Mim8 for Hemophilia A

Recruiting in Cleveland (>99 mi)

+67 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Novo Nordisk A/S

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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.

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.

+4 more

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.

+12 more

Participant Groups

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.

1Treatment groups

Experimental Treatment

Group I: Mim8Experimental Treatment1 Intervention

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