Ongoing Clinical Trials for Haemophilia A With Anti Factor VIII

Currently, there are 2 ongoing clinical trials investigating new treatment approaches for haemophilia A with anti factor VIII (also known as haemophilia A with inhibitors). These studies are taking place across several European countries and are evaluating investigational medications aimed at reducing bleeding episodes in patients who have developed inhibitors against standard treatments.

Clinical trial locations

• Belgium
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Bulgaria
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Croatia
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
• Denmark
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
• France
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Germany
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Italy
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Poland
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Portugal
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
• Spain
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
  • Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
• Sweden
  • Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors

Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors

This clinical trial is investigating concizumab, a medication designed to prevent bleeding episodes in patients with haemophilia A or B who have developed inhibitors. Inhibitors are antibodies that develop in some patients, making standard treatments less effective and making it more difficult to control bleeding.

Main inclusion criteria: To participate in this study, you must be male, at least 12 years old, and weigh more than 25 kilograms. You need to have congenital haemophilia A or B of any severity, meaning you were born with this condition. You must also have a documented history of an inhibitor with a level of 0.6 BU or higher. Additionally, you should have been prescribed, or need, treatment with bypassing agents in the last 24 weeks before screening. Bypassing agents are special treatments used when regular treatments don’t work well due to inhibitors.

Main exclusion criteria: Only male patients are eligible, so female patients cannot participate. Patients who are part of a vulnerable population, which generally refers to groups who may need special protection, are not eligible for this study.

Focus and goal: The main purpose of this study is to see how well concizumab works in reducing the number of bleeding episodes compared to no regular preventive treatment. Participants will be randomly divided into groups, with some receiving concizumab and others receiving their usual on-demand treatment with bypassing agents. The study will last for at least 32 weeks, during which researchers will monitor the number of bleeding episodes, any side effects, physical functioning, and quality of life. The trial is expected to conclude by the end of 2025.

Investigational drug: Concizumab is given as a solution for injection under the skin using a pen-injector device. It works by inhibiting a protein called tissue factor pathway inhibitor, which plays a role in regulating blood clotting. By blocking this protein, concizumab aims to enhance clot formation and reduce bleeding risks. The medication is used as a prophylactic treatment, meaning it is given regularly to help prevent bleeding rather than treating it after it occurs.

Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B

This clinical trial is focused on evaluating SerpinPC, a treatment for individuals with severe haemophilia A or moderately severe to severe haemophilia B. The study aims to assess how effective and safe SerpinPC is when given as a regular preventive treatment through injections under the skin.

Main inclusion criteria: You must be a male between 12 and 65 years old with severe haemophilia A or moderately severe to severe haemophilia B. If you have haemophilia B, you must not have a high titer inhibitor, defined as 5 or more Bethesda Units per milliliter. You should be part of a prophylaxis program, which is a preventive treatment plan, or have had at least 6 bleeding episodes that needed treatment in the last 6 months if on an on-demand treatment plan. You must have at least 12 weeks or 24 weeks of recorded bleeding episodes or be willing to complete an observation period. You should not have any bleeding in the 7 days before starting the study, and you must have adequate blood, liver, and kidney function.

Main exclusion criteria: Patients who do not have moderately severe to severe haemophilia B cannot participate. Only male patients are eligible, and patients who are part of a vulnerable population may not be eligible.

Focus and goal: The study aims to evaluate the efficacy and safety of SerpinPC in reducing bleeding episodes. Before starting the medication, there will be an observation period to document any bleeding episodes. Throughout the study, your health and response to the medication will be closely monitored through regular check-ups. The study will evaluate the number of treated bleeds, spontaneous bleeds, and joint bleeds, as well as overall consumption of coagulation factors. The trial is expected to continue until July 2026.

Investigational drug: SerpinPC is a solution for injection that contains a modified form of a protein called human alpha-1 proteinase inhibitor. It is given as a subcutaneous injection, meaning it is injected under the skin. The medication works by inhibiting a specific protein in the blood that normally slows down clotting, thereby promoting better blood clot formation. The goal is to prevent bleeding episodes and improve quality of life for patients with severe forms of haemophilia.

Summary

Both ongoing clinical trials for haemophilia A with inhibitors are investigating novel therapeutic approaches to prevent bleeding episodes in patients where standard treatments are less effective. The trials are taking place across multiple European countries, with particularly strong representation in France, Italy, Poland, and Spain, where both studies are being conducted.

Each trial is testing a different investigational medication: concizumab, which inhibits tissue factor pathway inhibitor, and SerpinPC, which is based on a modified form of human alpha-1 proteinase inhibitor. Both medications are administered through subcutaneous injection and aim to enhance blood clotting through different mechanisms. The trials are focused exclusively on male participants aged 12 years and older, reflecting the genetic nature of haemophilia. Results from these studies are expected between 2025 and 2026, potentially offering new treatment options for this challenging patient population.