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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
age >= 2
diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding <= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB < 50 U/dl based on historical medical records of study site.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 18 months
Awards & highlights
No Placebo-Only Group

BCDI-XII Trial Summary

This trial is testing whether a new drug, Emicizumab, is safe and effective for people with Von Willebrand Disease, a common inherited bleeding disorder.

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.Check my eligibility
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.See study design
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.

BCDI-XII Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 2 years old.
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I have a severe type of bleeding disorder confirmed by genetic tests.
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I have been diagnosed with VWD or mild to severe hemophilia A.
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I need treatment to prevent bleeding as advised by my doctor.
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I have VWD or VWD/hemophilia A, haven't taken emicizumab for 18 months, and am willing to stop my current treatment.
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I agree to use effective birth control or remain abstinent during the study.
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I am using a highly effective birth control method or my partner is sterilized.
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I am at least 2 years old.
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I have been diagnosed with severe type 3 Von Willebrand Disease or a variant confirmed by genetic testing.
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I have been diagnosed with VWD or mild to severe hemophilia A.
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I plan to follow the emicizumab treatment as prescribed during the study.

BCDI-XII Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
Decreased bleed occurrence
Hemorrhage
Reduced treatment burdent vs VWF/FVIII prophylaxis
Other outcome measures
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%
PHARYNGITIS
29%
CONTUSION
29%
BACK PAIN
29%
UPPER RESPIRATORY TRACT INFECTION
29%
URINARY TRACT INFECTION
29%
HYPERTENSION
29%
Arthralgia
29%
Osteoarthritis
29%
Headache
29%
ODYNOPHAGIA
29%
Upper respiratory tract infection
29%
Back pain
29%
ABDOMINAL PAIN
29%
EAR INFECTION
14%
MUSCULOSKELETAL CHEST PAIN
14%
JOINT LOCK
14%
JOINT CONTRACTURE
14%
DEVICE BREAKAGE
14%
CHOLELITHIASIS OBSTRUCTIVE
14%
PARAESTHESIA
14%
SUBCUTANEOUS ABSCESS
14%
MYALGIA
14%
TINEA CAPITIS
14%
ARTHRITIS
14%
EXOSTOSIS
14%
FALL
14%
GREATER TROCHANTERIC PAIN SYNDROME
14%
CHEST PAIN
14%
HEAD INJURY
14%
GINGIVAL INJURY
14%
Injection site reaction
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%
TOOTHACHE
14%
COMPLICATION ASSOCIATED WITH DEVICE
14%
TONGUE INJURY
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%
COVID-19
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.

BCDI-XII 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 4
~620

Find a Location

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,538 Previous Clinical Trials
567,944 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any vacancies in the clinical trial for participants?

"Clinicaltrials.gov reveals that this medical trial is no longer enrolling patients, with its launch date being November 1st 2022 and the last edit to its information on August 12th 2022. Fortunately, there are 100 other clinical studies recruiting participants at present."

Answered by AI

Has this treatment been sanctioned by the Federal Drug Administration?

"At Power, we assigned a score of 1 to this medication's safety rating due to its Phase 1 status. This means that there is minimal evidence supporting the efficacy and security of the treatment."

Answered by AI

Does the trial include participants over 40 years of age?

"This medical experiment is recruiting participants aged between 2 and 90 years."

Answered by AI

Does my profile fit the criteria for participating in this experiment?

"This trial is targeting 40 people between the ages of 2 and 90 diagnosed with von Willebrand Disease type 3, who also meet other criteria. These include a signed informed consent form, ability to comply with protocol at the investigator's discretion, diagnosis of severe VWD type 3 or milder variants confirmed by genetic mutation or VWF antigen activity/collagen binding levels below 50 U/dl; for menstruating individuals an agreement to remain abstinent (no heterosexual intercourse) or use contraceptive methods resulting in <1% failure rate per year during study period; plan to be adherent to emicizumab prophylaxis as recommended by"

Answered by AI
Recent research and studies
~22 spots leftby Nov 2025