What side effects are associated with this type of intervention?

Common treatments for Hemophilia A, such as recombinant factor VIII and extended half-life factor VIII products, can cause side effects including the development of inhibitors (antibodies that neutralize the effectiveness of the treatment), allergic reactions, and injection site reactions. Emicizumab, another treatment option, may cause injection site reactions, headache, and in rare cases, thrombotic events and thrombotic microangiopathy. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

Several FDA-approved treatments for Hemophilia A involve the replacement of clotting factor VIII. These include recombinant factor VIII (rFVIII) products such as Kogenate, which has been studied extensively for its safety and efficacy in treating bleeding episodes. Another notable product is Eloctate, a recombinant factor VIII-Fc fusion protein that extends the half-life of factor VIII, allowing for less frequent dosing. Additionally, pegylated factor VIII products have been developed to further prolong the half-life and improve treatment convenience. These treatments aim to prevent and control bleeding episodes by replacing the deficient clotting factor VIII in patients with Hemophilia A.

What other types of research exist?

Promising novel alternatives to Mim8 for Hemophilia A patients include gene therapy, such as adeno-associated virus mediated gene transfer, which has shown sustained elevation of clotting factors. Additionally, recombinant factor VIII products like Simoctocog Alfa (Nuwiq) and other long-acting recombinant factors are also viable options. These treatments offer different mechanisms of action and have demonstrated efficacy in clinical trials.

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Replacement 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

Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: ≥5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed

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

Must not have

Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above 1.5 times the upper limit measured at screening

Ongoing or planned immune tolerance induction (ITI) therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up no prophylaxis treatment (arms 1, 2a and 2b): from randomisation (week 0) to end of main (week 26) prophylaxis treatment (arms 3 and 4): from initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing Mim8, a new medicine to prevent bleeding in people with haemophilia A. It works by replacing the missing clotting protein. The study includes all patients with haemophilia A.

Who is the study for?

Adults and adolescents with Hemophilia A, either with or without inhibitors, can join this trial. They must have had treatment for Hemophilia A in the last 26 weeks and weigh at least 30 kg. Participants should be over 12 years old and willing to follow study procedures. Pregnant women, those on certain other treatments, or with conditions that could affect safety are not eligible.

What is being tested?

The trial is testing Mim8, a new medicine designed to prevent bleeding by replacing clotting factor VIII (FVIII). It's given as an injection into the stomach skinfold weekly or monthly. The study compares Mim8's effectiveness against other treatments over a period of up to 29 months.

What are the potential side effects?

While specific side effects of Mim8 aren't listed here, common side effects for hemophilia treatments include reactions at the injection site, potential development of inhibitors to the treatment itself leading to reduced effectiveness, allergic reactions, and increased risk of blood clots.

Eligibility Criteria

Inclusion Criteria

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

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I've had 5 or more bleeding episodes treated with specific medication in the last 6 months.

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I have been diagnosed with congenital haemophilia A.

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I am 12 years old or older.

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I have had at least one bleeding episode in the last 6 months that required treatment.

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My body weight is at least 30 kg.

Exclusion Criteria

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

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My liver tests are higher than normal.

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I am undergoing or planning to start therapy to boost my immune system.

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I am mentally capable and willing to follow study procedures.

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I have a major surgery scheduled after my screening.

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I have received gene therapy.

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

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My kidney function is low, with an eGFR of 30 ml/min/1.73 m^2 or less.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ no prophylaxis treatment (arms 1, 2a and 2b): from randomisation (week 0) to end of main (week 26) prophylaxis treatment (arms 3 and 4): from initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~no prophylaxis treatment (arms 1, 2a and 2b): from randomisation (week 0) to end of main (week 26) prophylaxis treatment (arms 3 and 4): from initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)

This trial's timeline: 3 weeks for screening, Varies for treatment, and no prophylaxis treatment (arms 1, 2a and 2b): from randomisation (week 0) to end of main (week 26) prophylaxis treatment (arms 3 and 4): from initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26) 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 treated bleeds

Secondary study objectives

Change in patient's joint pain score using Joint Pain Rating Scale

Change in patient's treatment burden using the Hemo-TEM (haemophilia treatment experience measure)

Change in physical function domain of PEDS-QL (pediatric quality of life inventory)

+7 more

Awards & Highlights

No Placebo-Only Group

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

Pivotal Trial

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

Trial Design

5Treatment groups

Experimental Treatment

Group I: no PPX- no PPX - Mim8 PPXQW/QMExperimental Treatment1 Intervention

Participants not receiving prophylaxis will not enter the run-in period. In arm 1, participants will be randomised to continue no prophylaxis (on-demand treatment with their Standard of Care products) or Mim8 once-weekly or once-monthly prophylaxis in agreement with investigators in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) in agreement with the investigator, either weekly or monthly Mim8 prophylaxis regimen.

Group II: no PPX - Mim8 PPXQW - Mim8 PPXQWExperimental Treatment1 Intervention

Participants not receiving prophylaxis will not enter the run-in period. In arm 2a, participants will be randomised to Mim8 once-weekly prophylaxis in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen.

Group III: no PPX - Mim8 PPXQM - Mim8 PPXQMExperimental Treatment1 Intervention

Participants not receiving prophylaxis will not enter the run-in period. In arm 2b, participants will be randomised to Mim8 once-monthly prophylaxis in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-monthy Mim8 prophylaxis regimen.

Group IV: PPX- Mim8 PPXQMExperimental Treatment1 Intervention

Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 4, participants will be randomised to once-monthly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-monthly Mim8 prophylaxis regimen.

Group V: PPX - Mim8 PPXQWExperimental Treatment1 Intervention

Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 3, participants will be randomised to once-weekly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

NNC0365-3769 (Mim8)

2020

Completed Phase 2

~480

0 / 12Eligibility 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 involve the replacement of the missing clotting factor VIII, either through recombinant or plasma-derived factor VIII concentrates. This replacement therapy is crucial as it restores the blood's ability to clot, preventing and managing bleeding episodes that can lead to severe complications. Mim8, an investigational drug, works similarly by replacing the function of the missing factor VIII, offering a potential new option for effective prophylactic and on-demand treatment in patients with or without inhibitors.

The management of cardiovascular diseases in patients with hemophilia.