Mim8 for Hemophilia A

No longer recruiting at 102 trial locations

Overseen By Novo Nordisk

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novo Nordisk A/S

Must be taking: FVIII concentrate, Bypassing agent

Must not be taking: Non-factor haemostatics

Disqualifiers: Coagulation disorders, Autoimmune disease, 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: 52 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 children with hemophilia A, a condition where blood doesn't clot properly, leading to frequent bleeding. The goal is to evaluate how well Mim8 prevents these bleeds compared to other treatments. Participants will receive Mim8 injections over 52 weeks and can use additional medicine if bleeding occurs. Children aged 1-11 who have been diagnosed with hemophilia A and have had recent treatment are suitable candidates for this study. 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.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop your current medications. However, if you are using non-factor haemostatic products for bleeding prevention, you should not have used them within 6 months before joining the trial.

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

Research shows that Mim8 is generally safe for people with hemophilia A. Studies have not identified any major safety concerns with Mim8. In one study, no participants stopped treatment due to safety issues. Another study found that 98% of caregivers preferred Mim8 over previous treatments, indicating that serious side effects were rare. Overall, these findings suggest that Mim8 is safe for humans, based on the available data. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Mim8 is unique because it offers a new approach to managing Hemophilia A. Unlike traditional treatments that often involve frequent intravenous infusions of factor VIII, Mim8 is designed as a subcutaneous prophylactic treatment, which might simplify administration and improve patient compliance. It works by mimicking the activity of factor VIII, providing a more consistent and stable control of bleeding episodes. Researchers are excited because this could mean fewer treatments and less disruption to daily life for patients.

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

Research has shown that Mim8 holds promise for children with hemophilia A. In one study, 74.3% of children who received weekly Mim8 experienced no treated bleeds. Another report noted an average of just 0.53 bleeds per year, with many children experiencing none. Mim8 mimics the action of a protein called factor VIIIa, which aids in blood clotting and is crucial for preventing bleeds in hemophilia A. These findings suggest that Mim8 can effectively reduce bleeding in children with this condition. ^{1 2 3 6 7}

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 children aged 1-11 with severe haemophilia A, regardless of whether they have inhibitors. They must have used FVIII concentrate or a bypassing agent recently and be able to follow the study plan. Children can't join if they've had thromboembolic disease treatment (except certain past catheter-associated thrombosis), other coagulation disorders, conditions that increase bleeding or thrombosis risk, hepatic dysfunction, high serum creatinine levels, recent participation in another interventional study, mental incapacity or lack of parental support.

Inclusion Criteria

Informed consent obtained before any study-related activities

I have been treated with FVIII or a bypassing agent in the last 6 months.

I am between 1 and 11 years old.

Exclusion Criteria

Pregnancy (female participants, assessed at investigator's discretion)

I have a blood clotting disorder that is not haemophilia A.

Known or suspected hypersensitivity to trial product or related products (assessed at the investigator's discretion for China mainland)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Mim8 prophylaxis for prevention of bleeds

52 weeks

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

20 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mim8

Trial Overview The trial tests Mim8 as a preventive treatment for bleeds in children with haemophilia A. Mim8 will be injected under the skin and compared to other medicines over approximately one to two years. If participants experience bleeds during the trial, additional treatments may be provided.

How Is the Trial Designed?

Group I: Mim8 Experimental Treatment 1 Intervention

52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis

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. 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.

4. 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 ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124039594

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

6. 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.

7. 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 ...