Mim8 for Hemophilia A

Recruiting at 67 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

70 Participants Needed

Trial Summary

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.

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

Research shows that Mim8, a next-generation treatment, effectively helps blood clotting in Hemophilia A by mimicking a missing protein. In studies with animals and lab tests, it was found to be more potent than similar treatments, helping to reduce bleeding significantly. ^{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 a new medicine called Mim8, which is injected under the skin to help prevent bleeding in children with haemophilia A. The study includes children with and without inhibitors. Mim8 works by aiding the blood clotting process. Mim8 is a next-generation treatment developed for regular use to prevent bleeding in hemophilia A.

Research Team

Clinical Transparency (dept. 2834)

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

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)

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

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

Timeline

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

Treatment Details

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.

Participant Groups

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

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

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

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

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

4. 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]

5. 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]