Treatment for von Willebrand Disease, Type 3

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Bleeding and Clotting Disorders Institute, Peoria, IL
von Willebrand Disease, Type 3+3 More
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.

Eligible Conditions

  • von Willebrand Disease, Type 3
  • Concomitant VWD and Hemophilia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for von Willebrand Disease, Type 3

Study Objectives

2 Primary · 3 Secondary · Reporting Duration: 18 months

12 months
Decreased bleed occurrence
18 months
Diminish bleed severity
Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A
Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A
Improve health related QOL in study participants
Reduce produce use during surgery
Reduce produce use for spontaneous or traumatic bleeds
Reduce self-reported treatment burden for HMB
Reduced treatment burdent vs VWF/FVIII prophylaxis

Trial Safety

Safety Progress

1 of 3

Other trials for von Willebrand Disease, Type 3

Trial Design

0 Treatment Group

40 Total Participants · 0 Treatment Group

Primary Treatment: Treatment · No Placebo Group · Phase 1

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 18 months

Trial Background

Jonathan Roberts, Assistance Medical Director
Principal Investigator
Bleeding and Clotting Disorders Institute Peoria, Illinois
Closest Location: Bleeding and Clotting Disorders Institute · Peoria, IL
2014First Recorded Clinical Trial
0 TrialsResearching von Willebrand Disease, Type 3
11 CompletedClinical Trials

Eligibility Criteria

Age Any Age · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a history of bleeding disorder and you have a positive test result for VWD/hemophilia A.
You plan to be adherent to emicizumab prophylaxis during the study.
Patient's bleeding phenotype necessitating prophylaxis per treating provider recommendations.
You are currently on a prophylaxis agent for VWD or VWD/hemophilia A.
This study is evaluating whether the following contraceptive methods are effective: male sterilization, hormonal contraceptives, intrauterine devices, and copper intrauterine devices.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References