Rare diseases
Investigational molecules
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Recruiting trials first
2 clinical trials in this condition
Croatia
FranceIn short
There are currently 2 ongoing clinical trials investigating new treatment options for people with Haemophilia B who have developed inhibitors (anti-Factor IX antibodies). These trials are taking place across several European countries and are testing innovative medications designed to reduce bleeding episodes in patients where standard treatments are less effective.
This trial is investigating whether concizumab, an investigational medication given as a regular injection under the skin, can reduce bleeding episodes in people with Haemophilia B who have developed inhibitors. Inhibitors are antibodies that make standard treatments less effective, creating significant challenges in managing the condition.
Who can join this trial?
To participate, you must meet several criteria:
Who cannot participate?
The trial excludes female patients and those who are part of vulnerable populations who may need special protection or are unable to give proper consent.
What is the focus of this trial?
The main goal is to compare how well concizumab works in preventing bleeding episodes compared to having no regular preventive treatment. Participants will be randomly divided into two groups: one receiving regular concizumab injections and another receiving their usual on-demand treatment with bypassing agents. The study will last at least 32 weeks, during which researchers will carefully monitor the number of bleeding episodes, any side effects such as injection site reactions, and changes in physical functioning and pain levels. The trial is expected to conclude by the end of 2025.
About concizumab
Concizumab works by inhibiting a protein called tissue factor pathway inhibitor (TFPI), which plays a role in regulating blood clotting. By blocking this protein, concizumab aims to enhance the body's ability to form clots and reduce bleeding risks. The medication is administered using a pen-injector device in doses of either 40 mg/mL or 100 mg/mL, depending on individual needs.
This clinical trial is evaluating SerpinPC, a modified form of a protein called human alpha-1 proteinase inhibitor, as a potential treatment for people with Haemophilia B who have developed inhibitors. The medication is given as an injection under the skin on a regular basis.
Who can join this trial?
Eligibility requirements include:
Who cannot participate?
Female patients cannot participate, as the trial is limited to males. Patients who do not have Haemophilia B, those outside the specified age range, and individuals considered part of vulnerable populations are also excluded.
What is the focus of this trial?
The primary objective is to evaluate how effective and safe SerpinPC is in reducing bleeding episodes over the first 24 weeks of treatment. The study will also assess bleeding rates over 48 weeks and beyond, monitor overall health and quality of life using specialized questionnaires, and track any side effects. Participants will keep a diary to record any bleeding events and medication usage throughout the study. The trial is estimated to conclude by October 2025.
About SerpinPC
SerpinPC is designed to inhibit certain proteins that interfere with the blood clotting process, thereby promoting better clot formation and reducing bleeding episodes. As a therapeutic agent in hematology (the study of blood disorders), it offers a new approach to managing bleeding in patients where conventional treatments are less effective due to inhibitors.
Both ongoing trials focus on innovative approaches to managing bleeding in people with Haemophilia B who have developed inhibitors. These trials represent important research efforts, as inhibitors make standard replacement therapy less effective and create significant treatment challenges.
The concizumab trial is available in eight European countries (Denmark, Poland, France, Spain, Sweden, Italy, Portugal, and Croatia), while the SerpinPC trial is being conducted in four countries (Italy, Spain, Germany, and France). France, Italy, and Spain are hosting both trials, providing more options for patients in these locations.
Both trials use similar approaches, requiring regular injections under the skin and focusing on reducing the frequency of bleeding episodes. However, they test different medications with distinct mechanisms of action. Concizumab works by targeting tissue factor pathway inhibitor, while SerpinPC is a modified protein designed to support the clotting process differently.
These trials are particularly important for males aged 12 and older who struggle with frequent bleeding episodes despite current treatments. Both studies emphasize careful monitoring of bleeding patterns, safety, and quality of life, reflecting a comprehensive approach to evaluating these potential new treatment options.
This guide is here to help you understand the condition. It does not replace a conversation with your doctor, who knows your situation best.
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