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Human Coagulation Factor Viii

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.

Table of Contents

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.[1][2]

One study focuses on people with Haemophilia A who have FVIII inhibitors, and the other focuses on people with Type 3 Von Willebrand Disease.[1][2]

Haemophilia A with FVIII inhibitors

The trial with NCT04023019 is an international investigator-initiated study in Haemophilia A patients with FVIII inhibitors.[1]

It is a Phase 3 interventional study with 123 planned participants and an authorised status.[1]

This study compares different treatment approaches, including products such as Nuwiq, NovoSeven, OCTANATE, FEIBA, Wilate, Hemlibra, and CEVENFACTA, as listed in the trial record.[1]

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.[1]

The trial also looks at bleeding control by comparing the annualised bleeding rate in one of the study groups.[1]

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.[2]

It is also a Phase 3 interventional study, authorised, with 79 planned participants.[2]

The study compares scheduled preventive treatment, called prophylaxis, with on-demand standard care, which means treatment is given only when bleeding happens.[2]

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.[2]

The main endpoint is the number of treated bleeds over time, which shows how often bleeding episodes need treatment during the study.[2]

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.[1][2]

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.[1][2]

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.[1][2]

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.[1]

These measurements help show whether treatment is working as expected in patients with inhibitors.[1]

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.[1]

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.[2]

Patient glossary of key terms

Interventional study means the researchers give a treatment and then measure the results.[1][2]

Authorised means the study has been approved to go forward in the source record.[1][2]

Inhibitor means the immune system has made a substance that blocks treatment.[1]

Bethesda unit (BU/mL) is a lab measure used to report inhibitor levels in the blood.[1]

Recovery means how much the factor level rises after treatment is given.[1]

Half-life means how long a treatment stays active in the body before half of it is gone.[1]

Bleed means a bleeding episode that may need treatment.[1][2]

Prophylactic treatment means treatment given on a schedule to help prevent bleeding.[2]

Trial ID Phase Condition studied Status Enrollment
NCT04023019 Phase 3 Haemophilia A with FVIII inhibitors Authorised 123
2024-515622-80-00 Phase 3 Type 3 Von Willebrand Disease Authorised 79

FAQ

  • Who can join clinical trials of Human Coagulation Factor Viii? The trials in the source data focus on people with Haemophilia A, including patients with FVIII inhibitors, and people with Type 3 Von Willebrand Disease. Each study has its own rules, so participation depends on the exact condition and treatment group.
  • What are these trials trying to find out? One trial measures immune tolerance induction (ITI) outcomes and bleeding rates in Haemophilia A with FVIII inhibitors. Another trial studies safety and effectiveness of prophylactic emicizumab compared with on-demand standard care in Type 3 Von Willebrand Disease.
  • What phase are the trials in? Both trials listed are Phase 3 studies. Phase 3 trials usually compare treatments in larger groups of patients to see how well they work and to measure important outcomes.
  • What outcomes are being measured? The studies measure things like inhibitor titre, FVIII recovery, FVIII half-life, annualised bleeding rate, and number of treated bleeds over time. These are ways to see whether treatment is helping control bleeding and improve treatment response.
  • Are these trials testing only Human Coagulation Factor Viii? The trial data include Human Coagulation Factor Viii-related treatment approaches, but they also include other medicines used for bleeding disorders. The studies are focused on treatment strategies and outcomes, not only one product.

Ongoing Clinical Trials on Human Coagulation Factor Viii

  • Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

    Recruiting

    1 1 1 1
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Poland +2

  • Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

    Recruiting

    1 1 1 1
    Investigated drugs:
    Bulgaria Croatia Finland Germany Norway Spain +1

Glossary

  • Haemophilia A: A bleeding disorder where the blood does not clot normally because of low or missing factor VIII.
  • Type 3 Von Willebrand Disease: A severe form of Von Willebrand disease, a bleeding disorder that can cause frequent or serious bleeding.
  • FVIII inhibitor: An antibody that blocks factor VIII treatment, making it less effective.
  • Inhibitor titre: A measurement of how much inhibitor is present in the blood. A lower number means less blocking of treatment.
  • Bethesda units (BU/mL): A lab unit used to measure factor VIII inhibitors in the blood.
  • Immune tolerance induction (ITI): A treatment approach used to help the immune system stop attacking factor VIII.
  • FVIII recovery: How much factor VIII level rises after treatment is given.
  • Half-life: The time it takes for half of a medicine to leave the body.
  • Annualised bleeding rate (ABR): The number of bleeding episodes expected over one year.
  • Prophylactic treatment: Treatment given on a regular schedule to help prevent bleeding.
  • On-demand treatment: Treatment given only after a bleed happens.

References