In short
Currently, there is one ongoing clinical trial exploring new treatment options for patients with haemophilia B without inhibitors. This trial is testing an investigational medication called SerpinPC, designed to prevent bleeding episodes through subcutaneous injections. The study is being conducted across multiple European countries and aims to evaluate the safety and effectiveness of this preventive treatment approach.
Clinical trial locations
- Belgium
- Bulgaria
- France
- Germany
- Italy
- Poland
- Spain
Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
This clinical trial is investigating a new preventive treatment called SerpinPC for individuals living with moderately severe to severe haemophilia B without inhibitors, as well as those with severe haemophilia A. The study focuses on whether this treatment can reduce bleeding episodes and improve quality of life for patients.
Who can participate:
- Males between 12 and 65 years of age
- Individuals with moderately severe to severe haemophilia B without high titer inhibitors (less than 5 Bethesda Units per milliliter)
- Patients currently on a preventive treatment program or those who have experienced at least 6 bleeding episodes requiring treatment in the past 6 months
- Participants must have documented bleeding episodes from a previous study or be willing to complete an observation period of at least 12 to 24 weeks
- No active bleeding in the 7 days before starting the study treatment
- Adequate blood, liver, and kidney function with D-dimer levels of 750 micrograms per liter or less
Who cannot participate:
- Female patients are not eligible
- Individuals who do not have moderately severe to severe haemophilia B
- Members of vulnerable populations may not be eligible, including certain groups like children with specific vulnerabilities, elderly individuals, or those with certain disabilities
What the trial involves:
The study evaluates SerpinPC, a solution that contains a modified form of human alpha-1 proteinase inhibitor. This medication is given as an injection under the skin, known as a subcutaneous injection. The treatment works by inhibiting a specific protein in the blood that normally slows down clotting, thereby helping the blood to clot more effectively and preventing bleeding episodes.
Participants will first undergo an initial assessment to confirm eligibility, including a review of their medical history and recent bleeding episodes. Before starting the medication, there will be an observation period lasting at least 12 to 24 weeks to document any bleeding episodes. Once treatment begins, patients will receive regular SerpinPC injections, with the dosage and frequency determined by the study protocol.
Throughout the trial, participants will be closely monitored with regular check-ups to track their response to the medication. The study will record the number of treated bleeds, spontaneous bleeds, and joint bleeds, as well as overall consumption of clotting factors. The trial is designed to continue until July 2026, and the results will help determine whether SerpinPC can be a beneficial treatment option for managing this condition.
Main focus of the trial: The study aims to assess how effective SerpinPC is in reducing bleeding episodes and whether it is safe for regular preventive use. By helping the blood to clot more effectively, the treatment hopes to provide patients with fewer bleeding problems and an improved quality of life.
Summary
Currently, one clinical trial is available for patients with haemophilia B without inhibitors. This international study is being conducted across seven European countries: Belgium, Bulgaria, France, Germany, Italy, Poland, and Spain. The wide geographic distribution provides access opportunities for patients across multiple regions.
The trial focuses on SerpinPC, an investigational medication designed for subcutaneous administration as a preventive treatment. This represents a novel approach to managing bleeding episodes by promoting better blood clot formation through the inhibition of specific proteins that normally slow down clotting.
The study includes both adolescents from age 12 and adults up to 65 years, reflecting an effort to evaluate the treatment across different age groups. Participants must commit to a comprehensive monitoring program that includes an initial observation period and regular follow-up assessments extending until 2026.
