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Factor VIII deficiency – Trials in Disease

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Ongoing Clinical Trials for Factor VIII Deficiency

Factor VIII deficiency, also known as Hemophilia A, is a genetic bleeding disorder where blood does not clot properly. Currently, 19 clinical trials are ongoing across Europe and beyond, testing new treatments including gene therapies, long-acting clotting factors, and prophylactic medications aimed at reducing bleeding episodes and improving quality of life for patients. (Also known as: Hemophilia A, Haemophilia A)

Clinical trial locations

Continued Access Study for Patients with Severe Hemophilia A Using Efanesoctocog Alfa

This study provides continued access to efanesoctocog alfa for patients with severe Hemophilia A who have already participated in previous trials and are benefiting from this treatment. The medication is designed to replace the missing Factor VIII protein, which is essential for proper blood clotting.

Who can participate: Male and female patients of all ages with severe Hemophilia A who completed previous trials with efanesoctocog alfa and are experiencing clinical benefits. Participants must be willing to join within 7 days of their last dose in the previous study. They need to provide informed consent and be able to use a study diary to record treatment information.

Who cannot participate: Patients who have not been previously treated for Hemophilia A, do not have the severe form of the condition, are pregnant or breastfeeding, or have participated in another recent clinical trial are excluded. Those with other medical conditions that might interfere with the study or who have a history of allergic reactions to the study medication cannot join.

Treatment approach: Efanesoctocog alfa is administered as an intravenous injection. The medication helps prevent bleeding episodes by providing regular prophylactic treatment. Throughout the study, participants are monitored for safety and efficacy, including tracking bleeding episodes and any side effects. The study aims to gather long-term data on the treatment’s safety and effectiveness.

Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A

This trial evaluates the long-term safety of BMN 270 (valoctocogene roxaparvovec), a gene therapy designed to help the body produce Factor VIII. The study involves participants who have previously received this treatment in earlier clinical trials.

Who can participate: Male participants who have completed a primary treatment study with BMN 270 or are enrolled in one at the time of its closure. They must have severe Hemophilia A with Factor VIII levels less than 6 IU/mL and weigh at least 3 kg. Participants can rejoin even if they have restarted regular Factor VIII prophylaxis or emicizumab treatment.

Who cannot participate: Female patients and those without severe Hemophilia A are excluded. The study is not open to vulnerable populations.

Treatment approach: This is a gene therapy administered as a single intravenous infusion. The study monitors participants over an extended period for adverse events, including liver problems, blood clots, and the development of inhibitors. Researchers assess changes in bleeding rates, Factor VIII activity levels, and overall quality of life.

Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

This study examines different treatment approaches for patients with Hemophilia A who have developed inhibitors against Factor VIII. It evaluates the effectiveness of Emicizumab combined with immune tolerance induction therapy using various Factor VIII products.

Who can participate: Male patients of any age with severe or moderate Hemophilia A and a historical inhibitor titre of at least 0.6 BU/mL. Participants must be undergoing immune tolerance induction therapy with specific Factor VIII products and may also receive prophylactic treatment with emicizumab or bypassing agents.

Who cannot participate: Female patients cannot participate. Those not within specified age ranges or without documented Hemophilia A with inhibitors are excluded.

Treatment approach: The study involves administering Emicizumab subcutaneously alongside intravenous Factor VIII concentrates. The goal is to evaluate the success of immune tolerance induction, measured by achieving an inhibitor titre below 0.6 BU/mL. Researchers also assess bleeding rates, joint health, and any adverse events throughout the treatment period.

Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A

This trial focuses on evaluating the long-term effectiveness of efanesoctocog alfa in preventing joint bleeds in patients with Hemophilia A who are receiving this medication as prophylactic treatment.

Who can participate: Male and female patients with a confirmed diagnosis of Hemophilia A who have been prescribed efanesoctocog alfa for prophylactic treatment within the last six months. They must have been on prophylactic treatment for at least 12 months prior with any approved Factor VIII product or emicizumab, with documented treatment history and bleeding episodes.

Who cannot participate: Patients without Hemophilia A, those outside specified age ranges, or those unwilling to follow study procedures are excluded. Those with other medical conditions interfering with the study, pregnant or breastfeeding women, and those with allergic reactions to the medication cannot participate.

Treatment approach: Efanesoctocog alfa is administered intravenously on a regular schedule. The study uses ultrasound and patient-reported outcomes to monitor joint health over time. Researchers track bleeding episodes, joint health scores, and overall quality of life throughout the study period.

Study on Long-Term Safety and Effects of Marstacimab for Patients with Severe Hemophilia A or B, With or Without Inhibitors

This long-term extension study evaluates the safety and effectiveness of Marstacimab for individuals with severe Hemophilia A or moderately severe to severe Hemophilia B, whether or not they have developed inhibitors.

Who can participate: Male participants aged 1 to 74 years who successfully completed either Study B7841005 or Study B7841008 without needing to leave early. They must meet age and weight requirements from their previous study and be willing to attend all scheduled visits and follow the treatment plan.

Who cannot participate: Patients with Factor VIII activity of 1% or more (for Hemophilia A) or Factor IX activity of more than 2% (for Hemophilia B) are excluded. Female patients and those outside the age range of 1 to less than 75 years cannot participate.

Treatment approach: Marstacimab is administered as a subcutaneous injection using a pre-filled pen or syringe. The study monitors participants for side effects, bleeding episodes, and joint health over an extended period. Regular assessments track the medication’s effectiveness in preventing bleeding and improving overall quality of life.

Study on Marstacimab for Children with Severe Hemophilia A or B, With or Without Inhibitors

This study investigates the effectiveness and safety of Marstacimab in children and teenagers under 18 years old with severe forms of Hemophilia A or B.

Who can participate: Male participants aged 1 to 18 years with severe Hemophilia A or moderately severe to severe Hemophilia B, with or without inhibitors. They must have a minimum body weight depending on age and documented treatment history or bleeding episodes. For ages 12 to 18, specific weight requirements apply.

Who cannot participate: The study excludes patients with serious medical conditions that could interfere with the trial, those with allergic reactions to the medication, active infections, liver or kidney disease, heart problems, or recent participation in another clinical trial. Patients with mental health conditions affecting study participation or those with planned surgeries are also excluded.

Treatment approach: Marstacimab is given as a subcutaneous injection according to the study protocol. The trial compares the new treatment with the standard treatment participants received over the past year, measuring bleeding episodes, joint health, and quality of life over approximately 48 weeks.

Study on Personalized Dosing of Emicizumab for Patients with Congenital Hemophilia A

This trial investigates whether a personalized dosing approach for emicizumab, guided by drug levels in the blood, is as effective as the standard dosing method in preventing bleeding episodes.

Who can participate: Male and female patients older than 1 year with confirmed congenital Hemophilia A and Factor VIII levels less than 6 IU/mL. They must have been receiving emicizumab at a conventional dose for at least 12 months with good bleeding control (no unexpected joint or muscle bleeds in the last 6 months and no more than two treated injury-related bleeds).

Who cannot participate: Patients without congenital Hemophilia A, females in certain circumstances, those outside specified age ranges, or those considered part of vulnerable populations cannot participate.

Treatment approach: Emicizumab is administered subcutaneously at 6 mg per kilogram of body weight every four weeks. The study compares conventional dosing with pharmacokinetic-guided dosing, where doses are adjusted based on how the body processes the drug. Researchers monitor bleeding events, joint health, and quality of life throughout different study phases.

Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

This study observes changes in joint health in patients with Hemophilia A who receive regular doses of efanesoctocog alfa, focusing on synovial hypertrophy (thickening of joint lining).

Who can participate: Male and female patients at least 12 years old with moderate or severe Hemophilia A (5% or less Factor VIII levels). They must have been receiving regular preventive treatment with approved Factor VIII products or emicizumab for at least 12 months, have at least one qualifying joint, and possess 12 months of recorded treatment history.

Who cannot participate: Patients without Hemophilia A, those outside specified age ranges, or those from vulnerable populations are excluded.

Treatment approach: Efanesoctocog alfa is administered intravenously once weekly. The study uses ultrasound and MRI imaging to monitor joint condition over 12 months, specifically looking for improvements in synovial hypertrophy. Researchers also track bleeding episodes, physical function, and quality of life.

Study on the Effectiveness of Concizumab for Children Under 12 with Hemophilia A or B, With or Without Inhibitors

This trial evaluates how well Concizumab works in preventing bleeding episodes in children under 12 years old with Hemophilia A or B, comparing it to their previous treatment.

Who can participate: Male children under 12 years old with severe Hemophilia A or moderate/severe Hemophilia B, with or without inhibitors. They must have medical records documenting treatment and bleeding episodes for specific periods and provide informed consent.

Who cannot participate: Female children and those 12 years or older are excluded.

Treatment approach: Concizumab is administered subcutaneously using a pen-injector device. The study monitors the number of bleeding episodes over time, comparing results to previous treatments. Researchers also track injection site reactions and any development of antibodies against Concizumab throughout the treatment period.

Study on the Use of Simoctocog Alfa and Emicizumab for Surgery in Patients with Severe Hemophilia A

This study evaluates the effectiveness of Nuwiq (simoctocog alfa) in managing bleeding during and after major surgery in patients with severe Hemophilia A who are already receiving Emicizumab prophylaxis.

Who can participate: Male patients at least 12 years old with severe Hemophilia A (less than 1% Factor VIII levels) who have been treated with any Factor VIII product for at least 150 exposure days. They must be on regular preventive treatment with emicizumab for at least 3 months before planned major surgery requiring Factor VIII treatment.

Who cannot participate: Patients without severe Hemophilia A, female patients, and those from vulnerable populations are excluded.

Treatment approach: Nuwiq is administered intravenously in combination with ongoing emicizumab treatment. The study monitors Factor VIII levels before and after injections, bleeding control during surgery, and overall treatment success. Researchers assess for side effects, the development of inhibitors, and any thrombotic events throughout the trial.

Summary

The 19 ongoing clinical trials for Factor VIII deficiency reflect a diverse research landscape spanning multiple European countries and addressing various aspects of treatment. Several notable patterns emerge from this overview.

Geographically, France, Germany, Italy, and Spain show the highest concentration of trials, each hosting 10 or more studies. This distribution suggests these countries have well-established hemophilia treatment centers and research infrastructure.

Therapeutically, the trials focus on several key approaches: long-acting Factor VIII replacement therapies like efanesoctocog alfa, gene therapies such as valoctocogene roxaparvovec and giroctocogene fitelparvovec, and novel prophylactic medications including emicizumab and concizumab. Many studies specifically address the needs of patients with inhibitors, a significant complication in hemophilia treatment.

The trials encompass various age groups, from young children to adults, with several studies specifically designed for pediatric populations. Some research focuses on special situations such as surgical management or includes female patients with hemophilia, a historically underrepresented group.

The emphasis on long-term safety monitoring, joint health assessment, and quality of life measures reflects a holistic approach to hemophilia care that extends beyond simply preventing bleeding episodes. These ongoing studies promise to expand treatment options and improve outcomes for people living with Factor VIII deficiency.

Ongoing Clinical Trials on Factor VIII deficiency

  • Study on the Effectiveness of Concizumab for Children Under 12 with Hemophilia A or B, With or Without Inhibitors

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria France Greece Italy Lithuania Norway +4

  • Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Norway Spain Sweden

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