What is the mechanism of action of this treatment?

Von Willebrand Disease (VWD) is commonly treated with Von Willebrand Factor (VWF) concentrates, which are administered intravenously to replace the deficient or defective VWF, thereby aiding in platelet adhesion and aggregation at sites of vascular injury. Desmopressin (DDAVP) is another treatment that stimulates the release of VWF stored in the endothelial cells. Emicizumab, although primarily used for Hemophilia A, is being studied for VWD due to its ability to bind activated FIX and FX, mimicking the function of FVIII, which is crucial for the formation of a stable blood clot. These treatments are vital for VWD patients as they directly address the underlying clotting deficiencies, reducing the risk of severe bleeding episodes and improving overall hemostatic control.

What side effects are associated with this type of intervention?

Common treatments for Von Willebrand Disease (VWD) include Emicizumab and VWF concentrates. Emicizumab, which mimics FVIII cofactor functionality, can cause injection site reactions, headache, and arthralgia, with rare but serious risks of thrombotic microangiopathy (TMA) and thromboembolism. VWF concentrates may lead to allergic reactions, thrombotic events, and infusion-related reactions.

What prior FDA approvals exist?

Von Willebrand Disease (VWD) is typically treated with Von Willebrand factor (VWF) concentrates, which are administered intravenously to manage severe bleeding events. These treatments have been approved by the FDA and are the standard care for VWD. Emicizumab, a monoclonal bispecific antibody that binds activated FIX and FX to mimic FVIII cofactor functionality, is currently being studied for its efficacy and safety in VWD and concomitant VWD/hemophilia patients. While Emicizumab is approved for hemophilia A, its use in VWD is still under investigation.

What other types of research exist?

Concizumab and Marstacimab are promising novel alternatives to Emicizumab for Von Willebrand Disease patients. Concizumab is a monoclonal antibody against TFPI that has shown significant reduction in annualized bleeding rates in clinical trials. Marstacimab is another monoclonal antibody against TFPI, which can be administered subcutaneously or intravenously and has demonstrated good hemostatic efficacy in early studies.

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Monoclonal Antibodies

Emicizumab for Von Willebrand Disease (BCDI-XII Trial)

Phase 1

Recruiting

Led By Jonathan C Roberts, MD

Research Sponsored by Bleeding and Clotting Disorders Institute Peoria, Illinois

Eligibility Criteria Checklist

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

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 18 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests Emicizumab, an injectable medication that helps blood to clot, in patients with severe Von Willebrand Disease (VWD) and those with both VWD and hemophilia A. These patients often have severe bleeding that current treatments can't easily manage. Emicizumab works by mimicking a natural blood-clotting protein to help prevent bleeding. It has been approved globally for reducing bleeding in hemophilia A patients.

Who is the study for?

This trial is for individuals with severe Von Willebrand Disease (VWD) or VWD combined with Hemophilia A. Participants must be at least 2 years old, willing to adhere to the study's protocol and use emicizumab prophylaxis. They should not have used emicizumab in the past 18 months unless switching from a different prophylaxis. Menstruating participants must agree to effective contraception or abstinence.

What is being tested?

The trial is testing Emicizumab, a monoclonal antibody designed as a preventive treatment for severe bleeding in patients with VWD and those who also have Hemophilia A. It aims to show that Emicizumab can safely replace intravenous treatments by being administered through subcutaneous injections.

What are the potential side effects?

Potential side effects of Emicizumab may include reactions at the injection site, headaches, joint pain, general discomfort, and possibly an increased risk of developing blood clots or experiencing abnormal bleeding if underlying conditions are present.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 18 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A

Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A

Secondary study objectives

Decreased bleed occurrence

Hemorrhage

Reduced treatment burdent vs VWF/FVIII prophylaxis

Other study objectives

Improve health related QOL in study participants

Operative Surgical Procedures

Reduce produce use for spontaneous or traumatic bleeds

+1 more

Side effects data

From 2022 Phase 3 trial • 48 Patients • NCT03020160

57%

ARTHRALGIA

57%

HEADACHE

43%

OSTEOARTHRITIS

43%

SYNOVITIS

29%

BACK PAIN

29%

ODYNOPHAGIA

29%

PHARYNGITIS

29%

CONTUSION

29%

EAR INFECTION

29%

UPPER RESPIRATORY TRACT INFECTION

29%

URINARY TRACT INFECTION

29%

HYPERTENSION

29%

Arthralgia

29%

Osteoarthritis

29%

Headache

29%

Upper respiratory tract infection

29%

Back pain

29%

ABDOMINAL PAIN

14%

EXOSTOSIS

14%

COMPLICATION ASSOCIATED WITH DEVICE

14%

COVID-19

14%

JOINT LOCK

14%

SUBCUTANEOUS ABSCESS

14%

MUSCULOSKELETAL CHEST PAIN

14%

TONGUE INJURY

14%

JOINT CONTRACTURE

14%

HEAD INJURY

14%

GREATER TROCHANTERIC PAIN SYNDROME

14%

Injection site reaction

14%

TINEA CAPITIS

14%

FALL

14%

GINGIVAL INJURY

14%

CHEST PAIN

14%

CHOLELITHIASIS OBSTRUCTIVE

14%

TOOTHACHE

14%

ARTHRITIS

14%

DEVICE BREAKAGE

14%

MYALGIA

14%

PARAESTHESIA

14%

MEDICAL DEVICE DISCOMFORT

14%

POST PROCEDURAL INFLAMMATION

14%

ANXIETY

14%

RASH

14%

Temporomandibular joint syndrome

14%

Post procedural inflammation

14%

INFLAMMATION

14%

INJECTION SITE REACTION

14%

Dyspepsia

14%

Device related infection

14%

Contusion

14%

Synovitis

14%

Tendon disorder

14%

Joint lock

14%

Cholelithiasis

14%

Myalgia

14%

HYPERCHOLESTEROLAEMIA

14%

VITAMIN D DEFICIENCY

14%

Abdominal pain

14%

Tongue injury

14%

Eczema eyelids

14%

Ear infection

14%

Osteitis

14%

Pharyngitis

14%

Hypertension

14%

Musculoskeletal chest pain

14%

IRON DEFICIENCY ANAEMIA

14%

ECZEMA EYELIDS

14%

DIARRHOEA

14%

DYSPEPSIA

14%

PYREXIA

14%

SEASONAL ALLERGY

14%

DEVICE RELATED INFECTION

14%

SPINAL OSTEOARTHRITIS

14%

TEMPOROMANDIBULAR JOINT SYNDROME

14%

TENDON DISORDER

14%

MOTOR DYSFUNCTION

100%

80%

60%

40%

20%

Study treatment Arm

Emicizumab: PK Run-In Cohort

Emicizumab: Expansion Cohort

Awards & Highlights

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: Open Label EmicizumabExperimental Treatment1 Intervention

Emicizumab prophylaxis

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Emicizumab

2016

Completed Phase 3

~620

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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

Von Willebrand Disease (VWD) is commonly treated with Von Willebrand Factor (VWF) concentrates, which are administered intravenously to replace the deficient or defective VWF, thereby aiding in platelet adhesion and aggregation at sites of vascular injury. Desmopressin (DDAVP) is another treatment that stimulates the release of VWF stored in the endothelial cells. Emicizumab, although primarily used for Hemophilia A, is being studied for VWD due to its ability to bind activated FIX and FX, mimicking the function of FVIII, which is crucial for the formation of a stable blood clot. These treatments are vital for VWD patients as they directly address the underlying clotting deficiencies, reducing the risk of severe bleeding episodes and improving overall hemostatic control.