What side effects are associated with this type of intervention?

Common treatments for Hemophilia A that replace the function of the missing clotting factor VIII (FVIII) include recombinant factor VIII products and emicizumab. Recombinant factor VIII products can cause side effects such as the development of inhibitors (antibodies against factor VIII), allergic reactions, and injection site reactions. Emicizumab, which is administered subcutaneously, has been associated with injection site reactions, and in rare cases, thrombotic events and thrombotic microangiopathy (TMA) when used with activated prothrombin complex concentrates. Overall, these treatments are generally well-tolerated, but monitoring for these potential side effects is important.

What prior FDA approvals exist?

The FDA has approved several treatments for Hemophilia A that replace the function of the missing clotting factor VIII (FVIII). These include recombinant factor VIII products such as Kogenate, Advate, and Eloctate. These treatments are designed to prevent and control bleeding episodes by providing the necessary clotting factor. Emicizumab, another FDA-approved treatment, works differently by bridging activated factor IX and factor X to restore the blood clotting process, although it does not directly replace FVIII. These therapies have significantly improved the management of Hemophilia A, offering various dosing schedules and administration routes to suit patient needs.

What other types of research exist?

Promising novel alternatives to Mim8 for Hemophilia A patients include: 1) Fourth generation recombinant human factor VIII products such as Afstyla and Nuwiq, which are produced in human cell lines and have no added human or animal protein. 2) Recombinant human factor VIII pegylated products like Adynovate, which offer extended half-life. 3) Recombinant human factor VIII Fc fusion protein (Eloctate), which also provides prolonged activity. These alternatives have shown efficacy in clinical trials and represent advanced therapeutic options for Hemophilia A patients.

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Replacement Therapy

Mim8 for Hemophilia A (FRONTIER 5 Trial)

Phase 3

Waitlist Available

Research Sponsored by Novo Nordisk A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from visit 2 (week 0) until week 26

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing the safety of switching from emicizumab to Mim8 in people with haemophilia A. Mim8 is a new medicine that helps blood clot by replacing a missing protein. Participants will use Mim8 regularly as advised by their doctor.

Who is the study for?

This trial is for males and females, 12 years or older with haemophilia A who have been treated with emicizumab for at least 8 weeks. They must be willing to switch to Mim8 and follow the study procedures. Excluded are those with hypersensitivity to similar drugs, other coagulation disorders, planned surgeries during the study, liver or kidney issues, pregnant or breastfeeding women, and anyone not using effective contraception.

What is being tested?

The safety of switching from Emicizumab (a current treatment) to Mim8 (a new medicine) in preventing bleeding in people with haemophilia A is being tested. Participants will self-inject Mim8 under their skin at different frequencies chosen in consultation with a doctor over a period of about half a year.

What are the potential side effects?

While specific side effects aren't listed here, participants should watch out for any signs of allergic reactions due to known sensitivities. The restrictions on certain conditions suggest potential risks like increased bleeding or thrombosis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from visit 2 (week 0) until week 26

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from visit 2 (week 0) until week 26

This trial's timeline: 3 weeks for screening, Varies for treatment, and from visit 2 (week 0) until week 26 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of treatment-emergent adverse events

Secondary study objectives

Change in participants' treatment burden using the Hemophilia treatment experience measure (Hemo-TEM) total score

Device handling experience using the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: NNC0365-3769 (Mim8) PPXExperimental Treatment1 Intervention

Participants will receive Mim8 prophylaxis (PPX) subcutaneous (s.c.) injection using a prefilled fixed dose DV3407-C1 pen-injector.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Hemophilia A include factor VIII replacement therapy, bispecific antibodies like emicizumab, and investigational treatments such as Mim8. Factor VIII replacement therapy involves infusing recombinant or plasma-derived factor VIII to replace the missing or deficient protein, directly aiding in blood clot formation. Bispecific antibodies, such as emicizumab, mimic the function of factor VIII by bridging activated factor IX and factor X, facilitating the clotting process. Mim8, similar to emicizumab, is a bispecific antibody that replaces the function of factor VIII. These treatments are crucial for Hemophilia A patients as they help prevent and control bleeding episodes, reducing the risk of joint damage and other complications associated with uncontrolled bleeding.