What side effects are associated with this type of intervention?

Common treatments for Hemophilia A, such as recombinant factor VIII, emicizumab, and bypassing agents like FEIBA and rFVIIa, have several known side effects. Recombinant factor VIII can cause the development of inhibitors (antibodies) that reduce its effectiveness, allergic reactions, and infusion site reactions. Emicizumab, which is administered subcutaneously, may cause injection site reactions, and there is a risk of thrombotic events and thrombotic microangiopathy, particularly when used with activated prothrombin complex concentrates. Bypassing agents like FEIBA and rFVIIa can be prothrombotic, leading to potential complications such as blood clots. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

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 (rFVIII) products like Eloctate, which is a factor VIII-Fc fusion protein designed to extend the half-life of FVIII, and PEGylated factor VIII, which also aims to prolong its duration of action. Additionally, Emicizumab, a bispecific antibody that mimics the function of FVIII, has been approved for prophylactic treatment in Hemophilia A patients with and without inhibitors.

What other types of research exist?

Promising novel alternatives to Mim8 for Hemophilia patients in 2024 include: 1) Recombinant human factor VIII products like Advate, Kovaltry, and Novoeight, which are third-generation products with no added human or animal protein. 2) 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. 3) Recombinant human factor VIII pegylated products like Adynovate, which offer extended half-life. 4) Recombinant human factor VIII Fc fusion protein products like Eloctate, which also provide extended half-life and reduced infusion frequency. These alternatives offer various benefits such as improved safety profiles, extended half-life, and reduced infusion frequency.

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Replacement Therapy

Mim8 for Hemophilia A (FRONTIER4 Trial)

Phase 3

Recruiting

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 8 (week 26) until end of treatment (up to 262 weeks)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing Mim8, a new medicine to prevent bleeding in people with haemophilia A. It works by replacing the missing part in their blood that helps it clot. The study will last several years and involves frequent injections under the skin.

Who is the study for?

This trial is for males and females with congenital haemophilia A who are already participating in certain ongoing Mim8 studies. They must be able to follow the study schedule and procedures, including diary completion. Pregnant or breastfeeding women, those planning pregnancy, or not using effective contraception are excluded.

What is being tested?

The trial tests long-term use of Mim8, a new medication designed to prevent bleeding in people with haemophilia A by mimicking clotting factor VIII. Participants will receive up to 262 subcutaneous injections over a maximum of 5.5 years or until Mim8 becomes commercially available.

What are the potential side effects?

While specific side effects for Mim8 aren't listed here, similar treatments may cause injection site reactions, potential development of inhibitors (antibodies against the treatment), allergic reactions, and could potentially affect other medications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from visit 8 (week 26) until end of treatment (up to 262 weeks)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from visit 8 (week 26) until end of treatment (up to 262 weeks)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from visit 8 (week 26) until end of treatment (up to 262 weeks) 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

Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only)

Mim8 plasma concentration

Number of injection site reactions

+6 more

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

2Treatment groups

Experimental Treatment

Group I: Arm 2Experimental Treatment1 Intervention

Participants entering from study NN7769-4514 or NN7769-4516. In part 1, participants will receive Mim8 PPX QW or QM with s.c. administration using DV3407 pen-injector for 26 weeks. In part 2, participants will receive Mim8 PPX QW, Q2W or QM with s.c. administration using DV3407 pen-injector.

Group II: Arm 1Experimental Treatment1 Intervention

Participants entering from the multiple ascending dose (MAD) part of study NN7769-4513. In part 1, participants will receive Mim8 prophylaxis (PPX) once every two weeks (Q2W) with subcutaneous (s.c.) administration using enhanced cartridge for 26 weeks. In part 2, participants will receive Mim8 PPX once-weekly (QW), Q2W or once-monthly (QM) with s.c. administration using enhanced cartridge or DV3407 pen-injector once it is approved.

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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 involve replacing the missing clotting factor VIII (FVIII) to prevent or control bleeding episodes. Traditional treatments include infusions of plasma-derived or recombinant FVIII concentrates. Newer treatments, like Mim8, are designed to mimic the function of FVIII, enhancing the body's ability to form blood clots. These treatments are crucial for Hemophilia patients as they significantly reduce the frequency and severity of bleeding episodes, improving quality of life and reducing the risk of long-term joint damage and other complications.