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

Purpose

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.(e.g. Velora Pioneer)

Conditions

  • Von Willebrand Disease (VWD)
  • Von Willebrand Disease (VWD), Type 1
  • Von Willebrand Disease (VWD), Type 2
  • Von Willebrand Disease (VWD), Type 3
  • Von Willebrand Disease, Type 2A
  • Von Willebrand Disease, Type 2M
  • Von Willebrand Disease, Type 2N

Eligibility

Eligible Ages
Over 16 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations. 2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions. 3. ≥ 16 years at the time of screening. 4. Has congenital Type 1 VWD with a residual VWF antigen and/or activity <30 IU/dL and/or meets the bleeding event rate inclusion criteria. Other congenital VWD subtypes may be enrolled with Sponsor approval. 5. Has symptomatic disease as defined by a history of bruising or bleeding events, with an expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) per year that require treatment to control bleeding symptoms, and/or has recurrent and ongoing episodes of heavy menstrual bleeding at the time of enrollment.

Exclusion Criteria

  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. If the participant was enrolled and dosed in Velora Pioneer (study HMB-002-102; NCT06754852), they must have completed their End of Study Visit.

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL)
  • Other: Clinical outcomes of patients with VWD, Type 1
    Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1
VWD Type 2A, Type 2M, Type 2N, or Type 3
  • Other: Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
    Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.

Recruiting Locations

Tulane University School of Medicine
New Orleans, Louisiana 70112-2699
Contact:
Melody S. Benton
504-988-3596
mbenton@tulane.edu

More Details

Status
Recruiting
Sponsor
Hemab ApS

Study Contact

Clinical Trials (USA; UK)
+1 888 493 8148; 080 8304 6409
Clinicaltrials@hemab.com

Detailed Description

This is a prospective, screening study in participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with confirmed Type 1 VWD and associated bleeding symptoms will be enrolled. The study may also be opened to participants with Type 2 and Type 3 VWD with Sponsor approval. Up to a total of 200 participants may be enrolled in the study. The study includes screening, a baseline evaluation, and an approximately 4 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.