RECRUITING

A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

Description

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.

Study Overview

Study Details

Study overview

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.

A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease

A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

Condition
Von Willebrand Disease (VWD)
Intervention / Treatment

-

Contacts and Locations

Phoenix

Phoenix Children's Hospital, Phoenix, Arizona, United States, 85016

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202-3591

Miami

University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center, Miami, Florida, United States, 33136

Atlanta

Emory Children's Center, Atlanta, Georgia, United States, 30322

Indianapolis

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, United States, 46260

New Orleans

Tulane University School of Medicine, New Orleans, Louisiana, United States, 70112-2699

Ann Arbor

University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders, Ann Arbor, Michigan, United States, 48109

Rochester

Mayo Clinic - Rochester, Rochester, Minnesota, United States, 55905

Portland

Oregon Health & Science University, Portland, Oregon, United States, 97239-3098

Dallas

The University of Texas Southwestern Medical Center, Dallas, Texas, United States, 75390

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Has the ability to provide informed consent to participate in the study, in accordance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guideline E6 (R2) (2016) and applicable national and international regulations, before any protocol directed interventions are carried out.
  • 2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
  • 3. Is 16 years or older at the time of screening.
  • 4. First approximately 50 participants: congenital Type 1 VWD with a residual VWF antigen and/or activity less than 40 IU/dL (40%)
  • 5. Has symptomatic disease as defined by a history of bruising or bleeding events, and typically experiencing bleeding symptoms every month.
  • 1. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
  • 2. Has a significant family history of unprovoked thromboembolic events in first degree relatives.
  • 3. Has a congenital or acquired bleeding disorder other than VWD.
  • 4. Has planned major surgery within the next 6 months.
  • 5. Is pregnant or plans to become pregnant within the next 6 months.
  • 6. Has any concurrent disease, treatment (including ongoing anticoagulation, antiplatelet, or non-steroidal anti-inflammatory drugs), condition, medication, or abnormality in clinical laboratory tests which may impact on the participant's bleeding symptoms or affect their ability to complete the study, in the Investigator's opinion.
  • 7. Has received any investigational product within 30 days prior to screening.

Ages Eligible for Study

16 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Hemab ApS,

VP of Clinical Research, STUDY_DIRECTOR, Hemab ApS

Study Record Dates

2026-02