What side effects are associated with this type of intervention?

The most common treatments for Hemophilia A include factor VIII replacement therapy and newer agents like Emicizumab. Factor VIII replacement therapy can cause side effects such as the development of inhibitors (antibodies against factor VIII), allergic reactions, and infusion-related reactions. Emicizumab, a bispecific monoclonal antibody, has been associated with injection site reactions, headache, and, rarely, thrombotic microangiopathy (TMA) and thromboembolic events. Overall, Emicizumab is well-tolerated with a favorable safety profile compared to traditional factor VIII replacement.

What prior FDA approvals exist?

Emicizumab, a bispecific monoclonal antibody that bridges activated factor IX and factor X to restore the function of missing factor VIII, has been approved by the FDA for prophylaxis in Hemophilia A. Other FDA-approved treatments for Hemophilia A include recombinant factor VIII products such as BAY 81-8973, rVIII-SingleChain, and recombinant factor VIII Fc fusion protein, which are used for both prophylactic and on-demand treatment. These treatments aim to replace the missing factor VIII, thereby preventing or controlling bleeding episodes.

What other types of research exist?

Promising novel alternatives to Emicizumab for Hemophilia A patients include bispecific antibodies like ABT-122 and COVA322, which are designed to target multiple pathways. Additionally, chimeric-antigen-receptor (CAR) T cell therapies and antibody-drug-conjugates (ADCs) are emerging as potential treatments. These therapies are in various stages of development and have shown potential in preclinical and early clinical studies.

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Factor IX + Emicizumab for Hemophilia A

Q: What is the mechanism of action of this treatment?

The most common treatments for Hemophilia A focus on replacing or mimicking the function of the missing factor VIII, which is essential for blood clotting. Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX and factor X to restore the function of factor VIII, helping to form stable blood clots. This is particularly important for Hemophilia A patients as it prevents or controls bleeding episodes. Other treatments include recombinant factor VIII products, which directly replace the missing factor, and bypassing agents like recombinant activated factor VII (rFVIIa) for patients with inhibitors. These treatments are vital for managing bleeding risks and enhancing the quality of life for those with Hemophilia A.

Phase < 1

Recruiting

Led By Maissa Janbain, MD, MS

Research Sponsored by Tulane University

Eligibility Criteria Checklist

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

Must have

Diagnosis of severe hemophilia A and are receiving emicizumab prophylaxis, on a weekly maintenance regimen

Male patients, Age 12 years and older at time of signing Informed Consent Form

Must not have

Not able to sign consent

Patients carrying the diagnoses of other coagulopathies in addition to hemophilia A

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is testing different blood-clotting substances in patients with severe hemophilia A who are already on Emicizumab. The goal is to see which substance helps their blood clot better. Researchers will measure the effectiveness of these substances in helping the blood to form clots.

Who is the study for?

This trial is for male patients aged 12 and older with severe hemophilia A who are already on a weekly emicizumab prophylaxis regimen. Participants must be able to sign a consent form and follow the study protocol. Those who have used any hemostatic agents recently or have other coagulopathies, or are unwilling to provide extra blood samples, cannot join.

What is being tested?

The trial is testing Factor IX as an additional treatment alongside emicizumab in patients with severe hemophilia A. It's a preclinical pilot study where patient blood will be tested in the lab with different molecules to see how they work together.

What are the potential side effects?

Since this is an in vitro (lab-based) study using patients' blood samples, there's minimal direct risk involved. Therefore, no significant side effects from interventions are expected for participants.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have severe hemophilia A and am on a weekly emicizumab treatment.

Select...

I am a male aged 12 or older.

Exclusion Criteria

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

Select...

I am unable to give consent by myself.

Select...

I have hemophilia A and another blood clotting disorder.

Select...

I haven't taken any blood clotting medication recently.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)

Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin

Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)

+1 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

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: Patient GroupExperimental Treatment1 Intervention

In vitro spiking experiments will be realized in plasmas from patients with severe haemophilia A on emicizumab using increasing concentrations of factor IX (rFIX), Activated prothrombin complex (aPCC) and recombinant VIIa (rFVIIa).

0 / 5Eligibility 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 focus on replacing or mimicking the function of the missing factor VIII, which is essential for blood clotting. Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX and factor X to restore the function of factor VIII, helping to form stable blood clots. This is particularly important for Hemophilia A patients as it prevents or controls bleeding episodes. Other treatments include recombinant factor VIII products, which directly replace the missing factor, and bypassing agents like recombinant activated factor VII (rFVIIa) for patients with inhibitors. These treatments are vital for managing bleeding risks and enhancing the quality of life for those with Hemophilia A.