Ongoing Clinical Trials for Haemophilia A without Inhibitors
There are currently 3 ongoing clinical trials testing new treatments for Haemophilia A without inhibitors. These studies are evaluating medications designed to prevent bleeding episodes and improve joint health and quality of life. The trials are taking place across several European countries and involve participants of various ages, from infants to adults. The main treatments being studied include emicizumab and SerpinPC, both given as injections under the skin.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- France
- Germany
- Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab
- Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
- Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
- Hungary
- Italy
- Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab
- Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
- Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
- Poland
- Spain
- Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab
- Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
- Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab
This study is evaluating the medication emicizumab, also known as Hemlibra, in people aged 13 to 69 years with severe or moderate Haemophilia A. The trial aims to understand how this treatment affects joint health, overall health, and physical activity levels over time.
Who can participate: To join this trial, participants must have a diagnosis of severe or moderate Haemophilia A with very low levels of factor VIII. They must test negative for FVIII inhibitors, meaning their body has not developed substances that block the action of factor VIII. Participants should have been on standard FVIII prophylaxis treatment for at least 24 weeks before entering the study. They must also have adequate blood, liver, and kidney function. Women of childbearing age must agree to use contraception during the treatment and for at least 24 weeks after the last dose.
Who cannot participate: The study excludes patients who do not have severe or moderate Haemophilia A, those outside the specified age range, pregnant or breastfeeding women, and anyone currently participating in another clinical trial. Patients with other medical conditions that might interfere with the study, those who have had recent surgery or are planning surgery soon, people with a history of allergic reactions to the study medication, or those with a history of drug or alcohol abuse are also excluded.
What the study involves: Participants will receive emicizumab as an injection under the skin at a dosage of 150 mg/mL. The study will monitor joint health using ultrasound and MRI scans at the start and at 36 months. Physical activity will be tracked using wearable devices like Fitbit to record daily steps and active minutes. Researchers will also assess quality of life through questionnaires and monitor the number and types of bleeding episodes. Safety will be monitored throughout, including checking for injection-site reactions and other potential side effects. The study is expected to conclude by December 2026.
Treatment being tested: Emicizumab is designed to prevent bleeding by helping blood clot more effectively. It is given regularly to reduce bleeding episodes and improve joint health and quality of life.
Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
This clinical trial focuses on testing emicizumab in infants from birth to 12 months old. The study aims to evaluate how effective and safe this medication is in preventing bleeding episodes in very young patients who do not have inhibitors.
Who can participate: Only male infants with a diagnosis of severe congenital Haemophilia A are eligible. They must have natural factor VIII levels less than 1% and test negative for FVIII inhibitors during the screening period. Infants should be either previously untreated or minimally treated, meaning they have had up to 5 days of exposure to hemophilia-related treatments. They must have received vitamin K prophylaxis according to local standards, and documentation of any previous treatments and bleeding episodes since birth is required.
Who cannot participate: Infants with any other bleeding disorders besides Haemophilia A are excluded. Those who have had recent surgery or are planning surgery, have active infections, or have a history of allergic reactions to similar treatments cannot participate. Patients currently in another clinical trial or with serious medical conditions that might interfere with the study are also excluded.
What the study involves: After a two-week screening period to confirm eligibility, participants will receive emicizumab through subcutaneous injection. The medication is available in two forms: 30 mg/mL and 150 mg/mL solutions. Throughout the study, infants will be monitored for bleeding episodes, including treated and spontaneous bleeds. Joint health will be assessed using specific scoring systems and imaging techniques during a long-term follow-up period of up to seven years. Safety will be carefully monitored, with particular attention to adverse events and injection-site reactions. The study is expected to conclude by May 2030.
Treatment being tested: Emicizumab mimics the activity of factor VIII by bridging activated factor IX and factor X, thereby promoting blood clotting and preventing or reducing bleeding episodes.
Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B
This study is evaluating SerpinPC, a new treatment for people with severe Haemophilia A or moderately severe to severe Haemophilia B. The medication is a modified form of a protein called human alpha-1 proteinase inhibitor, given as a regular preventive treatment through injection under the skin.
Who can participate: Male participants between 12 and 65 years old with severe Haemophilia A or moderately severe to severe Haemophilia B can join. For those with Haemophilia B, they must not have a high titer inhibitor (5 or more Bethesda Units per milliliter). Participants should either be part of a prophylaxis program or have experienced at least 6 bleeding episodes requiring treatment in the past 6 months if on an on-demand treatment plan. They must have at least 12 to 24 weeks of recorded bleeding episodes from a previous study or be willing to complete an observation period. No bleeding should occur in the 7 days before starting the study. Adequate blood, liver, and kidney function is required, along with a D-dimer level of 750 micrograms per liter or less.
Who cannot participate: Patients without moderately severe to severe hemophilia B, females, and those who are part of vulnerable populations such as children, elderly, or those with certain disabilities may not be eligible.
What the study involves: After an initial assessment to confirm eligibility, participants will undergo an observation period lasting at least 12 to 24 weeks to document baseline bleeding episodes. They will then receive SerpinPC as a subcutaneous injection, with dosage and frequency determined by the study protocol. Throughout the study, health and response to the medication will be closely monitored through regular check-ups. The study will track the number of treated bleeds, spontaneous bleeds, joint bleeds, and overall consumption of coagulation factors. The trial is expected to continue until July 2026.
Treatment being tested: SerpinPC works by inhibiting a specific protein in the blood that normally slows down clotting, thereby promoting better blood clot formation and preventing bleeding episodes. The goal is to help patients experience fewer bleeding problems and improve their quality of life.
Summary
The three ongoing clinical trials for Haemophilia A without inhibitors are being conducted across multiple European countries, with Germany, Italy, and Spain participating in all three studies. This suggests these countries have established research infrastructure for hemophilia treatment research.
Two of the three trials focus on emicizumab, targeting different age groups: infants from birth to 12 months and adolescents and adults aged 13 to 69 years. This demonstrates comprehensive research efforts to evaluate this medication across the lifespan. The third trial introduces a different approach by testing SerpinPC, offering a potential alternative treatment option for patients with severe forms of the condition.
All three studies share a common goal of reducing bleeding episodes and improving quality of life through preventive treatment given as subcutaneous injections. They employ comprehensive monitoring approaches, including assessment of joint health, physical activity levels, and safety profiles. The long-term nature of these studies, with some extending up to seven years of follow-up, reflects the commitment to understanding the sustained effects and safety of these treatments in managing this chronic condition.
