Factor IX deficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 23 Jun 2026 04:04:25 +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 IX deficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 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 of Etranacogene Dezaparvovec (CSL222) in Adolescent Males with Severe or Moderately Severe Hemophilia B https://clinicaltrials.eu/trial/study-of-etranacogene-dezaparvovec-csl222-in-adolescent-males-with-severe-or-moderately-severe-hemophilia-b/ Wed, 29 Apr 2026 15:06:07 +0000 https://clinicaltrials.eu/trial/study-of-etranacogene-dezaparvovec-csl222-in-adolescent-males-with-severe-or-moderately-severe-hemophilia-b/ Hemophilia B is a rare bleeding disorder in which the blood does not clot properly due to a deficiency in a protein called Factor IX. This study aims to investigate the effectiveness, safety, and tolerability of CSL222 (also known as etranacogene dezaparvovec) in adolescent males between 12 and 18 years of age who have severe or moderately severe hemophilia B. Hemophilia B is characterized by prolonged bleeding after injuries, surgeries, or even spontaneous bleeding into joints and muscles.

The treatment being studied, CSL222, is a gene therapy designed to help the body produce its own Factor IX protein, which is essential for normal blood clotting. This therapy is administered as a single dose, potentially reducing the need for regular Factor IX replacement treatments that patients typically need. Before receiving the study treatment, participants will complete a lead-in period of at least 6 months where their current treatment and bleeding episodes are monitored.

During the study, researchers will track several outcomes including how often participants experience bleeding episodes, the amount of Factor IX protein in their blood, whether they still need regular Factor IX treatments, and any side effects that might occur. The study will also measure how the treatment affects participants’ quality of life using various assessment tools.

]]> Study on the Safety and Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Adults with Severe or Moderately Severe Hemophilia B and AAV5 Antibodies https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-etranacogene-dezaparvovec-gene-therapy-for-adults-with-severe-or-moderately-severe-hemophilia-b-and-aav5-antibodies/ Wed, 29 Apr 2026 15:03:58 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-etranacogene-dezaparvovec-gene-therapy-for-adults-with-severe-or-moderately-severe-hemophilia-b-and-aav5-antibodies/ This clinical trial is focused on studying a condition called Hemophilia B, which is a genetic disorder that affects the blood’s ability to clot properly. People with this condition often experience excessive bleeding because they have low levels of a protein called Factor IX, which is essential for blood clotting. The study is testing a new treatment called CSL222 (Etranacogene Dezaparvovec), which is a type of gene therapy. This treatment uses a harmless virus to deliver a healthy version of the Factor IX gene to the liver, where it can help produce the necessary protein to improve blood clotting.

The purpose of the study is to evaluate the effectiveness and safety of this gene therapy in adults with severe or moderately severe Hemophilia B who have specific antibodies, known as AAV5 neutralizing antibodies, before treatment. Participants will receive a single dose of the gene therapy through an intravenous infusion, which means it will be delivered directly into the bloodstream. The study will monitor participants over a period of time to see how well the treatment works in reducing bleeding episodes compared to their previous standard care, which involved regular Factor IX replacement therapy.

Throughout the study, researchers will keep track of various health indicators, including the number of bleeding episodes, any side effects, and changes in liver function. The study aims to provide valuable information on whether this gene therapy can offer a more effective and convenient treatment option for people living with Hemophilia B. Participants will be closely monitored by healthcare professionals to ensure their safety and well-being during the trial.

]]> Title: Study of gene therapy Etranacogene Dezaparvovec (Hemgenix) in adult patients with moderate to severe Hemophilia B https://clinicaltrials.eu/trial/study-on-gene-therapy-etranacogene-dezaparvovec-for-adults-with-severe-or-moderately-severe-hemophilia-b/ Wed, 29 Apr 2026 15:01:04 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-etranacogene-dezaparvovec-for-adults-with-severe-or-moderately-severe-hemophilia-b/ This clinical trial studies a gene therapy treatment for people with Hemophilia B, a rare inherited blood clotting disorder. The study uses etranacogene dezaparvovec (also known as Hemgenix), which is given as a single intravenous infusion. This medication contains a modified virus that carries a working copy of the blood clotting factor gene that is defective in people with Hemophilia B.

The purpose of this study is to determine if this one-time gene therapy treatment works as well as the standard treatment of regular preventive blood clotting factor injections. The treatment involves receiving a single dose of the gene therapy through a vein, and then patients are monitored for 18 months to see how well it works.

The gene therapy works by using a harmless virus called adeno-associated virus to deliver a working copy of the Factor IX gene to the liver. This gene helps the body produce the blood clotting protein that is missing in people with Hemophilia B. The study will measure how well this treatment prevents bleeding episodes compared to regular preventive treatment with clotting factor injections.

]]> 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.

]]> Long-term Safety Study of Etranacogene Dezaparvovec in Adult Men with Hemophilia B https://clinicaltrials.eu/trial/long-term-safety-study-of-etranacogene-dezaparvovec-in-adult-men-with-hemophilia-b/ Wed, 29 Apr 2026 14:48:02 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-etranacogene-dezaparvovec-in-adult-men-with-hemophilia-b/ This clinical trial is focused on studying the long-term safety and effectiveness of a treatment for Hemophilia B, a genetic disorder that affects the blood’s ability to clot properly. The treatment being studied is called Etranacogene Dezaparvovec, also known by its code name CSL222. This treatment is a type of gene therapy, which involves using a specially designed virus to deliver a healthy version of a gene to the liver, where it can help produce a protein called Factor IX that is essential for blood clotting.

The purpose of this study is to monitor adult male participants who have previously received the CSL222 treatment in earlier studies. Participants will be followed over a long period to observe any potential side effects and to assess how well the treatment continues to work in reducing bleeding episodes. The study will also look at the participants’ quality of life and their need for additional treatments to manage their condition.

Throughout the study, participants will have regular check-ups to track their health and any changes in their condition. The study aims to provide valuable information on the long-term benefits and safety of Etranacogene Dezaparvovec for individuals with Hemophilia B, helping to improve future treatment options for this condition.

]]> Study on the Safety and Effectiveness of REGV131 and LNP1265 for Adults with Hemophilia B https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-regv131-and-lnp1265-for-adults-with-hemophilia-b/ Wed, 29 Apr 2026 14:30:02 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-regv131-and-lnp1265-for-adults-with-hemophilia-b/ This clinical trial is focused on studying a treatment for Hemophilia B, a genetic disorder that affects the blood’s ability to clot properly. The study will use a treatment called REGV131-LNP1265, which is a new type of therapy involving a method known as CRISPR/Cas9. This method is designed to help the body produce a protein called clotting factor IX, which is essential for blood clotting. The treatment is given as a solution through an intravenous (IV) infusion, meaning it is delivered directly into the bloodstream.

The purpose of the study is to evaluate the safety and effectiveness of REGV131-LNP1265 in helping the body make clotting factor IX in adults with Hemophilia B. The study is divided into two parts. In the first part, participants will receive a single dose of the treatment to assess its safety and how well it helps the body produce clotting factor IX. In the second part, participants will receive the recommended dose to further evaluate its effectiveness in reducing bleeding events over time.

Participants in the study will be monitored for any side effects and changes in their ability to produce clotting factor IX. The study will also track the rate of bleeding events and the need for additional clotting factor treatments. The trial is expected to last several years, allowing researchers to gather comprehensive data on the long-term effects and benefits of the treatment. This study aims to provide valuable insights into a potential new therapy for managing Hemophilia B.

]]> Study on Long-Term Safety and Efficacy of Etranacogene Dezaparvovec for Adults with Severe or Moderately Severe Hemophilia B https://clinicaltrials.eu/trial/study-on-long-term-safety-and-efficacy-of-etranacogene-dezaparvovec-for-adults-with-severe-or-moderately-severe-hemophilia-b/ Wed, 29 Apr 2026 14:28:27 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-efficacy-of-etranacogene-dezaparvovec-for-adults-with-severe-or-moderately-severe-hemophilia-b/ This clinical trial is focused on studying the long-term safety and effectiveness of a gene therapy treatment for adults with Hemophilia B, a genetic disorder that affects the blood’s ability to clot properly. The treatment being studied is called AAV5-hFIX, which is a type of gene therapy. It uses a harmless virus to deliver a modified version of the human factor IX gene, which is important for blood clotting, into the patient’s liver cells. This therapy aims to help the body produce more of the factor IX protein, potentially reducing bleeding episodes in patients with severe or moderately severe Hemophilia B.

The purpose of this study is to assess the long-term safety of the AAV5-hFIX treatment over a period of 6 to 10 years after it has been administered. Participants in this study have previously received this gene therapy in an earlier phase of the trial. Throughout the study, researchers will monitor participants for any side effects that may be related to the treatment, as well as changes in liver function and the presence of any antibodies that might affect the treatment’s effectiveness. Additionally, the study will evaluate how well the treatment works in terms of increasing factor IX activity, reducing the need for additional factor IX treatments, and decreasing the frequency of bleeding events.

Participants will undergo regular check-ups and assessments to track their health and the impact of the gene therapy on their quality of life. This includes monitoring for any bleeding episodes, evaluating joint health, and assessing overall well-being through quality of life questionnaires. The study aims to provide valuable information on the long-term benefits and safety of AAV5-hFIX for individuals living with Hemophilia B.

]]> 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 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 Efficacy and Safety of Fidanacogene Elaparvovec Gene Therapy in Adult Males with Moderate to Severe Hemophilia B https://clinicaltrials.eu/trial/study-on-the-efficacy-and-safety-of-fidanacogene-elaparvovec-gene-therapy-in-adult-males-with-moderate-to-severe-hemophilia-b/ Wed, 29 Apr 2026 14:24:08 +0000 https://clinicaltrials.eu/trial/study-on-the-efficacy-and-safety-of-fidanacogene-elaparvovec-gene-therapy-in-adult-males-with-moderate-to-severe-hemophilia-b/ This clinical trial is focused on studying a condition known as hemophilia B, which is a genetic disorder that affects the blood’s ability to clot properly. People with this condition can experience excessive bleeding even from minor injuries. The study is specifically looking at adult males with moderately severe to severe forms of this condition. The treatment being tested is a gene therapy called fidanacogene elaparvovec, also known by its code name PF-06838435. This therapy involves a single infusion of a solution that contains a modified gene designed to help the body produce more of a protein called Factor IX, which is essential for blood clotting.

The purpose of the study is to evaluate how effective and safe this gene therapy is for individuals with hemophilia B. Participants in the study will receive a single infusion of the gene therapy and will be monitored over a period of time to see how their condition changes. The study will look at various factors, such as the number of bleeding episodes participants experience and their overall health, to determine the therapy’s effectiveness. Participants will also be observed for any side effects or adverse reactions to the treatment.

The study is designed to provide valuable information about the potential benefits and risks of using fidanacogene elaparvovec as a treatment for hemophilia B. By participating in this research, scientists hope to find a new way to help people with this condition manage their symptoms and improve their quality of life. The study will continue for several years to gather comprehensive data on the long-term effects of the therapy.

]]>