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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

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 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

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%

BACK PAIN

29%

PHARYNGITIS

29%

ODYNOPHAGIA

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%

CHEST PAIN

14%

TOOTHACHE

14%

CHOLELITHIASIS OBSTRUCTIVE

14%

COVID-19

14%

MYALGIA

14%

COMPLICATION ASSOCIATED WITH DEVICE

14%

MUSCULOSKELETAL CHEST PAIN

14%

JOINT CONTRACTURE

14%

SUBCUTANEOUS ABSCESS

14%

HEAD INJURY

14%

DEVICE BREAKAGE

14%

FALL

14%

JOINT LOCK

14%

TONGUE INJURY

14%

EXOSTOSIS

14%

GREATER TROCHANTERIC PAIN SYNDROME

14%

ARTHRITIS

14%

TINEA CAPITIS

14%

GINGIVAL INJURY

14%

PARAESTHESIA

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%

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

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 3

~620

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor

1,541 Previous Clinical Trials

568,103 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

Blood AdvancesJournal

ASH ISTH NHF WFH 2021 Guidelines on the Management of Von Willebrand Disease

Connell, Nathan T., Veronica H. Flood, Romina Brignardello-Petersen, Rezan Abdul-Kadir, Alice Arapshian, Susie Couper, Jean M. Grow, et al.. 2021. “ASH ISTH NHF WFH 2021 Guidelines on the Management of Von Willebrand Disease”. Blood Advances. American Society of Hematology. doi:10.1182/bloodadvances.2020003264.

Seminars in Thrombosis and HemostasisJournal

Prophylaxis in Von Willebrand Disease: Coming of Age?

Saccullo, Giorgia, and Mike Makris. 2016. “Prophylaxis in Von Willebrand Disease: Coming of Age?”. Seminars in Thrombosis and Hemostasis. Georg Thieme Verlag KG. doi:10.1055/s-0036-1581106.

Blood AdvancesJournal

ASH ISTH NHF WFH 2021 Guidelines on the Diagnosis of Von Willebrand Disease

James, Paula D., Nathan T. Connell, Barbara Ameer, Jorge Di Paola, Jeroen Eikenboom, Nicolas Giraud, Sandra Haberichter, et al.. 2021. “ASH ISTH NHF WFH 2021 Guidelines on the Diagnosis of Von Willebrand Disease”. Blood Advances. American Society of Hematology. doi:10.1182/bloodadvances.2020003265.

Blood AdvancesJournal