In short
Clinical trials of Human Coagulation Factor Viii are studying treatment approaches in people with bleeding disorders, especially Haemophilia A and Type 3 Von Willebrand Disease. These trials look at safety, effectiveness, bleeding control, and treatment outcomes in patients with FVIII inhibitors or severe bleeding risk.
Key points
- Clinical trials investigating Human Coagulation Factor Viii in the source data focus on bleeding disorders, mainly Haemophilia A and Type 3 Von Willebrand Disease. Both studies are Phase 3 and are authorised. One trial studies patients with FVIII inhibitors and measures immune tolerance induction outcomes and annualised bleeding rate. The other trial studies safety and effectiveness of prophylactic emicizumab compared with on-demand standard care and measures the number of treated bleeds over time. Together, these trials look at how well treatment strategies control bleeding in different patient groups.
Trial overview
The source data include two authorised Phase 3 interventional studies that investigate treatment approaches related to Human Coagulation Factor Viii in bleeding disorders.
One study focuses on people with Haemophilia A who have FVIII inhibitors, and the other focuses on people with Type 3 Von Willebrand Disease.
Haemophilia A with FVIII inhibitors
The trial with NCT04023019 is an international investigator-initiated study in Haemophilia A patients with FVIII inhibitors.
It is a Phase 3 interventional study with 123 planned participants and an authorised status.
This study compares different treatment approaches, including products such as Nuwiq, NovoSeven, OCTANATE, FEIBA, Wilate, Hemlibra, and CEVENFACTA, as listed in the trial record.
The main goal is to evaluate immune tolerance induction (ITI) outcomes, which means checking whether the body becomes less likely to attack factor VIII treatment.
The trial also looks at bleeding control by comparing the annualised bleeding rate in one of the study groups.
Type 3 Von Willebrand Disease
The second trial, identified as 2024-515622-80-00, studies safety and effectiveness of prophylactic emicizumab in patients with Type 3 Von Willebrand Disease.
It is also a Phase 3 interventional study, authorised, with 79 planned participants.
The study compares scheduled preventive treatment, called prophylaxis, with on-demand standard care, which means treatment is given only when bleeding happens.
The trial record lists several other medicines used in the study setting, including ADVATE, ELOCTA, Voncento, VEYVONDI, Haemate P, Fanhdi, Willfact, FEIBA, NovoSeven, and Wilate.
The main endpoint is the number of treated bleeds over time, which shows how often bleeding episodes need treatment during the study.
Trial phases and participants
Both studies are in Phase 3, which usually means the treatment is being tested in larger patient groups to compare outcomes and gather stronger evidence.
The target populations are specific: the first trial is for people with Haemophilia A and FVIII inhibitors, while the second is for people with Type 3 Von Willebrand Disease.
Because the studies are different, the exact people who can join will depend on the condition being studied and the treatment group in the trial.
Endpoints and measures
In the Haemophilia A study, the primary endpoint for Groups 1 and 2 is ITI outcome, measured by three criteria: inhibitor titre below 0.6 BU/mL for at least two consecutive measurements, FVIII recovery of at least 66% of the predefined reference value, and FVIII half-life of at least 6 hours.
These measurements help show whether treatment is working as expected in patients with inhibitors.
For Group 3 in the same study, the primary endpoint is the annualised bleeding rate (ABR) compared with the ABR in Groups 1 and 2.
In the Type 3 Von Willebrand Disease study, the main endpoint is the number of treated bleeds over time, which is used to judge whether preventive treatment reduces bleeding compared with standard care.
Patient glossary of key terms
Interventional study means the researchers give a treatment and then measure the results.
Authorised means the study has been approved to go forward in the source record.
Inhibitor means the immune system has made a substance that blocks treatment.
Bethesda unit (BU/mL) is a lab measure used to report inhibitor levels in the blood.
Recovery means how much the factor level rises after treatment is given.
Half-life means how long a treatment stays active in the body before half of it is gone.
Bleed means a bleeding episode that may need treatment.
Prophylactic treatment means treatment given on a schedule to help prevent bleeding.