Concizumab for Hemophilia
(explorer7 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores how well a new medicine, concizumab (an anti-TFPI monoclonal antibody), can help people with hemophilia A or B who have inhibitors. These inhibitors make usual treatments less effective. The trial aims to determine if concizumab can prevent bleeding and is safe to use. Participants will either begin taking concizumab immediately or continue their usual treatment for six months before switching to concizumab. This trial may suit those managing hemophilia with on-demand or preventive treatments and who have recently used or need bypassing agents. Participants will self-administer daily injections with a pen-like device at home. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop your current medications. However, if you usually take medicine to prevent bleeds, you will start the study medicine right away. If you take medicine only when you have a bleed, you might continue your normal medicine for the first 6 months.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that concizumab is generally safe for people with hemophilia A and B. Studies have found that patients tolerate it well when administered either intravenously or as a subcutaneous injection. It has a good safety record, with serious side effects being rare.
Early results suggest it effectively reduces bleeding without major safety concerns. Recent data have revealed no new safety issues, although more information is needed about its long-term safety. Overall, concizumab has demonstrated safety for many patients, but additional data will help confirm this over time.12345Why are researchers excited about this study treatment for hemophilia?
Unlike the standard treatments for hemophilia, which often involve regular infusions of clotting factors, concizumab offers a novel approach by targeting the tissue factor pathway inhibitor (TFPI). This mechanism helps promote clotting without directly replacing missing factors, potentially offering a more consistent and convenient management strategy. Moreover, concizumab is administered via a simple subcutaneous injection, making it easier and less invasive than traditional intravenous infusions. Researchers are excited about concizumab because it could improve the quality of life for patients by reducing bleeding episodes with a more straightforward treatment regimen.
What evidence suggests that concizumab might be an effective treatment for hemophilia?
Research has shown that concizumab reduces bleeding in people with hemophilia A and B. In this trial, participants will join different treatment arms, including those receiving concizumab prophylaxis. Studies have found that concizumab works well as a preventive treatment, stopping bleeds before they start. For people with hemophilia who have inhibitors (antibodies that make treatment less effective), concizumab successfully reduces the number of bleeding episodes. It has also proven safe for patients. Overall, concizumab is a promising option for managing bleeding in hemophilia, improving the quality of life for those affected.14678
Who Is on the Research Team?
Clinical Reporting Anchor and Disclosure (1452)
Principal Investigator
Novo Nordisk A/S
Are You a Good Fit for This Trial?
This trial is for males aged 12 or older with Hemophilia A or B and inhibitors, who have used bypassing agents in the last 24 weeks. It's not for those with thromboembolic disease, high risk of blood clots, ongoing immune treatments, hypersensitivity to similar drugs, or other coagulation disorders.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive daily concizumab injections under the skin, which they administer themselves at home. The treatment phase includes randomization into different groups, with some starting concizumab immediately and others after 6 months.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including recording information in an electronic diary and possibly wearing an activity tracker.
Extension
Participants in the extension part receive daily concizumab injections, continuing the treatment regimen.
What Are the Treatments Tested in This Trial?
Interventions
- Concizumab
Concizumab is already approved in European Union, United States, Canada for the following indications:
- Hemophilia A with inhibitors
- Hemophilia B with inhibitors
- Hemophilia A with factor VIII inhibitors
- Hemophilia B with factor IX inhibitors
- Hemophilia A with inhibitors
- Hemophilia B with inhibitors
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novo Nordisk A/S
Lead Sponsor
Lars Fruergaard Jørgensen
Novo Nordisk A/S
Chief Executive Officer since 2017
MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark
Martin Holst Lange
Novo Nordisk A/S
Chief Medical Officer since 2021
MD from University of Copenhagen