Table of contents
- Trial overview
- Haemophilia A with FVIII inhibitors
- Type 3 Von Willebrand Disease
- Main endpoints and what they mean
- Study design and phases
- Patient-friendly terms
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.[1][2]
These trials are focused on people with Haemophilia A and people with Type 3 Von Willebrand Disease.[1][2]
Both studies are authorised and are looking at treatment outcomes in real patient groups rather than healthy volunteers.[1][2]
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.[1]
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.[1]
The main goal for this trial is to assess immune tolerance induction (ITI) outcome in Groups 1 and 2.[1]
ITI means trying to reduce or remove the body’s blocking response so factor VIII can work better again.[1]
The trial also compares the annualised bleeding rate in Group 3 with the rate seen in Groups 1 and 2.[1]
Type 3 Von Willebrand Disease
Trial 2024-515622-80-00 is a Phase 3 study in patients with Type 3 Von Willebrand Disease.[2]
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.[2]
The source data lists Human Von Willebrand Factor-related treatment products among the medicines used in the study, along with other clotting treatments.[2]
The main endpoint is the number of treated bleeds over time, which shows how often bleeding episodes need treatment during the study.[2]
Main endpoints and what they mean
Endpoints are the main results a trial measures to see whether a treatment is working.[1][2]
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.[1]
This means the study is checking both the lab response and how long factor VIII stays in the body.[1]
In the same trial, Group 3 is measured by annualised bleeding rate, which is the expected number of bleeds in one year.[1]
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.[2]
Study design and phases
Both trials are Phase 3 studies, which usually means the treatment has moved into later testing in larger patient groups.[1][2]
Both are also interventional, meaning the researchers give treatment and then measure the results.[1][2]
NCT04023019 has an enrollment of 123 people, while 2024-515622-80-00 has an enrollment of 79 people.[1][2]
The status of both studies is listed as Authorised in the source data.[1][2]
Patient-friendly terms
Bleeding disorder means a condition where blood does not clot normally, so bleeding can last longer than expected.[1][2]
Inhibitors are antibodies that can stop a treatment from working well.[1]
Prophylactic treatment means regular treatment given to help prevent bleeding before it starts.[2]
On-demand treatment means treatment is given after a bleed happens.[2]
Investigational use in these trials means the treatment approach is being studied, not simply used as routine care.[1][2]
