In short
This article provides information about ongoing clinical trials for acquired haemophilia, a rare bleeding disorder. Currently, there is 1 clinical trial recruiting participants in several European countries. The trial focuses on testing a new treatment called SerpinPC for patients who have developed inhibitors that make standard treatments less effective.
Clinical trial locations
- France
- Germany
- Italy
- Spain
Study on the Safety and Effectiveness of SerpinPC for Patients with Hemophilia B with Inhibitors
This trial is testing a new treatment called SerpinPC for people with Hemophilia B who have developed inhibitors. Inhibitors are antibodies that can make standard treatments less effective, leading to more frequent bleeding episodes. SerpinPC is given as an injection under the skin and is designed to help the blood clot better by blocking certain proteins that interfere with clotting.
Who can participate?
This study is looking for male participants between 12 and 65 years old who have been diagnosed with Hemophilia B. To be eligible, participants must have:
- A history of factor IX levels at 5% or less
- Developed inhibitors that require treatment with bypass agents
- At least 6 bleeding episodes per year without preventive treatment, or at least 2 episodes per year if on preventive treatment
- Good overall health, including adequate blood counts (platelet count of at least 100,000 per microliter and hemoglobin of at least 10 grams per deciliter)
- Healthy liver function with no signs of cirrhosis
- Adequate kidney function
Participants must also be willing to document bleeding episodes in a diary for at least 12 weeks and, if sexually active with a partner who can become pregnant, agree to use effective birth control during the study.
Who cannot participate?
The study excludes female patients, as only males can participate. Additionally, individuals who do not have Hemophilia B, those outside the specified age range, or those considered part of a vulnerable population requiring special protection are not eligible.
What happens during the trial?
The study lasts 48 weeks and is divided into several phases. After joining and providing written consent, participants undergo initial assessments to confirm they meet the health requirements. If they haven't already documented their bleeding episodes for 12 weeks, they will need to complete this observation period to establish a baseline.
Once enrolled, participants receive regular injections of SerpinPC under the skin. The main goal is to see how well the treatment reduces bleeding episodes. During the first 24 weeks, bleeding rates are closely monitored, and participants continue to record any bleeding events in a diary. The study then extends to 48 weeks and beyond to evaluate long-term effectiveness and safety. Researchers also assess participants' overall health and quality of life using specialized questionnaires.
The trial is being conducted in France, Germany, Italy, and Spain and is expected to conclude by October 2025.
The investigational drug: SerpinPC
SerpinPC is a modified form of a protein called human alpha-1 proteinase inhibitor. It works by inhibiting certain proteins that interfere with blood clotting, helping to promote better clot formation. This approach aims to provide a new treatment option for people with Hemophilia B who have inhibitors, potentially reducing the frequency of bleeding episodes and improving their quality of life.
Summary
Currently, there is one active clinical trial for acquired haemophilia focusing on patients with Hemophilia B who have developed inhibitors. The trial is testing SerpinPC, a novel treatment designed to reduce bleeding episodes by improving blood clotting. The study is being conducted across four European countries: France, Germany, Italy, and Spain, providing access to patients in multiple locations. This trial represents an important step in finding new treatment options for individuals whose condition has become resistant to standard therapies. Patients interested in participating should consult with their healthcare provider to determine if they meet the eligibility criteria.
