoctocog alfa prophylaxis in patients with Hemophilia A without inhibitors https://clinicaltrials.eu/trial/a-phase-iii-study-of-nxt007-compared-with-simoctocog-alfa-prophylaxis-in-patients-with-hemophilia-a-without-inhibitors/ Wed, 17 Jun 2026 04:03:02 +0000 https://clinicaltrials.eu/trial/a-phase-iii-study-of-nxt007-compared-with-simoctocog-alfa-prophylaxis-in-patients-with-hemophilia-a-without-inhibitors/ Hemophilia A is a rare bleeding disorder in which the blood lacks enough of a clotting protein called factor VIII, making it difficult for blood to stop bleeding after an injury. In people without inhibitors (antibodies that block treatment), regular preventive treatment, known as prophylaxis, is used to lower the risk of bleeding episodes. This study compares a new medicine called NXT007, a humanised IgG4 monoclonal antibody that links two clotting proteins (FIXa and FX) and is given by subcutaneous injection (an injection under the skin), with standard prophylaxis using Factor VIII that is administered intravenously (through a vein). Both approaches aim to reduce the number of bleeds.

The purpose of the study is to evaluate whether the new medicine can lower the number of treated bleeds compared with standard therapy. Participants will be assigned to receive either the new medicine or the standard factor VIII for about six months, attending regular visits where any bleeding events are recorded, quality‑of‑life questionnaires are completed, and safety checks such as blood tests for antibodies or reactions at the injection site are performed. The trial will monitor how often injections are needed, the amount of medicine used, and any side effects that arise during the treatment period.

]]> A study looking at the safety of switching from emicizumab to denecimig in adults and adolescents with haemophilia A with or without inhibitors https://clinicaltrials.eu/trial/a-study-looking-at-the-safety-of-switching-from-emicizumab-to-denecimig-in-adults-and-adolescents-with-haemophilia-a-with-or-without-inhibitors/ Mon, 04 May 2026 08:05:50 +0000 https://clinicaltrials.eu/trial/a-study-looking-at-the-safety-of-switching-from-emicizumab-to-denecimig-in-adults-and-adolescents-with-haemophilia-a-with-or-without-inhibitors/ This study involves people with haemophilia A, a condition where blood does not clot properly because the body lacks enough of a specific clotting protein. This condition can occur with or without the presence of inhibitors, which are substances in the blood that can block the clotting protein from working. The study will use a medication called denecimig, also known by its code name NNC0365-3769 or Mim8, which is given as an injection under the skin. Participants will be switching from their current medication called emicizumab to the study medication.

The purpose of this study is to look at how safe it is for people with haemophilia A to switch from emicizumab to Mim8. The study will observe what happens during the time when emicizumab is leaving the body while people are starting treatment with Mim8. This includes watching for any unwanted effects that might occur during this switch.

During the study, participants will receive Mim8 injections under the skin for up to 26 weeks. They will need to attend regular visits and keep track of their experiences using an electronic diary. The study will also ask participants to complete questionnaires about how they find using the injection device and how the treatment affects their daily life. The study involves adults and adolescents aged 12 years and older who have been taking emicizumab for at least 8 weeks and who have decided with their doctor to stop taking emicizumab.

]]> A study of long-term safety and effectiveness of denecimig in people with haemophilia A with or without inhibitors https://clinicaltrials.eu/trial/a-study-of-long-term-safety-and-effectiveness-of-denecimig-in-people-with-haemophilia-a-with-or-without-inhibitors/ Thu, 30 Apr 2026 13:08:43 +0000 https://clinicaltrials.eu/trial/a-study-of-long-term-safety-and-effectiveness-of-denecimig-in-people-with-haemophilia-a-with-or-without-inhibitors/ This study involves haemophilia A, a bleeding disorder where the blood does not clot properly because it lacks enough of a protein called clotting factor VIII. People with this condition may experience prolonged bleeding after injuries or surgery, and sometimes bleeding can occur without any obvious cause, particularly into joints and muscles. Some people with haemophilia A also develop inhibitors, which are proteins made by the body’s immune system that block the clotting factor treatments from working properly. The study uses denecimig, also known as Mim8 or NNC0365-3769, which is given as an injection under the skin. This medication is a type of antibody that helps the blood clot by bringing together two clotting factors in the blood.

The purpose of the study is to learn about the long-term safety of Mim8 when used as a preventive treatment in people with haemophilia A, whether or not they have inhibitors. The study will look at what side effects occur over time and how well the treatment works to prevent bleeding episodes. Participants joining this study are already taking part in or have completed other research studies with Mim8, and this study allows them to continue receiving the treatment for a longer period. Some very young participants who have not yet received much treatment for haemophilia A may also join the study.

During the study, participants will receive regular injections of Mim8 under the skin for up to 262 weeks, which is about five years. Throughout this time, they will attend regular clinic visits where doctors will check their health, take blood samples to measure the level of medication in the blood and look for any antibodies the body might make against the treatment, and ask about any bleeding episodes or side effects. Participants or their caregivers will keep a diary to record any bleeding episodes, treatments used, and any problems with the injection device. The study will also look at how easy the injection device is to use for those participants who use it.

]]> A study on the long-term safety and effectiveness of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B https://clinicaltrials.eu/trial/a-study-on-the-long-term-safety-and-effectiveness-of-giroctocogene-fitelparvovec-or-fidanacogene-elaparvovec-in-patients-with-hemophilia-a-or-hemophilia-b/ Wed, 29 Apr 2026 15:10:01 +0000 https://clinicaltrials.eu/trial/a-study-on-the-long-term-safety-and-effectiveness-of-giroctocogene-fitelparvovec-or-fidanacogene-elaparvovec-in-patients-with-hemophilia-a-or-hemophilia-b/ This study focuses on individuals living with Hemophilia A or Hemophilia B. These are rare bleeding disorders where the blood does not clot properly due to a lack of specific proteins needed for clotting. The purpose of this study is to describe the long-term safety and effectiveness of a single-dose treatment previously received by participants. The medications being observed are fidanacogene elaparvovec, used for those with Hemophilia A, and giroctocogene fitelparvovec, used for those with Hemophilia B.

The treatment involves gene therapy, which is a method used to introduce genetic material into cells to help the body produce the missing clotting proteins. Participants in this study will be monitored over a long period of time to observe how the body responds to these treatments. This includes tracking the level of clotting factor, which is the protein responsible for stopping bleeding, and checking for any potential issues such as thromboembolic events, which are blood clots that can travel through the bloodstream, or changes in liver health.

]]> Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A https://clinicaltrials.eu/trial/study-on-long-term-effects-of-efanesoctocog-alfa-in-preventing-joint-bleeds-in-patients-with-hemophilia-a/ Wed, 29 Apr 2026 15:04:42 +0000 https://clinicaltrials.eu/trial/study-on-long-term-effects-of-efanesoctocog-alfa-in-preventing-joint-bleeds-in-patients-with-hemophilia-a/ This clinical trial focuses on studying the long-term effects of a treatment called efanesoctocog alfa in people with hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly, leading to excessive bleeding. The treatment being tested is a type of medication known as a coagulation factor VIII, which helps the blood to clot and is given as an injection into a vein.

The purpose of this study is to evaluate how well efanesoctocog alfa works in preventing joint bleeds over a long period. Participants in the study will receive regular injections of the medication and will be monitored for any bleeding episodes, particularly in their joints. The study will also look at how the treatment affects joint health over time, including any changes in joint condition and the number of bleeding events.

Participants will be observed for several years to gather information on the effectiveness of the treatment. The study will track the number of bleeding episodes, the amount of medication needed to control bleeding, and any changes in joint health. This information will help to understand the benefits of using efanesoctocog alfa as a regular preventive treatment for people with hemophilia A.

]]> Study on Emicizumab for Patients with Mild or Moderate Hemophilia A Without FVIII Inhibitors https://clinicaltrials.eu/trial/study-on-emicizumab-for-patients-with-mild-or-moderate-hemophilia-a-without-fviii-inhibitors/ Wed, 29 Apr 2026 15:01:16 +0000 https://clinicaltrials.eu/trial/study-on-emicizumab-for-patients-with-mild-or-moderate-hemophilia-a-without-fviii-inhibitors/ This clinical trial is focused on studying the effects of a medication called Hemlibra, which contains the active substance emicizumab. The study is specifically for patients with hemophilia A, a condition where the blood does not clot properly, leading to excessive bleeding. This trial is for those with mild or moderate forms of hemophilia A who do not have inhibitors against factor VIII, a protein that helps blood clot.

The purpose of the study is to evaluate the safety and effectiveness of emicizumab in preventing bleeding episodes in these patients. Participants will receive emicizumab as a solution for injection under the skin. The study will monitor the number of bleeding episodes over time and assess any side effects or reactions to the medication. The trial will also look at how the medication affects joint health, quality of life, and physical activity.

Throughout the study, participants will be regularly assessed to ensure their safety and to gather information on how emicizumab is working in their bodies. The trial will also explore the presence of any antibodies that might develop against the medication. This research aims to provide valuable insights into the management of hemophilia A and improve treatment options for those affected by this condition.

]]> Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors https://clinicaltrials.eu/trial/study-on-emicizumab-and-drug-combination-for-patients-with-haemophilia-a-and-fviii-inhibitors/ Wed, 29 Apr 2026 15:00:10 +0000 https://clinicaltrials.eu/trial/study-on-emicizumab-and-drug-combination-for-patients-with-haemophilia-a-and-fviii-inhibitors/ This clinical trial focuses on studying different treatment approaches for patients with Hemophilia A, a condition where blood does not clot properly due to a lack of a specific protein called factor VIII. The study involves several treatments, including Hemlibra (also known as emicizumab), Nuwiq (simoctocog alfa), FEIBA (factor VIII inhibitor bypassing fraction), Wilate (human coagulation factor VIII and human von Willebrand factor), OCTANATE (human coagulation factor VIII), and NovoSeven (eptacog alfa, activated). These treatments are administered either through injections or infusions.

The purpose of the study is to evaluate the effectiveness of these treatments in patients with Hemophilia A who have developed inhibitors, which are antibodies that reduce the effectiveness of factor VIII treatments. The study is divided into three groups. Groups 1 and 2 will focus on evaluating the success of immune tolerance induction (ITI), a process aimed at reducing inhibitor levels, while Group 3 will assess the annualized bleeding rate (ABR) compared to the other groups. Participants will receive their assigned treatments over a period of up to 60 weeks, with regular monitoring to assess their response to the treatment.

Throughout the study, participants will be monitored for the frequency and severity of bleeding episodes, the number of infusions needed to control bleeding, and any side effects or adverse reactions. The study aims to provide valuable insights into the best treatment strategies for managing Hemophilia A in patients with inhibitors, ultimately improving their quality of life and treatment outcomes.

]]> Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis https://clinicaltrials.eu/trial/study-on-synovial-hypertrophy-in-patients-with-hemophilia-a-using-efanesoctocog-alfa-prophylaxis/ Wed, 29 Apr 2026 14:57:41 +0000 https://clinicaltrials.eu/trial/study-on-synovial-hypertrophy-in-patients-with-hemophilia-a-using-efanesoctocog-alfa-prophylaxis/ This clinical trial is focused on studying Haemophilia A, a condition where blood does not clot properly, leading to excessive bleeding. The study will use a treatment called efanesoctocog alfa, also known by its code name BIVV001. This treatment is a type of protein that helps blood to clot and is given as an injection into a vein. The purpose of the study is to observe changes in joint health in patients with Haemophilia A who are receiving regular doses of efanesoctocog alfa.

Participants in the study will receive the treatment over a period of 12 months. During this time, doctors will use imaging techniques like ultrasound and MRI (Magnetic Resonance Imaging) to monitor the condition of the joints. These imaging methods help in detecting changes in the joint tissues, specifically looking for improvements in a condition called synovial hypertrophy, which is a thickening of the joint lining that can occur in people with Haemophilia A.

The study aims to see if the treatment can reduce this thickening and improve joint health over time. Participants will be monitored for any changes in their joint condition and overall health. The study will also collect information on how the treatment affects the frequency of bleeding episodes and the participants’ quality of life. This information will help in understanding the effectiveness and safety of efanesoctocog alfa for people living with Haemophilia A.

]]> Study on How Concizumab Works for Patients with Hemophilia A or B Without Inhibitors https://clinicaltrials.eu/trial/study-on-how-concizumab-works-for-patients-with-hemophilia-a-or-b-without-inhibitors/ Wed, 29 Apr 2026 14:57:10 +0000 https://clinicaltrials.eu/trial/study-on-how-concizumab-works-for-patients-with-hemophilia-a-or-b-without-inhibitors/ This clinical trial is focused on studying the effects of a medication called Concizumab in people with a bleeding disorder known as hemophilia. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. The study specifically looks at two types of hemophilia: hemophilia A and hemophilia B, both without inhibitors. Inhibitors are substances that can interfere with the treatment of hemophilia, so this study focuses on patients who do not have these inhibitors.

The purpose of the study is to see how well Concizumab works in reducing the number of bleeding episodes in patients with hemophilia A and B. The medication is given as a solution for injection using a device called the PDS290 pen-injector. Participants in the study will receive either the medication or a placebo, which is a substance with no active drug. The study will compare the effects of taking Concizumab regularly (prophylaxis) to not taking it regularly (on-demand treatment) to see which approach is more effective in preventing bleeding episodes.

Throughout the study, participants will receive injections of Concizumab and will be monitored for any changes in their condition, including the number of bleeding episodes they experience. The study will last for several months, allowing researchers to gather enough information to determine the effectiveness and safety of the medication. This research aims to provide better treatment options for people living with hemophilia, helping them manage their condition more effectively.

]]> Safety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A https://clinicaltrials.eu/trial/safety-study-of-damoctocog-alfa-pegol-for-children-aged-7-to-11-with-severe-hemophilia-a/ Wed, 29 Apr 2026 14:47:51 +0000 https://clinicaltrials.eu/trial/safety-study-of-damoctocog-alfa-pegol-for-children-aged-7-to-11-with-severe-hemophilia-a-2/ This clinical trial is focused on studying severe hemophilia A, a condition where the blood does not clot properly due to a lack of a protein called Factor VIII. The study involves a treatment known as BAY 94-9027, which is a form of Factor VIII designed to help manage bleeding episodes in children who have been previously treated for this condition. The treatment is administered as a solution for injection, and it is intended to be used both for regular prevention of bleeding and for treating bleeding episodes when they occur.

The purpose of the study is to evaluate the safety of BAY 94-9027 in children aged 7 to under 12 years with severe hemophilia A. Participants will receive the treatment over a period of time, and the study will monitor for any side effects or reactions to the medication. The study will also track how often bleeding occurs and how much of the medication is used. This information will help determine how well the treatment works and how safe it is for children in this age group.

Throughout the study, participants will be asked to document their experiences using an electronic diary. This will include recording any infusions of the medication and any bleeding episodes. The study aims to provide valuable information on the use of BAY 94-9027 in managing severe hemophilia A in children, contributing to better treatment options for this condition.

]]> Continued Access Study for Patients with Severe Hemophilia A Using Efanesoctocog Alfa https://clinicaltrials.eu/trial/continued-access-study-for-patients-with-severe-hemophilia-a-using-efanesoctocog-alfa/ Wed, 29 Apr 2026 14:30:15 +0000 https://clinicaltrials.eu/trial/continued-access-study-for-patients-with-severe-hemophilia-a-using-efanesoctocog-alfa/ This clinical trial is focused on studying a condition known as Hemophilia A, which is a genetic disorder that affects the blood’s ability to clot properly. The treatment being investigated in this study is called efanesoctocog alfa, also known by its code name BIVV001. This medication is designed to help manage bleeding episodes in patients with severe Hemophilia A by providing a replacement for a missing protein that is crucial for blood clotting.

The purpose of this study is to gather more information about the safety and effectiveness of efanesoctocog alfa in patients who have already participated in a previous trial with this treatment. Participants will receive the medication through an intravenous injection, which means it is administered directly into a vein. The study will monitor how well the treatment works in preventing bleeding episodes and how well it is tolerated by the patients over a period of time.

Throughout the study, participants will continue to receive efanesoctocog alfa as a prophylactic treatment, which means it is given regularly to prevent bleeding rather than treating it after it occurs. The study aims to ensure that patients have continued access to this treatment and to collect further data on its long-term use. Participants will be closely monitored for any side effects or adverse events to ensure their safety while receiving the treatment.

]]> Study on the Safety and Effectiveness of Giroctocogene Fitelparvovec for Adult Men with Moderate to Severe Hemophilia A https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-giroctocogene-fitelparvovec-for-adult-men-with-moderate-to-severe-hemophilia-a/ Wed, 29 Apr 2026 14:28:26 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-giroctocogene-fitelparvovec-for-adult-men-with-moderate-to-severe-hemophilia-a/ This clinical trial is focused on studying hemophilia A, a genetic disorder that affects the blood’s ability to clot properly, leading to excessive bleeding. The treatment being tested is called giroctocogene fitelparvovec, also known by its code name PF-07055480. This treatment is a type of gene therapy, which involves using a specially designed virus to deliver a healthy copy of the gene responsible for producing Factor VIII, a protein that helps blood clot. The goal of the study is to evaluate how effective and safe this gene therapy is for adult males with moderately severe to severe hemophilia A.

Participants in the study will receive a single infusion of the gene therapy. After the infusion, they will be monitored over a period of time to see how their condition changes. The study will look at various factors, such as the rate of bleeding episodes and the level of Factor VIII in the blood, to determine the treatment’s effectiveness. The study will also assess any side effects or adverse reactions to the treatment.

The trial is designed to provide valuable information about the potential benefits and risks of using gene therapy to treat hemophilia A. By participating in this study, researchers hope to find a new way to manage this condition and improve the quality of life for those affected by it. The study will continue for several years to gather comprehensive data on the long-term effects of the treatment.

]]> Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A https://clinicaltrials.eu/trial/long-term-safety-study-of-valoctocogene-roxaparvovec-for-patients-with-severe-hemophilia-a/ Wed, 29 Apr 2026 14:28:17 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-valoctocogene-roxaparvovec-for-patients-with-severe-hemophilia-a/ This clinical trial focuses on individuals with Hemophilia A, a genetic disorder that affects the blood’s ability to clot properly, leading to excessive bleeding. The study involves a treatment called BMN 270, also known as valoctocogene roxaparvovec, which is a gene therapy designed to help the body produce more of a protein called Factor VIII, essential for blood clotting. This treatment is administered as a solution for infusion, meaning it is given directly into the bloodstream through a vein.

The purpose of this study is to evaluate the long-term safety of BMN 270 in participants who have previously received this treatment in earlier clinical trials. Participants will be monitored over an extended period to observe any potential side effects or health changes. The study will look for specific events such as liver problems, blood clots, the development of inhibitors to Factor VIII, and any transmission of the treatment to others. Additionally, the study will assess changes in bleeding rates, Factor VIII activity levels, and the use of other medications for managing Hemophilia A.

Participants will also provide feedback on their overall health and quality of life throughout the study. This feedback will help researchers understand the impact of BMN 270 on daily living and any changes in the severity of symptoms. The study aims to gather comprehensive data to ensure the treatment’s safety and effectiveness for individuals with Hemophilia A over the long term.

]]> Study on Personalized Dosing of Emicizumab for Patients with Congenital Hemophilia A https://clinicaltrials.eu/trial/study-on-personalized-dosing-of-emicizumab-for-patients-with-congenital-hemophilia-a/ Wed, 29 Apr 2026 14:27:53 +0000 https://clinicaltrials.eu/trial/study-on-personalized-dosing-of-emicizumab-for-patients-with-congenital-hemophilia-a/ This clinical trial is focused on studying a condition known as congenital hemophilia A, a genetic disorder that affects the blood’s ability to clot properly, leading to excessive bleeding. The treatment being investigated is called emicizumab, which is a type of medication known as a humanized monoclonal antibody. Emicizumab is administered as a solution for injection under the skin, known as a subcutaneous injection. The study aims to determine if a personalized dosing approach, guided by the levels of the drug in the blood, is as effective as the standard dosing method in preventing bleeding episodes in patients with congenital hemophilia A.

Participants in the study will receive emicizumab, which is also known by its code names RO5534262 and ACE910. The study will compare two dosing strategies: the conventional dosing and a new approach that adjusts the dose based on how the drug is processed in the body, known as pharmacokinetic-guided dosing. The goal is to see if the personalized dosing can prevent treated bleeds, spontaneous joint or muscle bleeds, and overall bleeding as effectively as the conventional method.

The study will take place over several months, during which participants will be monitored for bleeding events and other health indicators. The trial will also assess the quality of life, joint health, and any pain experienced during the administration of emicizumab. By the end of the study, researchers hope to gather valuable information on the effectiveness and potential benefits of personalized dosing for individuals with congenital hemophilia A.

]]> Study on the Safety and Effects of NXT007 for Patients with Severe or Moderate Hemophilia A https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-nxt007-for-patients-with-severe-or-moderate-hemophilia-a/ Wed, 29 Apr 2026 14:26:56 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-nxt007-for-patients-with-severe-or-moderate-hemophilia-a/ This clinical trial is focused on studying a condition known as Hemophilia A, which is a bleeding disorder where blood doesn’t clot properly. The study is specifically looking at people with severe or moderate forms of this condition, with or without inhibitors against a protein called factor VIII. The main purpose of the study is to evaluate the safety of a new treatment called NXT007. This treatment is a type of medication known as a humanized monoclonal modified IgG4 antibody, which is designed to help manage bleeding in people with Hemophilia A.

Participants in the study will receive NXT007 through a subcutaneous injection, which means it is injected under the skin. The study will also involve other treatments, including Coagulation Factor IX, II, VII, and X in Combination, Coagulation Factor VIII, and Eptacog Alfa (Activated), which are given through intravenous injection, meaning they are injected directly into a vein. Another treatment being studied is Damoctocog Alfa Pegol, also known by its code name BAY 94-9027, which is a modified version of factor VIII.

The study will monitor participants over time to assess the safety and effects of NXT007. This includes checking for any side effects, changes in laboratory test results, and vital signs. The study will also measure how the body processes the medication and whether participants develop any antibodies against the drug. Additionally, the study will track the number of bleeding events and the annualized bleeding rate, which is the average number of bleeding episodes per year. The goal is to gather information that could help improve treatment options for people with Hemophilia A.

]]> Study on the Safety and Effects of BAY 2599023 (Peboctocogene Camaparvovec) for Adults with Severe Hemophilia A https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-bay-2599023-peboctocogene-camaparvovec-for-adults-with-severe-hemophilia-a/ Wed, 29 Apr 2026 14:25:39 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-bay-2599023-peboctocogene-camaparvovec-for-adults-with-severe-hemophilia-a/ This clinical trial is focused on studying a condition known as Hemophilia A, which is a genetic disorder that affects the blood’s ability to clot properly. The study is testing a new treatment called BAY 2599023 (also known as DTX201). This treatment uses a modified, non-infectious virus to deliver a healthy version of the defective Factor VIII gene into the liver cells. The goal is to help the body produce the necessary protein for blood clotting.

The purpose of the study is to investigate the safety and how well patients with severe Hemophilia A respond to this new treatment. Participants will receive a single dose of the treatment through an intravenous (IV) infusion. The study will monitor the participants over time to see how their bodies react to the treatment and to check for any side effects. The study will also measure how much of the Factor VIII protein is produced in the body after receiving the treatment.

Throughout the study, researchers will keep track of any adverse events, which are any unwanted effects that might occur. They will also look at how well the treatment works by measuring the levels of Factor VIII in the blood at different times. The study aims to find out if the treatment can help increase the levels of Factor VIII to a point where it can effectively help with blood clotting in patients with severe Hemophilia A.

]]> Study on Long-Term Safety and Effects of Marstacimab for Patients with Severe Hemophilia A or B, With or Without Inhibitors https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-marstacimab-for-patients-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ Wed, 29 Apr 2026 14:25:12 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-marstacimab-for-patients-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ This clinical trial is focused on studying the long-term safety and effectiveness of a treatment called Marstacimab for people with a condition known as hemophilia. Hemophilia is a rare bleeding disorder where blood doesn’t clot properly, leading to excessive bleeding. The study includes individuals with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors. Inhibitors are substances that can interfere with the treatment of hemophilia, making it more challenging to manage the condition.

The purpose of this study is to evaluate how safe and tolerable Marstacimab is when used over a long period. Participants will receive the treatment as a solution for injection, either in a pre-filled pen or syringe, which is administered under the skin. The study will monitor participants for any side effects, including reactions at the injection site, changes in vital signs, and any significant changes in laboratory test results. The study will also track the number of bleeding episodes and any changes in joint health over time.

Participants in this study will be observed for several years to gather comprehensive data on the treatment’s impact. The study aims to provide valuable insights into the long-term management of hemophilia using Marstacimab, helping to improve the quality of life for those affected by this condition. The study is open-label, meaning both the researchers and participants know what treatment is being administered, and it is an extension of previous studies, allowing for continued observation and data collection.

]]> Study on Physical Activity and Joint Health in Patients Aged 12 and Older with Severe Hemophilia A Treated with Efanesoctocog Alfa for 24 Months https://clinicaltrials.eu/trial/study-on-physical-activity-and-joint-health-in-patients-aged-12-and-older-with-severe-hemophilia-a-treated-with-efanesoctocog-alfa-for-24-months/ Wed, 29 Apr 2026 14:24:46 +0000 https://clinicaltrials.eu/trial/study-on-physical-activity-and-joint-health-in-patients-aged-12-and-older-with-severe-hemophilia-a-treated-with-efanesoctocog-alfa-for-24-months/ This clinical trial is focused on studying severe hemophilia A, a condition where the blood does not clot properly, leading to excessive bleeding. The treatment being tested is called efanesoctocog alfa, also known by its code name BIVV001. This medication is a type of protein that helps the blood to clot and is given as a powder mixed into a solution for injection into a vein.

The purpose of the study is to observe changes in physical activity and joint health in patients aged 12 and older who have been previously treated for severe hemophilia A. Participants will receive the treatment once a week for a period of 24 months. During the study, participants will use an activity tracker to monitor their physical activity and heart rate. They will also report their physical activities and any changes in their joint health.

The study will last for 24 months, during which participants will be monitored for any changes in their condition. The study aims to understand how the treatment affects physical activity levels and joint health over time. Participants will be asked to use a smartphone or tablet to record their activities and will have regular check-ups to assess their progress. The study will also look at the number of bleeding episodes and any side effects that may occur during the treatment period.

]]> Study on Marstacimab for Children with Severe Hemophilia A or B, With or Without Inhibitors https://clinicaltrials.eu/trial/study-on-marstacimab-for-children-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ Wed, 29 Apr 2026 14:24:33 +0000 https://clinicaltrials.eu/trial/study-on-marstacimab-for-children-with-severe-hemophilia-a-or-b-with-or-without-inhibitors/ This clinical trial is focused on studying the effectiveness and safety of a treatment called Marstacimab for children and teenagers with a condition known as Hemophilia. Hemophilia is a rare bleeding disorder where the blood doesn’t clot properly, leading to excessive bleeding. The study includes participants with two types of hemophilia: Hemophilia A, which involves a deficiency in a protein called factor VIII, and Hemophilia B, which involves a deficiency in factor IX. The trial is open to participants under 18 years of age, with severe forms of these conditions, whether or not they have developed inhibitors, which are antibodies that can interfere with treatment.

The purpose of the study is to compare the new treatment, Marstacimab, with the standard treatment that participants have been receiving over the past year. Marstacimab is administered as a solution for injection using a pre-filled pen, which is a device that allows the medication to be injected under the skin. The study will last for about 48 weeks, during which participants will receive regular doses of Marstacimab. The trial aims to see if Marstacimab can reduce the number of bleeding episodes and improve the overall health of the joints, which are often affected by bleeding in people with hemophilia.

Throughout the study, the safety of Marstacimab will be closely monitored, including any side effects or reactions at the injection site. The trial will also assess the impact of the treatment on the participants’ quality of life, using questionnaires designed for different age groups. This research is important for understanding how well Marstacimab works in young people with hemophilia and whether it can become a new option for managing this condition.

]]> Study on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa https://clinicaltrials.eu/trial/study-on-joint-health-in-hemophilia-a-and-b-patients-using-efmoroctocog-alfa-or-eftrenonacog-alfa/ Wed, 29 Apr 2026 14:24:09 +0000 https://clinicaltrials.eu/trial/study-on-joint-health-in-hemophilia-a-and-b-patients-using-efmoroctocog-alfa-or-eftrenonacog-alfa/ This clinical trial is focused on studying the effects of two treatments for patients with Hemophilia A and Hemophilia B. These are genetic disorders where blood does not clot properly, leading to excessive bleeding. The treatments being studied are called efmoroctocog alfa and eftrenonacog alfa. Efmoroctocog alfa is a recombinant fusion protein that acts like a human coagulation factor VIII, while eftrenonacog alfa is a similar protein that acts like factor IX. These treatments are given as injections and are used to prevent bleeding episodes in patients with hemophilia.

The purpose of this study is to assess the health of joints in patients with hemophilia who are receiving these treatments over an 18-month period. The study will use ultrasound, a type of imaging that uses sound waves to create pictures of the inside of the body, to evaluate joint health. Participants will receive either efmoroctocog alfa or eftrenonacog alfa as part of their regular treatment plan. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatments.

Throughout the study, participants will have regular check-ups to monitor their joint health and overall well-being. The study aims to understand how these treatments affect joint health and to gather information that could help improve care for people with hemophilia. The study will last for 18 months, during which participants will continue their usual care while being monitored for any changes in their condition.

]]> Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-serpinpc-for-patients-with-severe-hemophilia-a-or-b/ Wed, 29 Apr 2026 14:23:49 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-serpinpc-for-patients-with-severe-hemophilia-a-or-b/ This clinical trial is focused on studying the effects of a treatment called SerpinPC for individuals with Severe Hemophilia A or Moderately Severe to Severe Hemophilia B. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. The treatment being tested, SerpinPC, is a solution for injection that contains a modified form of a protein called human alpha-1 proteinase inhibitor. This study aims to evaluate how effective and safe SerpinPC is when given as a regular preventive treatment through subcutaneous injection, which means it is injected under the skin.

The purpose of the study is to assess the efficacy and safety of SerpinPC in people with these types of hemophilia. Participants in the study will receive the treatment over a period of time, and their health will be monitored to see how well the treatment works in reducing bleeding episodes. The study will also look at the overall health and quality of life of the participants while they are receiving the treatment.

Throughout the study, participants will be observed for any changes in their condition, and the number of bleeding episodes they experience will be recorded. The study will help determine if SerpinPC can be a beneficial treatment option for those living with Severe Hemophilia A or Moderately Severe to Severe Hemophilia B. The trial is designed to gather important information that could lead to improved care and management of these conditions in the future.

]]> Study on the Use of Simoctocog Alfa and Emicizumab for Surgery in Patients with Severe Hemophilia A https://clinicaltrials.eu/trial/study-on-the-use-of-simoctocog-alfa-and-emicizumab-for-surgery-in-patients-with-severe-hemophilia-a/ Wed, 29 Apr 2026 14:23:34 +0000 https://clinicaltrials.eu/trial/study-on-the-use-of-simoctocog-alfa-and-emicizumab-for-surgery-in-patients-with-severe-hemophilia-a/ This clinical trial is focused on studying the treatment of Hemophilia A, a genetic disorder that affects the blood’s ability to clot properly. The study involves the use of a medication called Nuwiq, which is a form of Coagulation Factor VIII. This medication is administered as a solution for injection and is used in combination with another medication called Emicizumab, which is already being used regularly by the patients participating in the study.

The purpose of the study is to evaluate how effective Nuwiq is in managing bleeding during and after major surgery in patients with severe Hemophilia A who are already on Emicizumab prophylaxis. Participants will receive Nuwiq through an intravenous injection, which means it is delivered directly into a vein. The study will monitor the patients’ response to the treatment over a period of time, focusing on how well the medication helps control bleeding during surgery.

Throughout the study, researchers will assess the overall success of the treatment by observing the patients’ bleeding control during surgery and any side effects that may occur. The study will also track the levels of Coagulation Factor VIII in the blood before and after the Nuwiq injections. The trial is expected to continue until March 2025, providing valuable insights into the effectiveness of this treatment approach for individuals with severe Hemophilia A.

]]> Study on the Use of Simoctocog Alfa for Women and Girls with Hemophilia A Undergoing Surgery https://clinicaltrials.eu/trial/study-on-the-use-of-simoctocog-alfa-for-women-and-girls-with-hemophilia-a-undergoing-surgery/ Wed, 29 Apr 2026 14:23:30 +0000 https://clinicaltrials.eu/trial/study-on-the-use-of-simoctocog-alfa-for-women-and-girls-with-hemophilia-a-undergoing-surgery/ This clinical trial is focused on studying the treatment of hemophilia A, a genetic disorder that affects the blood’s ability to clot properly, in women and girls who need surgery. The treatment being tested is called Nuwiq, which is a medication used to help the blood clot by providing a protein known as coagulation factor VIII. This study aims to evaluate how effective Nuwiq is in managing bleeding during and after surgery in patients with hemophilia A.

Participants in the study will receive Nuwiq through an intravenous injection, which means it will be administered directly into a vein. The study will observe the effects of Nuwiq during the surgery and the recovery period afterward. The treatment period for each participant is expected to last up to 30 days. The study will also monitor for any side effects or complications that may arise from the treatment.

The goal of this study is to gather information on the overall effectiveness of Nuwiq in controlling bleeding during surgical procedures in women and girls with hemophilia A. This information will help healthcare providers better understand how to manage bleeding in these patients during surgery. The study is open-label, meaning both the participants and the researchers know which treatment is being administered, and it is not controlled, so all participants will receive Nuwiq without a comparison to a placebo or another treatment.

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