Mim8 for Hemophilia A

(FRONTIER4 Trial)

Not currently recruiting at 297 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new medicine called Mim8 for individuals with hemophilia A, a condition where blood doesn't clot properly. Mim8 aims to prevent bleeding episodes by replacing the missing clotting factor in the body. The trial includes different groups, with some using a pen-injector and others an enhanced cartridge for administering Mim8. This trial may suit those with hemophilia A who are currently in related ongoing studies. Participants should be willing to follow the trial's procedures and restrictions on other medications. As a Phase 3 trial, this is 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 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.

Is there any evidence suggesting that Mim8 is likely to be safe for humans?

Research has shown that Mim8 is generally safe for people with hemophilia A. In a previous study, Mim8 did not cause major safety issues, and participants continued treatment without interruption. This suggests the drug is gentle on the body for most people. Another trial found that many patients did not experience serious side effects while using Mim8. These findings indicate its safety in treating hemophilia A. However, safety can vary for each person, so discussing any concerns with a healthcare provider is advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hemophilia A?

Researchers are excited about Mim8 for Hemophilia A because it offers a unique approach compared to traditional treatments like factor VIII replacement therapy. Unlike these standard treatments, Mim8 is administered subcutaneously, which is less invasive and potentially more convenient than intravenous options. Additionally, Mim8 uses a bispecific antibody mechanism to mimic factor VIII activity, which may provide more consistent bleeding control. This innovative approach could lead to improved management of Hemophilia A with fewer injections and a more straightforward delivery method.

What evidence suggests that Mim8 might be an effective treatment for hemophilia A?

Research shows that Mim8 holds promise for people with hemophilia A. Studies have found Mim8 effective in reducing bleeding episodes. For instance, one study found that 74.3% of participants experienced no treated bleeds when receiving Mim8 weekly. Another study showed an average of just 0.53 bleeding events per year with Mim8, indicating fewer bleeds over time. These results suggest that Mim8 can help manage hemophilia A by replacing the missing clotting factor VIII, leading to less bleeding and potentially fewer complications. Participants in this trial will receive Mim8 through various administration methods, such as the DV3407 pen-injector or an enhanced cartridge, to evaluate its effectiveness and convenience. ^{1 2 6 7 8}

Who Is on the Research Team?

Clinical Transparency dept. 2834

Principal Investigator

Novo Nordisk A/S

Are You a Good Fit for This Trial?

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

Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: Arm 3 Experimental Treatment 1 Intervention

In part 1 and 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector.

Group II: Arm 2 Experimental Treatment 1 Intervention

Participants entering from study NN7769-4514, NN7769-4728 and NN7769-4516. In part 1, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector for 26 weeks. In part 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector.

Group III: 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) subcutaneous (s.c.) administration using enhanced cartridge for 26 weeks. In part 2, participants will receive Mim8 PPX 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

Citations

1. rarediseaseadvisor.com rarediseaseadvisor.com/news/once-weekly-mim8-shows-promising-outcomes-in-pediatric-hemophilia-a/

Once-Weekly Mim8 Shows Promising Outcomes in ... Among 70 participants aged 1 to 11 years, 74.3% of those receiving once-weekly Mim8 experienced 0 treated bleeds, with an estimated mean ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/718/530807/Safety-and-Efficacy-of-Mim8-Prophylaxis

Safety and Efficacy of Mim8 Prophylaxis Administered Once ... This interim analysis investigates the safety and efficacy of Mim8 prophylaxis once every two weeks (Q2W) in Arm 1 of FRONTIER4 (26 weeks).

3. pharmexec.com pharmexec.com/view/mim8-demonstrates-promising-results-pediatric-patients-hemophilia-a

Mim8 Demonstrates Promising Results in Pediatric ... Results found that Mim8 demonstrated an estimated mean annualized bleeding rate (ABR) of 0.53 and a median ABR of zero. Additionally, 74.3% of ...

4. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/1212/532244/Mim8-Prophylaxis-Beyond-Bleeding-Investigating

Mim8 Prophylaxis Beyond Bleeding: Investigating Multifaceted ... In the phase 3 FRONTIER2 study (NCT05053139), Mim8 demonstrated hemostatic efficacy by reducing annualized bleeding rate (ABR) for treated ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10514552/

Mim8, a novel factor VIIIa mimetic bispecific antibody, ... Mim8 is a novel drug under development for patients with hemophilia A. We report the safety, tolerability, pharmacokinetics, and pharmacodynamics of Mim8.

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124034657

Safety and Efficacy of Mim8 Prophylaxis Administered ... This interim analysis of FRONTIER4 showed that Mim8 Q2W prophylaxis was well tolerated with no participants discontinuing treatment, and no safety concerns were ...

7. hematologyadvisor.com hematologyadvisor.com/news/mim8-pediatric-hemophilia-promising-outcomes-treatment-risk/

Once-Weekly Mim8 Shows Promising Outcomes in ... In the FRONTIER3 trial, 74.3% of children receiving once-weekly Mim8 prophylaxis experienced zero treated bleeds.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05053139

NCT05053139 | A Research Study Investigating Mim8 in ... This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors.