What side effects are associated with this type of intervention?

Common treatments for Haemophilia A, such as Emicizumab and recombinant factor VIII (rFVIII) products, have known side effects. Emicizumab, administered subcutaneously, can cause injection-site reactions, and there is a risk of developing unusual thrombotic events and thrombotic microangiopathy (TMA), particularly when used with activated prothrombin complex concentrate (aPCC). Recombinant factor VIII products can lead to the development of inhibitors (antibodies against factor VIII), allergic reactions, and infusion-related reactions. Both treatments aim to reduce bleeding episodes but require careful monitoring for these potential adverse effects.

What prior FDA approvals exist?

The FDA has approved several treatments for the prevention of bleeds in Hemophilia A. Emicizumab, a subcutaneous injection, is approved for prophylaxis in patients with or without inhibitors and has shown significant reduction in bleeding rates. It is administered with a loading dose followed by maintenance doses at various intervals (weekly, bi-weekly, or monthly). Another approved treatment is rFVIII-Fc (Eloctate), a recombinant factor VIII product fused with an Fc domain to extend its half-life, allowing for less frequent dosing compared to standard factor VIII. These treatments aim to maintain factor levels and reduce bleeding episodes, similar to the goals of the Mim8 trial.

What other types of research exist?

Promising novel alternatives to Mim8 for the prevention of bleeds in hemophilia A patients include: 1) Emicizumab, a monoclonal antibody that has shown significant reduction in annualized bleeding rates in both patients with and without inhibitors. 2) Concizumab, another monoclonal antibody targeting TFPI, which has demonstrated a significant reduction in bleeding rates in clinical trials. 3) Gene therapy approaches, such as adeno-associated virus mediated gene transfer, which have shown sustained elevation of factor levels in hemophilia B and are being explored for hemophilia A.

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Protein Therapy

Mim8 for Hemophilia A

Phase 3

Recruiting

Research Sponsored by Novo Nordisk A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Aged 1-11 years at the time of signing informed consent

Male and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records

Must not have

Known congenital or acquired coagulation disorders other than haemophilia A

Hepatic dysfunction defined as AST and/or ALT greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from run-in initiation to end of treatment (week -26 to week 52)

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, common ones associated with similar treatments include injection site reactions like pain or swelling, potential allergic reactions to medication components and increased risk of developing antibodies against the medicine which might reduce its effectiveness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1 and 11 years old.

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I have been diagnosed with congenital haemophilia A of any severity.

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I have severe haemophilia A with FVIII activity below 1%.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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My liver tests are higher than normal.

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My kidney function test shows creatinine levels above normal.

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I am not planning any major surgery after screening.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from run-in initiation to end of treatment (week -26 to week 52)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from run-in initiation to end of treatment (week -26 to week 52)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from run-in initiation to end of treatment (week -26 to week 52) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of treatment emergent adverse events

Secondary study objectives

Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure)

Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales

Consumption of factor product per bleed treatment (number of injections)

+9 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Hemophilia A include recombinant factor VIII, which replaces the deficient clotting factor, and bypassing agents like recombinant factor VIIa, which help to initiate clotting in the presence of inhibitors. Newer treatments, such as Mim8, are designed for the prevention of bleeds by mimicking the function of factor VIII or enhancing the body's natural clotting mechanisms. These treatments are crucial for Hemophilia A patients as they help to prevent spontaneous and trauma-induced bleeding episodes, thereby reducing the risk of joint damage and other complications associated with frequent bleeding.

The management of cardiovascular diseases in patients with hemophilia.