In short
Clinical trials investigating Human Von Willebrand Factor are studying how it is used in bleeding disorders, especially in people with Haemophilia A with FVIII inhibitors and Type 3 Von Willebrand Disease. These studies mainly look at safety, effectiveness, and bleeding outcomes in Phase 3 trials.
Key points
- Clinical trials investigating Human Von Willebrand Factor in the provided data focus on bleeding disorders. One Phase 3 trial studies treatment approaches in Haemophilia A patients with FVIII inhibitors, and another Phase 3 trial studies bleeding prevention and control in Type 3 Von Willebrand Disease. The first trial measures inhibitor titre, FVIII recovery, FVIII half-life, and annualised bleeding rate. The second trial measures the number of treated bleeds over time. Both studies are interventional and are authorised.
Trial overview
The source data includes two interventional Phase 3 clinical trials that mention Human Von Willebrand Factor as part of the treatment options being studied.
These trials are focused on people with Haemophilia A and people with Type 3 Von Willebrand Disease.
Both studies are authorised and are looking at treatment outcomes in real patient groups rather than healthy volunteers.
Haemophilia A with FVIII inhibitors
Trial NCT04023019 is an international investigator-initiated study in patients with Haemophilia A who have FVIII inhibitors, which are antibodies that block factor VIII treatment.
The study compares different treatment approaches and includes several medicines in the source list, including Human Von Willebrand Factor as one of the listed options.
The main goal for this trial is to assess immune tolerance induction (ITI) outcome in Groups 1 and 2.
ITI means trying to reduce or remove the body’s blocking response so factor VIII can work better again.
The trial also compares the annualised bleeding rate in Group 3 with the rate seen in Groups 1 and 2.
Type 3 Von Willebrand Disease
Trial 2024-515622-80-00 is a Phase 3 study in patients with Type 3 Von Willebrand Disease.
The study is designed to assess the safety and effectiveness of emicizumab when used on a scheduled basis to help prevent bleeds, compared with on-demand standard care.
The source data lists Human Von Willebrand Factor-related treatment products among the medicines used in the study, along with other clotting treatments.
The main endpoint is the number of treated bleeds over time, which shows how often bleeding episodes need treatment during the study.
Main endpoints and what they mean
Endpoints are the main results a trial measures to see whether a treatment is working.
In NCT04023019, the primary endpoint for Groups 1 and 2 requires three signs of success: inhibitor titre below 0.6 BU/mL for at least two tests in a row, FVIII recovery at least 66% of the reference value, and FVIII half-life of at least 6 hours.
This means the study is checking both the lab response and how long factor VIII stays in the body.
In the same trial, Group 3 is measured by annualised bleeding rate, which is the expected number of bleeds in one year.
In 2024-515622-80-00, the main endpoint is the number of treated bleeds over time, which helps show whether preventive treatment lowers bleeding episodes.
Study design and phases
Both trials are Phase 3 studies, which usually means the treatment has moved into later testing in larger patient groups.
Both are also interventional, meaning the researchers give treatment and then measure the results.
NCT04023019 has an enrollment of 123 people, while 2024-515622-80-00 has an enrollment of 79 people.
The status of both studies is listed as Authorised in the source data.
Patient-friendly terms
Bleeding disorder means a condition where blood does not clot normally, so bleeding can last longer than expected.
Inhibitors are antibodies that can stop a treatment from working well.
Prophylactic treatment means regular treatment given to help prevent bleeding before it starts.
On-demand treatment means treatment is given after a bleed happens.
Investigational use in these trials means the treatment approach is being studied, not simply used as routine care.