Mim8 for Hemophilia A

(FRONTIER4 Trial)

Recruiting at 241 trial locations

Overseen By Novo Nordisk

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Novo Nordisk A/S

Disqualifiers: Pregnancy, Major surgery, Mental incapacity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 262 weeks

451 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop your current medications. However, there are some restrictions on what medicines you can use, and the study doctor will provide more details.

What data supports the effectiveness of the drug Mim8 for treating Hemophilia A?

Research shows that Mim8, a next-generation treatment for Hemophilia A, has a strong ability to help blood clotting, which is crucial for people with this condition. In studies with animals and lab tests, Mim8 was found to be much more effective than similar treatments, helping to stop bleeding and improve clot formation. ^{1 2 3 4 5}

How is the drug Mim8 different from other treatments for hemophilia A?

Mim8 is a next-generation bispecific antibody that mimics factor VIII, used for preventing bleeding in hemophilia A patients. It is unique because it can be administered subcutaneously (under the skin) and has a higher potency compared to earlier treatments, making it effective even in patients with inhibitors. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Clinical Transparency dept. 2834

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

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.

Inclusion Criteria

I have been diagnosed with congenital haemophilia A.

Informed consent obtained before any study related activities

- Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728

Exclusion Criteria

Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol

Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728

I have no major surgeries planned during the early phase of the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Treatment

Participants receive Mim8 prophylaxis with subcutaneous administration, frequency depending on prior study participation

Up to 262 weeks

Regular visits for injections and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

21 weeks

Open-label extension

Participants continue to receive Mim8 until the study ends or the drug is marketed

Long-term, up to 5.5 years

Treatment Details

Interventions

• Mim8

Trial Overview 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.

Participant Groups

Group I: Arm 2 Experimental Treatment 1 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 1 Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Novo Nordisk A/S

Lead Sponsor

Trials

1,578

Recruited

3,813,000+

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

References

1. England pubmed.ncbi.nlm.nih.gov

A novel next-generation FVIIIa mimetic, Mim8, has a favorable safety profile and displays potent pharmacodynamic effects: Results from safety studies in cynomolgus monkeys. [2023]

2. England pubmed.ncbi.nlm.nih.gov

In vitro effects of combining Mim8 with factor VIII, FVIIa, and activated prothrombin complex concentrates in thrombin generation assays. [2023]

3. England pubmed.ncbi.nlm.nih.gov

The effect of a next generation factor VIII mimetic bispecific antibody (Mim8) on assays of factor VIII activity and thrombin generation. [2023]

4. England pubmed.ncbi.nlm.nih.gov

A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non-factor VIII related haemostasis assays. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. [2021]