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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 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%
MUSCULOSKELETAL CHEST PAIN
14%
EXOSTOSIS
14%
COVID-19
14%
SUBCUTANEOUS ABSCESS
14%
JOINT LOCK
14%
TONGUE INJURY
14%
COMPLICATION ASSOCIATED WITH DEVICE
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%
0%
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

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.

Find a Location

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,564 Previous Clinical Trials
570,158 Total Patients Enrolled
Bleeding and Clotting Disorders Institute Peoria, IllinoisLead Sponsor
Jonathan C Roberts, MDPrincipal InvestigatorBleeding and Clotting Disorders Institute

Media Library

Emicizumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05500807 — Phase 1
Von Willebrand Disease Research Study Groups: Open Label Emicizumab
Von Willebrand Disease Clinical Trial 2023: Emicizumab Highlights & Side Effects. Trial Name: NCT05500807 — Phase 1
Emicizumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05500807 — Phase 1
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